The 2021 Cell & Gene Meeting on the Mesa was delivered in a hybrid format with live programming available over the course of three days. Virtual registrants had access to all content via livestream during the program dates. Additionally, all content was made available on-demand within 24 hours of the live program time for all registrants to view.
ON-DEMAND VIRTUAL CONTENT
Available Beginning Tuesday, October 12, 2021
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)
DOING BUSINESS IN JAPAN – AN EVOLVING MARKET WORKSHOP
Promoting Harmonization and Commercialization of Regenerative Medicines Among Asian Countries/Regions
The Asia Partnership Conference of Regenerative Medicine (APACRM), is aimed at harmonizing and optimizing regulations among Asian countries and regions. FIRM has organized the APACRM since 2018 and has closely collaborated with industrial groups such as the China Medicinal Biotech Association (CMBA) in China; Association of Biotechnology Led Enterprises (ABLE) in India; Council for Advanced Regenerative Medicine (CARM) and Strategic Center for Regenerative Medicine (SCRM) in Korea; Singapore Association of Pharmaceuticals Industries (SAPI) in Singapore; and Biotechnology and Pharmaceutical Industries Promotion Office (BPIPO) in Taiwan. This session will focus on prospects and challenges for or against regenerative medicine to promote harmonization and optimization of the regulations on regenerative medicine products among Asian countries and regions.
Chair:
Masayuki (Max) Nomura, Ph.D., Chair, International Affairs Committee, Forum for Innovative Regenerative Medicine (FIRM)
Opening Remarks:
Kozo Saiki, Director, Bio-Industry Division, Ministry of Economy, Trade and Industry (METI)
Speakers:
Tomohiro Yoneda, New Modality and External Innovation Lead, PCS and Pharmacology, Translational Medicine, Novartis Pharma K.K.
Hiroshi Karasawa, Ph.D., Scientist, Cell Therapy Research Laboratories, Daiichi Sankyo Co.
Sponsored by Charles River Laboratories
FROM CONCEPT TO COMMERCIALIZATION IN LIVING THERAPEUTICS: HINDSIGHT IS 20/20 WORKSHOP
Challenges for developing a cell therapy product evolve and shift, and reducing these risks requires experience and expertise to execute around them. From discovery through late-phase clinical trials and into commercial manufacturing, different challenges and competing priorities are ever present – from coordinating multiple CROs/CDMOs, managing clinical operations effectively, and overcoming supply chain and logistic hurdles, all while keeping in line with regulatory standards. This discussion will take a look at these and many more real-world challenges and focus on the strategies involved with mitigating risks along the way while reducing the amount of lag time involved with bringing a cell therapy product to market.
Chair:
Matthew Hewitt, Ph.D., Senior Director, Scientific Solutions Cell and Gene Therapy, Charles River Laboratories
Speakers:
Douglas Doerfler, CEO, MaxCyte
Howard Federoff, M.D., Ph.D., President and CEO, Brooklyn ImmunoTherapeutics; Senior Advisor, Aspen Neuroscience
Kristin Yarema, Ph.D., Chief Commercial Officer, Atara Biotherapeutics
Sponsored by Pfizer
BESPOKE THERAPIES FOR ULTRA-RARE DISEASE
This session will explore the recently formed National Institutes of Health (NIH)’s, Foundation for the NIH, FDA’s and the biopharmaceutical industry’s innovative public-private partnership to help individualized gene therapies reach patients despite their lack of commercial viability. Panelists will provide an overview of this initiative including how it will maximize benefits to patients, as well as explore the specific opportunities and challenges related to its success.
Chair:
Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer Inc.
Speakers:
Philip John (P.J.) Brooks, Ph.D., Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)
Gregory LaRosa, Ph.D., Vice President, Head of Scientific Research, Rare Disease Research Unit, Pfizer Inc.
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
Courtney Silverthorn, Ph.D., Associate Vice President, Research Partnerships, Foundation for the National Institutes of Health (FNIH)
PRE-RECORDED VIRTUAL COMPANY PRESENTATIONS
Pre-recorded virtual company presentations will be available on-demand within the platform for all delegates to view starting October 12, 2021.
Accelerated Biosciences | Achilles Therapeutics | Adaptimmune | Advarra | AGC Biologics | AlloVir | American Gene Technologies | Amniotics | Andelyn Biosciences | ARMI | Aruvant | AskBio | Atara Biotherapeutics | Autolus | Avectas | Biogen | BrainStorm Cell Therapeutics | Cabaletta Bio | Caribou Biosciences | Carisma Therapeutics | Cellares | Cellino | CellPort Software | Cellusion | CEVEC | cGMPnow | Corning | CRC Oncology | Cryoport Systems | Cynata Therapeutics | Decibel Therapeutics | FluidForm | FUJIFILM Cellular Dynamics | G-CON Manufacturing | Gamida Cell | Genethon | Genocea Biosciences | GenSight Biologics | GPB Scientific | Healios | HemaCare | I Peace | InsightRX | Invetech | Iovance Biotherapeutics | Kytopen | Notch Therapeutics | Opsis Therapeutics | Polyplus-transfection | Precigen | Precision BioSciences | PTC Therapeutics | Regenerative Patch Technologies | Regeneus | ReNeuron | RoosterBio | SanBio | Santen Pharmaceutical | SCM Lifescience | Sernova | Sio Gene Therapies | SmartLabs | SwanBio Therapeutics | Talaris Therapeutics | Teknova | Univercells Technologies | VectorY Therapeutics | Vigene Biosciences | VisiCELL Medical | VIVEbiotech
BIDEN ADMINISTRATION PLANS FOR LIFE SCIENCE INNOVATION
Chair:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Speaker:
Carrie Wolinetz, Ph.D., Deputy Director for Health and Life Sciences, White House Office of Science and Technology Policy
FEATURED FIRESIDE CHAT: FDA PERSPECTIVE
Chair:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Speaker:
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
IN-PERSON PROGRAM AND LIVESTREAM
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by KBI Biopharma
7:15am – 8:45am | CONCURRENT WORKSHOPS
7:15am – 8:45am | BlueRock Therapeutics Ballroom
Sponsored by Precision ADVANCE
WHAT’S NEXT FOR ADVANCED THERAPIES WORKSHOP
This workshop will feature leading executives in the ATMP space who will discuss a number of topics including recent advancements in clinical trials, going from treating rare diseases to prevalent conditions, and implementing next-generation gene-editing technologies. The session will also touch upon resolving manufacturing and talent bottlenecks as the sector moves towards commercialization, as well as the evolution of reimbursement payment models.
Chair:
Anshul Mangal, President, Project Farma and Precision ADVANCE
Speakers:
Phil Cyr, Senior Vice President, Customer Solutions, Precision Value & Health
Palani Palaniappan, Ph.D., Executive Vice President and Chief Technology Officer, Aruvant Sciences
Derrell Porter, M.D., Founder and CEO, Cellevolve
RA Session II, Founder, President and CEO, Taysha Gene Therapies
Claudia Zylberberg, Ph.D., Founder and Executive Chair, Akron Biotech
7:15am – 8:45am | Aviara Salon A
Sponsored by Pointellis, powered by EY
UNLOCKING THE POWER OF COLLABORATION TO FUEL THE INDUSTRY TRANSFORMATION FOR CELL & GENE THERAPIES WORKSHOP
The groundwork for seamless cell and gene therapy (CGT) care delivery needs to be laid now. There are many stakeholders involved in the complexities of the CGT treatment journey that require a radically new approach from that of traditional mass-produced therapies. A complex balancing act of multi-stakeholder processes hinge on treatment advances, product availability, and the network’s ability to meet demand. And that’s before thinking about scaling CGT delivery to more than a few thousand patients. This workshop will bring together leading experts in managing interconnected supply and logistics networks, delivering superior patient experiences, manufacturing cell and gene therapies, and building world-class digital infrastructures.
Chair:
Adlai Goldberg, Global Digital, Social and Commercial Innovation Life Sciences Leader, EY
Speakers:
Sybil Danby, Senior Vice President, Business Development and Strategy, Center for Breakthrough Medicines
Tamie Joeckel, Global Business Lead, Cell and Gene Therapy COE, ICON
Orlando Serani, Program Lead Advanced Cellular Therapies Supply Chain, Johnson & Johnson
Julia Tarasenko, Vice President, Commercial Operations, Europe and the Americas, Marken, a UPS Healthcare company
9:00am | GENERAL SESSION AND PARTNERING OPENS
WELCOME REMARKS
{AVROBIO Ballroom}
9:00am – 9:15am
Speakers:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM); Chairman, BlueRock Therapeutics
PLENARY SESSION: CELLULAR IMMUNOTHERAPY PIONEERS – LIQUID VS. SOLID TUMOR STRATEGIES
{AVROBIO Ballroom}
9:15am – 10:15am
Chair:
Usman “Oz” Azam, M.D., President and CEO, Tmunity Therapeutics
Speakers:
Kanya Rajangam, M.D., Ph.D., Chief Medical Officer, Nkarta Therapeutics
Devon Shedlock, Ph.D., Senior Vice President, Research and Development, Poseida Therapeutics
Pascal Touchon, President and CEO, Atara Biotherapeutics
ON-DEMAND VIRTUAL CONTENT
Available Beginning Tuesday, October 12, 2021
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)
DOING BUSINESS IN JAPAN – AN EVOLVING MARKET WORKSHOP
Promoting Harmonization and Commercialization of Regenerative Medicines Among Asian Countries/Regions
The Asia Partnership Conference of Regenerative Medicine (APACRM), is aimed at harmonizing and optimizing regulations among Asian countries and regions. FIRM has organized the APACRM since 2018 and has closely collaborated with industrial groups such as the China Medicinal Biotech Association (CMBA) in China; Association of Biotechnology Led Enterprises (ABLE) in India; Council for Advanced Regenerative Medicine (CARM) and Strategic Center for Regenerative Medicine (SCRM) in Korea; Singapore Association of Pharmaceuticals Industries (SAPI) in Singapore; and Biotechnology and Pharmaceutical Industries Promotion Office (BPIPO) in Taiwan. This session will focus on prospects and challenges for or against regenerative medicine to promote harmonization and optimization of the regulations on regenerative medicine products among Asian countries and regions.
Chair:
Masayuki (Max) Nomura, Ph.D., Chair, International Affairs Committee, Forum for Innovative Regenerative Medicine (FIRM)
Opening Remarks:
Kozo Saiki, Director, Bio-Industry Division, Ministry of Economy, Trade and Industry (METI)
Speakers:
Tomohiro Yoneda, New Modality and External Innovation Lead, PCS and Pharmacology, Translational Medicine, Novartis Pharma K.K.
Hiroshi Karasawa, Ph.D., Scientist, Cell Therapy Research Laboratories, Daiichi Sankyo Co.
Sponsored by Charles River Laboratories
FROM CONCEPT TO COMMERCIALIZATION IN LIVING THERAPEUTICS: HINDSIGHT IS 20/20 WORKSHOP
Challenges for developing a cell therapy product evolve and shift, and reducing these risks requires experience and expertise to execute around them. From discovery through late-phase clinical trials and into commercial manufacturing, different challenges and competing priorities are ever present – from coordinating multiple CROs/CDMOs, managing clinical operations effectively, and overcoming supply chain and logistic hurdles, all while keeping in line with regulatory standards. This discussion will take a look at these and many more real-world challenges and focus on the strategies involved with mitigating risks along the way while reducing the amount of lag time involved with bringing a cell therapy product to market.
Chair:
Matthew Hewitt, Ph.D., Senior Director, Scientific Solutions Cell and Gene Therapy, Charles River Laboratories
Speakers:
Douglas Doerfler, CEO, MaxCyte
Howard Federoff, M.D., Ph.D., President and CEO, Brooklyn ImmunoTherapeutics; Senior Advisor, Aspen Neuroscience
Kristin Yarema, Ph.D., Chief Commercial Officer, Atara Biotherapeutics
Sponsored by Pfizer
BESPOKE THERAPIES FOR ULTRA-RARE DISEASE
This session will explore the recently formed National Institutes of Health (NIH)’s, Foundation for the NIH, FDA’s and the biopharmaceutical industry’s innovative public-private partnership to help individualized gene therapies reach patients despite their lack of commercial viability. Panelists will provide an overview of this initiative including how it will maximize benefits to patients, as well as explore the specific opportunities and challenges related to its success.
Chair:
Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer Inc.
Speakers:
Philip John (P.J.) Brooks, Ph.D., Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)
Gregory LaRosa, Ph.D., Vice President, Head of Scientific Research, Rare Disease Research Unit, Pfizer Inc.
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
Courtney Silverthorn, Ph.D., Associate Vice President, Research Partnerships, Foundation for the National Institutes of Health (FNIH)
PRE-RECORDED VIRTUAL COMPANY PRESENTATIONS
Pre-recorded virtual company presentations will be available on-demand within the platform for all delegates to view starting October 12, 2021. Accelerated Biosciences | Achilles Therapeutics | Adaptimmune | Advarra | AGC Biologics | AlloVir | American Gene Technologies | Amniotics | Andelyn Biosciences | ARMI | Aruvant | AskBio | Atara Biotherapeutics | Autolus | Avectas | Biogen | BrainStorm Cell Therapeutics | Cabaletta Bio | Caribou Biosciences | Carisma Therapeutics | Cellares | Cellino | CellPort Software | Cellusion | CEVEC | cGMPnow | Corning | CRC Oncology | Cryoport Systems | Cynata Therapeutics | Decibel Therapeutics | FluidForm | FUJIFILM Cellular Dynamics | G-CON Manufacturing | Gamida Cell | Genethon | Genocea Biosciences | GenSight Biologics | GPB Scientific | Healios | HemaCare | I Peace | InsightRX | Invetech | Iovance Biotherapeutics | Kytopen | Notch Therapeutics | Opsis Therapeutics | Polyplus-transfection | Precigen | Precision BioSciences | PTC Therapeutics | Regenerative Patch Technologies | Regeneus | ReNeuron | RoosterBio | SanBio | Santen Pharmaceutical | SCM Lifescience | Sernova | Sio Gene Therapies | SmartLabs | SwanBio Therapeutics | Talaris Therapeutics | Teknova | Univercells Technologies | VectorY Therapeutics | Vigene Biosciences | VisiCELL Medical | VIVEbiotechBIDEN ADMINISTRATION PLANS FOR LIFE SCIENCE INNOVATION
Chair:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Speaker:
Carrie Wolinetz, Ph.D., Deputy Director for Health and Life Sciences, White House Office of Science and Technology Policy
FEATURED FIRESIDE CHAT: FDA PERSPECTIVE
Chair:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Speaker:
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
IN-PERSON PROGRAM AND LIVESTREAM
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by KBI Biopharma
7:15am – 8:45am | CONCURRENT WORKSHOPS
7:15am – 8:45am | BlueRock Therapeutics Ballroom
Sponsored by Precision ADVANCE
WHAT’S NEXT FOR ADVANCED THERAPIES WORKSHOP
This workshop will feature leading executives in the ATMP space who will discuss a number of topics including recent advancements in clinical trials, going from treating rare diseases to prevalent conditions, and implementing next-generation gene-editing technologies. The session will also touch upon resolving manufacturing and talent bottlenecks as the sector moves towards commercialization, as well as the evolution of reimbursement payment models.
Chair:
Anshul Mangal, President, Project Farma and Precision ADVANCE
Speakers:
Phil Cyr, Senior Vice President, Customer Solutions, Precision Value & Health
Palani Palaniappan, Ph.D., Executive Vice President and Chief Technology Officer, Aruvant Sciences
Derrell Porter, M.D., Founder and CEO, Cellevolve
RA Session II, Founder, President and CEO, Taysha Gene Therapies
Claudia Zylberberg, Ph.D., Founder and Executive Chair, Akron Biotech
7:15am – 8:45am | Aviara Salon A
Sponsored by Pointellis, powered by EY
UNLOCKING THE POWER OF COLLABORATION TO FUEL THE INDUSTRY TRANSFORMATION FOR CELL & GENE THERAPIES WORKSHOP
The groundwork for seamless cell and gene therapy (CGT) care delivery needs to be laid now. There are many stakeholders involved in the complexities of the CGT treatment journey that require a radically new approach from that of traditional mass-produced therapies. A complex balancing act of multi-stakeholder processes hinge on treatment advances, product availability, and the network’s ability to meet demand. And that’s before thinking about scaling CGT delivery to more than a few thousand patients. This workshop will bring together leading experts in managing interconnected supply and logistics networks, delivering superior patient experiences, manufacturing cell and gene therapies, and building world-class digital infrastructures.
Chair:
Adlai Goldberg, Global Digital, Social and Commercial Innovation Life Sciences Leader, EY
Speakers:
Sybil Danby, Senior Vice President, Business Development and Strategy, Center for Breakthrough Medicines
Tamie Joeckel, Global Business Lead, Cell and Gene Therapy COE, ICON
Orlando Serani, Program Lead Advanced Cellular Therapies Supply Chain, Johnson & Johnson
Julia Tarasenko, Vice President, Commercial Operations, Europe and the Americas, Marken, a UPS Healthcare company
9:00am | GENERAL SESSION AND PARTNERING OPENS
WELCOME REMARKS
{AVROBIO Ballroom}
9:00am – 9:15am
Speakers:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM); Chairman, BlueRock Therapeutics
PLENARY SESSION: CELLULAR IMMUNOTHERAPY PIONEERS – LIQUID VS. SOLID TUMOR STRATEGIES
{AVROBIO Ballroom}
9:15am – 10:15am
Chair:
Usman “Oz” Azam, M.D., President and CEO, Tmunity Therapeutics
Speakers:
Kanya Rajangam, M.D., Ph.D., Chief Medical Officer, Nkarta Therapeutics
Devon Shedlock, Ph.D., Senior Vice President, Research and Development, Poseida Therapeutics
Pascal Touchon, President and CEO, Atara Biotherapeutics
10:15am – 10:45am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
10:45am – 12:00pm | CONCURRENT TRACKS
LESSONS LEARNED: THE ROLE OF PATIENT ENGAGEMENT IN APPROVALS & PAYOR DISCUSSIONS
{AVROBIO Ballroom}
10:45am – 11:15am
In a pre-recorded video, a mother of a child living with SMA who received Zolgensma will share her story and what she believes healthcare decision makers should include in their evaluations of gene therapies. ARM’s Paige Bischoff will then lead a live discussion with Novartis’ Amy Nicole Nayar on lessons emerging gene therapy companies can learn from Novartis’ experience incorporating the patient perspective in discussions with regulators, payors, and health technology assessors.
Chair:
Paige Bischoff, Senior Vice President of Global Public Affairs, Alliance for Regenerative Medicine (ARM)
Speakers:
Katee DellaMaggiora, Mother of a child living with SMA and Cure SMA advocate
Amy Nicole Nayar, Pharm.D., Vice President of U.S. Patient Advocacy and Government Affairs, Novartis Gene Therapies
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 10:45am – 11:00am | Emendo Biotherapeutics |
| 11:00am – 11:15am | Ensoma |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 11:15am – 11:30am | Oxford Biomedica |
| 11:30am – 11:45am | 4DMT |
| 11:45am – 12:00pm | Helixmith |
12:00pm – 1:15pm | LUNCH
Sponsored by Pall Corporation
1:15pm – 3:30pm | CONCURRENT TRACKS
WHERE ARE WE ON THE JOURNEY TO CURE SICKLE CELL DISEASE?
{AVROBIO Ballroom}
1:15pm – 2:15pm
The sickle cell mutation was discovered in 1956. Unfortunately, the translation of knowledge into developing treatments has been disproportionately slow and elusive…until now. In the last five years, there has been a vast increase in the number of therapies in development and the prominence of sickle cell has grown on the agendas of professional societies and the public sector. How is this combination of attention and technology bringing us closer to a cure? Hear from leading experts on the latest cell and gene therapies aiming to address this devastating disease.
Chair:
Jill Elliott, Vice President, Cell Therapy Commercialization, Novo Nordisk
Speakers:
Edmond Chen, M.D., Vice President, Head of Clinical Development, Hematology, Editas Medicine
Debbie Drane, Senior Vice President, Global Commercial Development and TA Strategy, CSL Behring
Thomas Klima, Chief Commercial Officer, bluebird bio
Sandy Macrae, Ph.D., President and CEO, Sangamo Therapeutics
Amy Simon, M.D., Chief Medical Officer, Beam Therapeutics
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 1:15pm – 1:30pm | AffyImmune Therapeutics |
| 1:30pm – 1:45pm | Angiocrine Bioscience |
| 1:45pm – 2:00pm | Artisan Bio |
| 2:00pm – 2:15pm | Catamaran Bio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 2:15pm – 2:30pm | BlueRock Therapeutics |
| 2:30pm – 2:45pm | Taysha Gene Therapies |
| 2:45pm – 3:00pm | Passage Bio |
| 3:00pm – 3:15pm | Athersys |
| 3:15pm – 3:30pm | Ginkgo Bioworks |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 2:15pm – 2:30pm | Rocket Pharmaceuticals |
| 2:30pm – 2:45pm | Luminary Therapeutics |
| 2:45pm – 3:00pm | Senti Biosciences |
| 3:00pm – 3:15pm | Shoreline Biosciences |
| 3:15pm – 3:30pm | DiscGenics |
3:30pm – 4:00pm | AFTERNOON BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
4:00pm – 6:00pm | CONCURRENT TRACKS
CURRENT REALITIES OF MAKE VS. BUY FOR CELL & GENE THERAPY MANUFACTURING
{AVROBIO Ballroom}
4:00pm – 5:00pm
This panel of leading experts will discuss key considerations and experiences related to the Make vs. Buy decision from a variety of perspectives. Speakers will debate whether those considerations are unique to today’s environment and how they might be different in the future. This session will solicit significant audience input to shape the discussion.
Chair:
Kelvin Lee, Ph.D., Institute Director, National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)
Speakers:
Melissa Carpenter, Ph.D., Chief Scientific Officer, Regenerative Medicine, ElevateBio
Thomas Fellner, Ph.D., Vice President, Global Head of Business Development, Account Management, and Program Management, Lonza Cell & Gene Technologies
Nirupama (Rupa) Pike, Ph.D., Director, Enterprise Science and Innovation Partnerships, Global Corporate Accounts, Thermo Fisher Scientific
Joe Tarnowski, Ph.D., Senior Vice President, Cell and Gene Therapy Platform, GlaxoSmithKline
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 4:00pm – 4:15pm | Cellatoz Therapeutics |
| 4:15pm – 4:30pm | Aspen Neuroscience |
| 4:30pm – 4:45pm | Labcorp |
| 4:45pm – 5:00pm | Mirus Bio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 5:00pm – 5:15pm | Amicus Therapeutics |
| 5:15pm – 5:30pm | Homology Medicines |
| 5:30pm – 5:45pm | AVROBIO |
| 5:45pm – 6:00pm | Orchard Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 5:00pm – 5:15pm | Forge Biologics |
| 5:15pm – 5:30pm | Life Edit Therapeutics |
| 5:30pm – 5:45pm | TreeFrog Therapeutics |
| 5:45pm – 6:00pm | GenEdit |
6:00pm | PROGRAM AND PARTNERING CLOSES
6:30pm – 9:30pm | NETWORKING BASH
Sponsored by Biopharma Excellence by PharmaLex, Cognate BioServices and L7 Informatics
10:15am – 10:45am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
10:45am – 12:00pm | CONCURRENT TRACKS
LESSONS LEARNED: THE ROLE OF PATIENT ENGAGEMENT IN APPROVALS & PAYOR DISCUSSIONS
{AVROBIO Ballroom}
10:45am – 11:15am
In a pre-recorded video, a mother of a child living with SMA who received Zolgensma will share her story and what she believes healthcare decision makers should include in their evaluations of gene therapies. ARM’s Paige Bischoff will then lead a live discussion with Novartis’ Amy Nicole Nayar on lessons emerging gene therapy companies can learn from Novartis’ experience incorporating the patient perspective in discussions with regulators, payors, and health technology assessors.
Chair:
Paige Bischoff, Senior Vice President of Global Public Affairs, Alliance for Regenerative Medicine (ARM)
Speakers:
Katee DellaMaggiora, Mother of a child living with SMA and Cure SMA advocate
Amy Nicole Nayar, Pharm.D., Vice President of U.S. Patient Advocacy and Government Affairs, Novartis Gene Therapies
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 10:45am – 11:00am | Emendo Biotherapeutics |
| 11:00am – 11:15am | Ensoma |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 11:15am – 11:30am | Oxford Biomedica |
| 11:30am – 11:45am | 4DMT |
| 11:45am – 12:00pm | Helixmith |
12:00pm – 1:15pm | LUNCH
Sponsored by Pall Corporation
1:15pm – 3:30pm | CONCURRENT TRACKS
WHERE ARE WE ON THE JOURNEY TO CURE SICKLE CELL DISEASE?
{AVROBIO Ballroom}
1:15pm – 2:15pm
The sickle cell mutation was discovered in 1956. Unfortunately, the translation of knowledge into developing treatments has been disproportionately slow and elusive…until now. In the last five years, there has been a vast increase in the number of therapies in development and the prominence of sickle cell has grown on the agendas of professional societies and the public sector. How is this combination of attention and technology bringing us closer to a cure? Hear from leading experts on the latest cell and gene therapies aiming to address this devastating disease.
Chair:
Jill Elliott, Vice President, Cell Therapy Commercialization, Novo Nordisk
Speakers:
Edmond Chen, M.D., Vice President, Head of Clinical Development, Hematology, Editas Medicine
Debbie Drane, Senior Vice President, Global Commercial Development and TA Strategy, CSL Behring
Thomas Klima, Chief Commercial Officer, bluebird bio
Sandy Macrae, Ph.D., President and CEO, Sangamo Therapeutics
Amy Simon, M.D., Chief Medical Officer, Beam Therapeutics
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 1:15pm – 1:30pm | AffyImmune Therapeutics |
| 1:30pm – 1:45pm | Angiocrine Bioscience |
| 1:45pm – 2:00pm | Artisan Bio |
| 2:00pm – 2:15pm | Catamaran Bio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 2:15pm – 2:30pm | BlueRock Therapeutics |
| 2:30pm – 2:45pm | Taysha Gene Therapies |
| 2:45pm – 3:00pm | Passage Bio |
| 3:00pm – 3:15pm | Athersys |
| 3:15pm – 3:30pm | Ginkgo Bioworks |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 2:15pm – 2:30pm | Rocket Pharmaceuticals |
| 2:30pm – 2:45pm | Luminary Therapeutics |
| 2:45pm – 3:00pm | Senti Biosciences |
| 3:00pm – 3:15pm | Shoreline Biosciences |
| 3:15pm – 3:30pm | DiscGenics |
3:30pm – 4:00pm | AFTERNOON BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
4:00pm – 6:00pm | CONCURRENT TRACKS
CURRENT REALITIES OF MAKE VS. BUY FOR CELL & GENE THERAPY MANUFACTURING
{AVROBIO Ballroom}
4:00pm – 5:00pm
This panel of leading experts will discuss key considerations and experiences related to the Make vs. Buy decision from a variety of perspectives. Speakers will debate whether those considerations are unique to today’s environment and how they might be different in the future. This session will solicit significant audience input to shape the discussion.
Chair:
Kelvin Lee, Ph.D., Institute Director, National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)
Speakers:
Melissa Carpenter, Ph.D., Chief Scientific Officer, Regenerative Medicine, ElevateBio
Thomas Fellner, Ph.D., Vice President, Global Head of Business Development, Account Management, and Program Management, Lonza Cell & Gene Technologies
Nirupama (Rupa) Pike, Ph.D., Director, Enterprise Science and Innovation Partnerships, Global Corporate Accounts, Thermo Fisher Scientific
Joe Tarnowski, Ph.D., Senior Vice President, Cell and Gene Therapy Platform, GlaxoSmithKline
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 4:00pm – 4:15pm | Cellatoz Therapeutics |
| 4:15pm – 4:30pm | Aspen Neuroscience |
| 4:30pm – 4:45pm | Labcorp |
| 4:45pm – 5:00pm | Mirus Bio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 5:00pm – 5:15pm | Amicus Therapeutics |
| 5:15pm – 5:30pm | Homology Medicines |
| 5:30pm – 5:45pm | AVROBIO |
| 5:45pm – 6:00pm | Orchard Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 5:00pm – 5:15pm | Forge Biologics |
| 5:15pm – 5:30pm | Life Edit Therapeutics |
| 5:30pm – 5:45pm | TreeFrog Therapeutics |
| 5:45pm – 6:00pm | GenEdit |
6:00pm | PROGRAM AND PARTNERING CLOSES
6:30pm – 9:30pm | NETWORKING BASH
Sponsored by Biopharma Excellence by PharmaLex, Cognate BioServices and L7 Informatics
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by Triangle Insights Group
7:15am – 8:45am | CONCURRENT WORKSHOPS
7:15am – 8:45am | BlueRock Therapeutics Ballroom
Sponsored by Thermo Fisher Scientific
INDUSTRIALIZATION OF CELL THERAPY MANUFACTURING – LESSONS LEARNED FROM BIOLOGICS WORKSHOP
The transformative impact that cell therapies have is evident from the observed efficacy of FDA-approved therapies to date. However, as an increasing number of drugs move from early discovery to commercialization, manufacturing innovations have moved to the forefront of the discussion. We have reached an inflection point that requires us to move from small scale, open workflows to robust, closed and automated manufacturing processes. In this session we will discuss and reflect on the various lessons that we have learned from biologics manufacturing and how they can be applied to advance the industrialization of the cell therapy manufacturing process. Join this session to hear from experts on:
- Innovation in “fit for purpose” closed cell therapy manufacturing equipment
- Controlling critical process parameters with digital integration utilizing a distributed control system (Delta V)
- Supply chain considerations
- Characterization and QC analytics for standardization
Chair:
Betty Woo, Ph.D., Vice President and General Manager, Cell and Gene Therapy, Thermo Fisher Scientific
Speakers:
Jerry Cacia, Chief Technical Officer, Graphite Bio
Andy Lin, Ph.D., Principal Technical Development Leader, Pharma Technical Development, Genentech
Sophia Sharp Donaldson, Executive Director, Supply Chain, Global Strategic Sourcing and Procurement, Kite, a Gilead company
7:15am – 8:45am | Aviara Salon A
Sponsored by IQVIA
DEMOCRATIZATION OF TRANSFORMATIVE CELL & GENE THERAPIES WORKSHOP
Recent years have seen dramatic success with gene therapy, even apparent cures for serious medical conditions (e.g., spinomuscular atrophy, sickle cell disease). Yet, for several of the diseases being cured with gene therapy, the vast majority of patients who could potentially benefit reside in low- and middle-income countries (LMIC), where such therapies are not available. The reasons underlying inaccessibility are multifold, and both near-term and long-term solutions need to be devised and implemented by the regenerative medicine industry. Near and next-term solutions come from disparate areas from utilizing information technology with patient databases/registries, to improving efficiencies and cost of manufacturing and distribution. The challenges of gene therapy implementation in LMIC span several functional areas including education and communication, regulatory, clinical preparedness, and technical / logistical aspects. Looking at the longer-range and worldwide aspirations, one initiative called the Global Gene Therapy Initiative (GGTI) includes diverse stakeholders covering broad areas of expertise from all sectors. The GGTI has set an aspirational goal of launching Phase I gene therapy clinical trials in each of two LMIC, Uganda and India, by 2024.
Chair:
Eugene Brandon, Ph.D., Head, Cell and Gene Therapy Translation, IQVIA
Speakers:
Jennifer Adair, Ph.D., Associate Professor, Clinical Research Division, Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center
Boro Dropulic, Ph.D., Executive Director, Caring Cross
Eric Faulkner, Vice President, Global Real World Evidence, Novartis Gene Therapies
Mike McCullough, Chief Information Officer, National Marrow Donor Program (NMDP)/Be The Match
7:15am – 8:45am | Aviara Salon B
Sponsored by Cell One Partners
OVERCOMING CHALLENGES TO GENE THERAPY MANUFACTURING THROUGH STANDARDIZATION & AUTOMATION WORKSHOP
Gene therapy clinical trials pose interesting challenges in GMP manufacturing. Plasmids and vectors often cannot be produced by the same vendors, may have different stabilities, and may require different storage conditions. Furthermore, some gene therapies require additional processing of the vector with target cells ex vivo. This expert-led session will explore innovations and best practices that are shortening timelines, lowering costs, and standardizing processes when manufacturing gene therapies.
Chair:
Anthony Gringeri, Ph.D., Strategic Advisor, Cell One Partners
Speakers:
David Backer, Chief Commercial Officer, Oxford Biomedica
Michelle Berg, President, GMP Nucleic Acids, Aldevron
Fabian Gerlinghaus, Co-Founder and CEO, Cellares
Judith Koliwer, Ph.D., Lead Consultant Software, Körber Business Area Pharma
9:00am | GENERAL SESSION AND PARTNERING OPENS
OVERVIEW OF ARM’S INITIATIVES
{AVROBIO Ballroom}
9:00am – 9:15am
Speaker:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
9:15am – 10:15am | CONCURRENT TRACKS
THE PAST, PRESENT & FUTURE OF GENE DELIVERY
{AVROBIO Ballroom}
9:15am – 10:15am
Gene therapy holds promise for treating and, possibly, curing a wide range of diseases, including cancer, cystic fibrosis, heart disease, and diabetes. Since the first FDA approved gene therapy trial more than 30 years ago, the advances in gene therapy have rapidly evolved. However, challenges to delivering these life-saving therapies persist, many of which are bespoke to patients. Answers may be provided in technology, automation, and scientific advances.
Chair:
Timothy Schroeder, Chairman and CEO, CTI Clinical Trial & Consulting Services
Speakers:
Patrick Boyle, Ph.D., Head of Codebase, Ginkgo Bioworks
Emma McBurney, Vice President, Business Development, Biogen
Leslie Meltzer, Ph.D., Chief Medical Officer, Orchard Therapeutics
Peter Nell, Ph.D., Chief Business Officer and Head of Therapeutics, Mammoth Biosciences
Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM); Chairman, BlueRock Therapeutics
Devyn Smith, Ph.D., CEO, Arbor Biotechnologies
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:15am – 9:30am | Cook MyoSite |
| 9:30am – 9:45am | BioLife Solutions |
| 9:45am – 10:00am | Terumo BCT |
| 10:00am – 10:15am | Bone Therapeutics |
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by Triangle Insights Group
7:15am – 8:45am | CONCURRENT WORKSHOPS
7:15am – 8:45am | BlueRock Therapeutics Ballroom
Sponsored by Thermo Fisher Scientific
INDUSTRIALIZATION OF CELL THERAPY MANUFACTURING – LESSONS LEARNED FROM BIOLOGICS WORKSHOP
The transformative impact that cell therapies have is evident from the observed efficacy of FDA-approved therapies to date. However, as an increasing number of drugs move from early discovery to commercialization, manufacturing innovations have moved to the forefront of the discussion. We have reached an inflection point that requires us to move from small scale, open workflows to robust, closed and automated manufacturing processes. In this session we will discuss and reflect on the various lessons that we have learned from biologics manufacturing and how they can be applied to advance the industrialization of the cell therapy manufacturing process. Join this session to hear from experts on:
- Innovation in “fit for purpose” closed cell therapy manufacturing equipment
- Controlling critical process parameters with digital integration utilizing a distributed control system (Delta V)
- Supply chain considerations
- Characterization and QC analytics for standardization
Chair:
Betty Woo, Ph.D., Vice President and General Manager, Cell and Gene Therapy, Thermo Fisher Scientific
Speakers:
Jerry Cacia, Chief Technical Officer, Graphite Bio
Andy Lin, Ph.D., Principal Technical Development Leader, Pharma Technical Development, Genentech
Sophia Sharp Donaldson, Executive Director, Supply Chain, Global Strategic Sourcing and Procurement, Kite, a Gilead company
7:15am – 8:45am | Aviara Salon A
Sponsored by IQVIA
DEMOCRATIZATION OF TRANSFORMATIVE CELL & GENE THERAPIES WORKSHOP
Recent years have seen dramatic success with gene therapy, even apparent cures for serious medical conditions (e.g., spinomuscular atrophy, sickle cell disease). Yet, for several of the diseases being cured with gene therapy, the vast majority of patients who could potentially benefit reside in low- and middle-income countries (LMIC), where such therapies are not available. The reasons underlying inaccessibility are multifold, and both near-term and long-term solutions need to be devised and implemented by the regenerative medicine industry. Near and next-term solutions come from disparate areas from utilizing information technology with patient databases/registries, to improving efficiencies and cost of manufacturing and distribution. The challenges of gene therapy implementation in LMIC span several functional areas including education and communication, regulatory, clinical preparedness, and technical / logistical aspects. Looking at the longer-range and worldwide aspirations, one initiative called the Global Gene Therapy Initiative (GGTI) includes diverse stakeholders covering broad areas of expertise from all sectors. The GGTI has set an aspirational goal of launching Phase I gene therapy clinical trials in each of two LMIC, Uganda and India, by 2024.
Chair:
Eugene Brandon, Ph.D., Head, Cell and Gene Therapy Translation, IQVIA
Speakers:
Jennifer Adair, Ph.D., Associate Professor, Clinical Research Division, Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center
Boro Dropulic, Ph.D., Executive Director, Caring Cross
Eric Faulkner, Vice President, Global Real World Evidence, Novartis Gene Therapies
Mike McCullough, Chief Information Officer, National Marrow Donor Program (NMDP)/Be The Match
7:15am – 8:45am | Aviara Salon B
Sponsored by Cell One Partners
OVERCOMING CHALLENGES TO GENE THERAPY MANUFACTURING THROUGH STANDARDIZATION & AUTOMATION WORKSHOP
Gene therapy clinical trials pose interesting challenges in GMP manufacturing. Plasmids and vectors often cannot be produced by the same vendors, may have different stabilities, and may require different storage conditions. Furthermore, some gene therapies require additional processing of the vector with target cells ex vivo. This expert-led session will explore innovations and best practices that are shortening timelines, lowering costs, and standardizing processes when manufacturing gene therapies.
Chair:
Anthony Gringeri, Ph.D., Strategic Advisor, Cell One Partners
Speakers:
David Backer, Chief Commercial Officer, Oxford Biomedica
Michelle Berg, President, GMP Nucleic Acids, Aldevron
Fabian Gerlinghaus, Co-Founder and CEO, Cellares
Judith Koliwer, Ph.D., Lead Consultant Software, Körber Business Area Pharma
9:00am | GENERAL SESSION AND PARTNERING OPENS
OVERVIEW OF ARM’S INITIATIVES
{AVROBIO Ballroom}
9:00am – 9:15am
Speaker:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
9:15am – 10:15am | CONCURRENT TRACKS
THE PAST, PRESENT & FUTURE OF GENE DELIVERY
{AVROBIO Ballroom}
9:15am – 10:15am
Gene therapy holds promise for treating and, possibly, curing a wide range of diseases, including cancer, cystic fibrosis, heart disease, and diabetes. Since the first FDA approved gene therapy trial more than 30 years ago, the advances in gene therapy have rapidly evolved. However, challenges to delivering these life-saving therapies persist, many of which are bespoke to patients. Answers may be provided in technology, automation, and scientific advances.
Chair:
Timothy Schroeder, Chairman and CEO, CTI Clinical Trial & Consulting Services
Speakers:
Patrick Boyle, Ph.D., Head of Codebase, Ginkgo Bioworks
Emma McBurney, Vice President, Business Development, Biogen
Leslie Meltzer, Ph.D., Chief Medical Officer, Orchard Therapeutics
Peter Nell, Ph.D., Chief Business Officer and Head of Therapeutics, Mammoth Biosciences
Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM); Chairman, BlueRock Therapeutics
Devyn Smith, Ph.D., CEO, Arbor Biotechnologies
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:15am – 9:30am | Cook MyoSite |
| 9:30am – 9:45am | BioLife Solutions |
| 9:45am – 10:00am | Terumo BCT |
| 10:00am – 10:15am | Bone Therapeutics |
10:15am – 10:45am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
10:45am – 12:00pm | CONCURRENT TRACKS
INNOVATIVE REGULATORY PATHWAYS FOR NEXT-GENERATION TREATMENTS
{AVROBIO Ballroom}
10:45am – 11:15am
Cell and gene therapies are complex and the current regulations around them are also multifaceted – and constantly evolving. Regulatory agencies need to be involved throughout the development process to keep in lockstep and build their insights into the program. Ultimately, the goal is to create and execute an effective and efficient strategy that leaves nothing to chance. This session will feature discussion between leading developers of innovative therapies sharing insights about their key regulatory considerations and pathways to market.
Chair:
Mark Lane, Ph.D., Vice President, Development Consulting and Scientific Affairs, Biopharma Excellence by PharmaLex
Speakers:
Jennifer Dittman, Vice President, Regulatory Affairs, Generation Bio
Rachelle Jacques, CEO, Enzyvant
Jeff Ross, Ph.D., CEO, Miromatrix
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 10:45am – 11:00am | Akron Biotech |
| 11:00am – 11:15am | MEDIPOST America |
STRATEGIES FOR MANAGING ACCESS & REIMBURSEMENT CHALLENGES
{AVROBIO Ballroom}
11:15am – 12:00pm
Chair:
Katy Spink, Ph.D., Chief Operating Officer and Managing Partner, Dark Horse Consulting Group
Speakers:
Eric Faulkner, Vice President, Global Real World Evidence, Novartis Gene Therapies
Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics
Kristin Wolff, Vice President, Global Policy Strategy and U.S. Government Payer, bluebird bio
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 11:15am – 11:30am | AGTC |
| 11:30am – 11:45am | ConeSight Therapeutics |
| 11:45am – 12:00pm | SparingVision |
12:00pm – 1:15pm | LUNCH
Sponsored by Yposkesi
1:15pm – 2:45pm | CONCURRENT TRACKS
UPDATE ON THE ARM FOUNDATION FOR CELL & GENE MEDICINE
{AVROBIO Ballroom}
1:15pm – 1:25pm
Speakers:
Brett Kopelan, Chairman of the Board, ARM Foundation for Cell & Gene Medicine; Executive Director, debra of America
Martha Rook, Ph.D., Treasurer, ARM Foundation for Cell & Gene Medicine; Chief Technical Operations Officer, Sigilon Therapeutics
COMPANY PRESENTATION
{BlueRock Therapeutics Ballroom}
| 1:15pm – 1:30pm | CTI Clinical Trial & Consulting |
THE INFORMATION PATIENTS WANT ABOUT REGENERATIVE MEDICINE
{AVROBIO Ballroom}
1:25pm – 2:00pm
Chair:
Brett Kopelan, Chairman of the Board, ARM Foundation for Cell & Gene Medicine; Executive Director, debra of America
Speakers:
Leonard Valentino, M.D., President and CEO, National Hemophilia Foundation
Ellen Wagner, Special Projects Coordinator, Parent Project Muscular Dystrophy (PPMD)
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 1:30pm – 1:45pm | ElevateBio |
| 1:45pm – 2:00pm | GentiBio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 2:00pm – 2:15pm | Sangamo Therapeutics |
| 2:15pm – 2:30pm | uniQure |
| 2:30pm – 2:45pm | Sigilon Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 2:00pm – 2:15pm | Virica Biotech |
| 2:15pm – 2:30pm | Enzyvant Therapeutics |
| 2:30pm – 2:45pm | Sentien Biotechnologies |
2:45pm – 3:15pm | AFTERNOON BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
3:15pm – 6:00pm | CONCURRENT TRACKS
PERSONALIZING CARDIOVASCULAR THERAPIES TO ADDRESS THE UNDERLYING CAUSES OF DISEASE
{AVROBIO Ballroom}
3:15pm – 4:15pm
Cell and gene therapies are some of the most cutting-edge innovations in cardiovascular (CV) medicine and represent the next frontier of this therapeutic area. Even though CV cell and gene therapies have great promise to significantly improve clinical outcomes, it is still an emerging field, with a history of clinical trials yielding mixed results. Some of the key challenges in developing these therapies include understanding the true mechanisms of action, identifying the most effective cell types, dosing, CV clinical targets, and routes and frequency of administration. Other important questions about CV cell and gene therapy clinical development focus on how best to design clinical trials, what target populations to focus on, and what clinical development paradigms to use to successfully advance the field. This session includes leaders in the field who have already grappled with many of these questions, and have successfully addressed them to launch CV clinical development programs.
Chair:
Monica Shah, M.D., Vice President and Head, Cell and Gene Therapy Center of Excellence, IQVIA
Speakers:
Faraz Ali, CEO, Tenaya Therapeutics
Joachim Fruebis, Ph.D., Chief Development Officer, BlueRock Therapeutics
Jordan Lancaster, Ph.D., Co-founder and CEO, Avery Therapeutics
William “B.J.” Lehmann, Interim CEO, Athersys
Michael Scott, Ph.D., Vice President, Cell Therapy Medical Devices, Novo Nordisk
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 3:15pm – 3:30pm | Immusoft |
| 3:30pm – 3:45pm | Capricor Therapeutics |
| 3:45pm – 4:00pm | PBS Biotech |
| 4:00pm – 4:15pm | L7 Informatics |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 4:15pm – 4:30pm | Allogene Therapeutics |
| 4:30pm – 4:45pm | Mission Bio |
| 4:45pm – 5:00pm | Synthego |
| 5:00pm – 5:15pm | MaxCyte |
| 5:15pm – 5:30pm | Vor Biopharma |
| 5:30pm – 5:45pm | Poseida Therapeutics |
| 5:45pm – 6:00pm | Kuur Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 4:15pm – 4:30pm | Sensorion |
| 4:30pm – 4:45pm | Likarda |
| 4:45pm – 5:00pm | Tenaya Therapeutics |
| 5:00pm – 5:15pm | Vineti |
| 5:15pm – 5:30pm | Rejuvenate Bio |
| 5:30pm – 5:45pm | Avery Therapeutics |
6:00pm | PROGRAM AND PARTNERING CLOSES
10:15am – 10:45am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
10:45am – 12:00pm | CONCURRENT TRACKS
INNOVATIVE REGULATORY PATHWAYS FOR NEXT-GENERATION TREATMENTS
{AVROBIO Ballroom}
10:45am – 11:15am
Cell and gene therapies are complex and the current regulations around them are also multifaceted – and constantly evolving. Regulatory agencies need to be involved throughout the development process to keep in lockstep and build their insights into the program. Ultimately, the goal is to create and execute an effective and efficient strategy that leaves nothing to chance. This session will feature discussion between leading developers of innovative therapies sharing insights about their key regulatory considerations and pathways to market.
Chair:
Mark Lane, Ph.D., Vice President, Development Consulting and Scientific Affairs, Biopharma Excellence by PharmaLex
Speakers:
Jennifer Dittman, Vice President, Regulatory Affairs, Generation Bio
Rachelle Jacques, CEO, Enzyvant
Jeff Ross, Ph.D., CEO, Miromatrix
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 10:45am – 11:00am | Akron Biotech |
| 11:00am – 11:15am | MEDIPOST America |
STRATEGIES FOR MANAGING ACCESS & REIMBURSEMENT CHALLENGES
{AVROBIO Ballroom}
11:15am – 12:00pm
Chair:
Katy Spink, Ph.D., Chief Operating Officer and Managing Partner, Dark Horse Consulting Group
Speakers:
Eric Faulkner, Vice President, Global Real World Evidence, Novartis Gene Therapies
Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics
Kristin Wolff, Vice President, Global Policy Strategy and U.S. Government Payer, bluebird bio
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 11:15am – 11:30am | AGTC |
| 11:30am – 11:45am | ConeSight Therapeutics |
| 11:45am – 12:00pm | SparingVision |
12:00pm – 1:15pm | LUNCH
Sponsored by Yposkesi
1:15pm – 2:45pm | CONCURRENT TRACKS
UPDATE ON THE ARM FOUNDATION FOR CELL & GENE MEDICINE
{AVROBIO Ballroom}
1:15pm – 1:25pm
Speakers:
Brett Kopelan, Chairman of the Board, ARM Foundation for Cell & Gene Medicine; Executive Director, debra of America
Martha Rook, Ph.D., Treasurer, ARM Foundation for Cell & Gene Medicine; Chief Technical Operations Officer, Sigilon Therapeutics
COMPANY PRESENTATION
{BlueRock Therapeutics Ballroom}
| 1:15pm – 1:30pm | CTI Clinical Trial & Consulting |
THE INFORMATION PATIENTS WANT ABOUT REGENERATIVE MEDICINE
{AVROBIO Ballroom}
1:25pm – 2:00pm
Chair:
Brett Kopelan, Chairman of the Board, ARM Foundation for Cell & Gene Medicine; Executive Director, debra of America
Speakers:
Leonard Valentino, M.D., President and CEO, National Hemophilia Foundation
Ellen Wagner, Special Projects Coordinator, Parent Project Muscular Dystrophy (PPMD)
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 1:30pm – 1:45pm | ElevateBio |
| 1:45pm – 2:00pm | GentiBio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 2:00pm – 2:15pm | Sangamo Therapeutics |
| 2:15pm – 2:30pm | uniQure |
| 2:30pm – 2:45pm | Sigilon Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 2:00pm – 2:15pm | Virica Biotech |
| 2:15pm – 2:30pm | Enzyvant Therapeutics |
| 2:30pm – 2:45pm | Sentien Biotechnologies |
2:45pm – 3:15pm | AFTERNOON BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
3:15pm – 6:00pm | CONCURRENT TRACKS
PERSONALIZING CARDIOVASCULAR THERAPIES TO ADDRESS THE UNDERLYING CAUSES OF DISEASE
{AVROBIO Ballroom}
3:15pm – 4:15pm
Cell and gene therapies are some of the most cutting-edge innovations in cardiovascular (CV) medicine and represent the next frontier of this therapeutic area. Even though CV cell and gene therapies have great promise to significantly improve clinical outcomes, it is still an emerging field, with a history of clinical trials yielding mixed results. Some of the key challenges in developing these therapies include understanding the true mechanisms of action, identifying the most effective cell types, dosing, CV clinical targets, and routes and frequency of administration. Other important questions about CV cell and gene therapy clinical development focus on how best to design clinical trials, what target populations to focus on, and what clinical development paradigms to use to successfully advance the field. This session includes leaders in the field who have already grappled with many of these questions, and have successfully addressed them to launch CV clinical development programs.
Chair:
Monica Shah, M.D., Vice President and Head, Cell and Gene Therapy Center of Excellence, IQVIA
Speakers:
Faraz Ali, CEO, Tenaya Therapeutics
Joachim Fruebis, Ph.D., Chief Development Officer, BlueRock Therapeutics
Jordan Lancaster, Ph.D., Co-founder and CEO, Avery Therapeutics
William “B.J.” Lehmann, Interim CEO, Athersys
Michael Scott, Ph.D., Vice President, Cell Therapy Medical Devices, Novo Nordisk
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 3:15pm – 3:30pm | Immusoft |
| 3:30pm – 3:45pm | Capricor Therapeutics |
| 3:45pm – 4:00pm | PBS Biotech |
| 4:00pm – 4:15pm | L7 Informatics |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 4:15pm – 4:30pm | Allogene Therapeutics |
| 4:30pm – 4:45pm | Mission Bio |
| 4:45pm – 5:00pm | Synthego |
| 5:00pm – 5:15pm | MaxCyte |
| 5:15pm – 5:30pm | Vor Biopharma |
| 5:30pm – 5:45pm | Poseida Therapeutics |
| 5:45pm – 6:00pm | Kuur Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 4:15pm – 4:30pm | Sensorion |
| 4:30pm – 4:45pm | Likarda |
| 4:45pm – 5:00pm | Tenaya Therapeutics |
| 5:00pm – 5:15pm | Vineti |
| 5:15pm – 5:30pm | Rejuvenate Bio |
| 5:30pm – 5:45pm | Avery Therapeutics |
6:00pm | PROGRAM AND PARTNERING CLOSES
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by CPC
7:15am – 8:45am | MORNING WORKSHOP
7:15am – 8:45am | Aviara Salon B
Sponsored by CSL Behring
DELIVERING THE INNOVATION OF GENE THERAPY TO PATIENTS THROUGH STRATEGIC COLLABORATIONS WORKSHOP
This workshop will explore case studies where strategic collaborations have been the pathway of choice for gene therapy innovators to ensure an optimal route to commercialization. Leading executives in the gene therapy sector will discuss the rationale of these strategic partnerships along with key learnings for successful implementation.
Chair:
Debbie Drane, Senior Vice President, Global Commercial Development and TA Strategy, CSL Behring
Speakers:
Lawrence Kau, Global Commercial Strategy Lead, Spark Therapeutics
Josh Leeman, Ph.D., Vice President, Business Development, Search and Evaluation, uniQure
Adam Simpson, Ph.D., Head, Gene Therapies BD&L, Novartis Institutes for BioMedical Research
9:00am | GENERAL SESSION AND PARTNERING OPENS
ARM GROW INTERNSHIP UPDATE {AVROBIO Ballroom}
9:00am – 9:15am
Speakers:
Ifeoluwa Awoleye, ARM GROW Intern, Prevail Therapeutics
Rashida Dujue-Jackson, AFE Program Director, Alliance for Regenerative Medicine (ARM)
Rachelle Jacques, CEO, Enzyvant
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Jamila Ritter, ARM GROW Intern, Amicus Therapeutics
9:15am – 10:45am | CONCURRENT TRACKS
SYNTHETIC BIOLOGY APPLICATIONS IN CELL & GENE THERAPY
{AVROBIO Ballroom}
9:15am – 9:45am
Chair:
Robert Deans, Ph.D., Chief Scientific Officer, Synthego
Speakers:
Timothy Lu, M.D., Ph.D., Co-founder and CEO, Senti Biosciences
Ali Nahvi, Ph.D., Head of New Therapeutic Modalities, Spark Therapeutics
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:15am – 9:30am | Mammoth Biosciences |
| 9:30am – 9:45am | Stemson Therapeutics |
WHAT CELL & GENE THERAPY INVESTORS ARE LOOKING FOR NOW
{AVROBIO Ballroom}
9:45am – 10:45am
Chair:
Phil Vanek, Ph.D., Chief Technology Officer, Gamma Biosciences
Speakers:
Gbola Amusa, M.D., Partner and Chief Scientific Officer, Chardan
Chris Garabedian, Chairman and CEO, Xontogeny
Ed Hurwitz, Managing Director, MPM Capital
Deborah Palestrant, Ph.D., Partner and Head of 4:59, 5AM Ventures
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:45am – 10:00am | Endsulin |
| 10:00am – 10:15am | Arbor Biotechnologies |
| 10:15am – 10:30am | ViaCyte |
| 10:30am – 10:45am | Castle Creek Biosciences |
10:45am – 11:00am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
11:00am – 12:00pm | CONCURRENT TRACKS
DOSING & REDOSING IN GENE THERAPY
{AVROBIO Ballroom}
11:00am – 12:00pm
Since the earliest days of gene therapy, dosing has been a key pivotal issue to balance with tremendous benefit of this technology with its inherent risks. Today, understanding considerations of dosing across diverse patient populations for which we intend treatment is much improved, but still requires empiric trials. With a considerable number of key questions still to be answered, our goal should be to collectively and comprehensively explore issues in dosing, and ultimately, re-dosing of patients. Are we doing enough as an industry to confidently employ dosing strategies for gene therapies?
Chair:
Chris Mason, M.D., Ph.D., Chief Scientific Officer, AVROBIO
Speakers:
Eric Crombez, M.D., Chief Medical Officer, Ultragenyx Gene Therapy
Peter Francis, M.D., Ph.D., Chief Scientific Officer, Ophthalmology Therapeutic Area Head, 4D Molecular Therapeutics
Timothy Miller, Ph.D., CEO, President and Co-Founder, Forge Biologics
Nicole Paulk, Ph.D., Professor, AAV Gene Therapy, University of California, San Francisco
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 11:00am – 11:15am | Aspect Biosystems |
| 11:15am – 11:30am | Trailhead Biosystems |
12:00pm – 1:30pm | LUNCH
Sponsored by VGXI
1:30pm | CONFERENCE CLOSES
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by CPC
7:15am – 8:45am | MORNING WORKSHOP
7:15am – 8:45am | Aviara Salon B
Sponsored by CSL Behring
DELIVERING THE INNOVATION OF GENE THERAPY TO PATIENTS THROUGH STRATEGIC COLLABORATIONS WORKSHOP
This workshop will explore case studies where strategic collaborations have been the pathway of choice for gene therapy innovators to ensure an optimal route to commercialization. Leading executives in the gene therapy sector will discuss the rationale of these strategic partnerships along with key learnings for successful implementation.
Chair:
Debbie Drane, Senior Vice President, Global Commercial Development and TA Strategy, CSL Behring
Speakers:
Lawrence Kau, Global Commercial Strategy Lead, Spark Therapeutics
Josh Leeman, Ph.D., Vice President, Business Development, Search and Evaluation, uniQure
Adam Simpson, Ph.D., Head, Gene Therapies BD&L, Novartis Institutes for BioMedical Research
9:00am | GENERAL SESSION AND PARTNERING OPENS
ARM GROW INTERNSHIP UPDATE {AVROBIO Ballroom}
9:00am – 9:15am
Speakers:
Ifeoluwa Awoleye, ARM GROW Intern, Prevail Therapeutics
Rashida Dujue-Jackson, AFE Program Director, Alliance for Regenerative Medicine (ARM)
Rachelle Jacques, CEO, Enzyvant
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Jamila Ritter, ARM GROW Intern, Amicus Therapeutics
9:15am – 10:45am | CONCURRENT TRACKS
SYNTHETIC BIOLOGY APPLICATIONS IN CELL & GENE THERAPY
{AVROBIO Ballroom}
9:15am – 9:45am
Chair:
Robert Deans, Ph.D., Chief Scientific Officer, Synthego
Speakers:
Timothy Lu, M.D., Ph.D., Co-founder and CEO, Senti Biosciences
Ali Nahvi, Ph.D., Head of New Therapeutic Modalities, Spark Therapeutics
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:15am – 9:30am | Mammoth Biosciences |
| 9:30am – 9:45am | Stemson Therapeutics |
WHAT CELL & GENE THERAPY INVESTORS ARE LOOKING FOR NOW
{AVROBIO Ballroom}
9:45am – 10:45am
Chair:
Phil Vanek, Ph.D., Chief Technology Officer, Gamma Biosciences
Speakers:
Gbola Amusa, M.D., Partner and Chief Scientific Officer, Chardan
Chris Garabedian, Chairman and CEO, Xontogeny
Ed Hurwitz, Managing Director, MPM Capital
Deborah Palestrant, Ph.D., Partner and Head of 4:59, 5AM Ventures
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:45am – 10:00am | Endsulin |
| 10:00am – 10:15am | Arbor Biotechnologies |
| 10:15am – 10:30am | ViaCyte |
| 10:30am – 10:45am | Castle Creek Biosciences |
10:45am – 11:00am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
11:00am – 12:00pm | CONCURRENT TRACKS
DOSING & REDOSING IN GENE THERAPY
{AVROBIO Ballroom}
11:00am – 12:00pm
Since the earliest days of gene therapy, dosing has been a key pivotal issue to balance with tremendous benefit of this technology with its inherent risks. Today, understanding considerations of dosing across diverse patient populations for which we intend treatment is much improved, but still requires empiric trials. With a considerable number of key questions still to be answered, our goal should be to collectively and comprehensively explore issues in dosing, and ultimately, re-dosing of patients. Are we doing enough as an industry to confidently employ dosing strategies for gene therapies?
Chair:
Chris Mason, M.D., Ph.D., Chief Scientific Officer, AVROBIO
Speakers:
Eric Crombez, M.D., Chief Medical Officer, Ultragenyx Gene Therapy
Peter Francis, M.D., Ph.D., Chief Scientific Officer, Ophthalmology Therapeutic Area Head, 4D Molecular Therapeutics
Timothy Miller, Ph.D., CEO, President and Co-Founder, Forge Biologics
Nicole Paulk, Ph.D., Professor, AAV Gene Therapy, University of California, San Francisco
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 11:00am – 11:15am | Aspect Biosystems |
| 11:15am – 11:30am | Trailhead Biosystems |
12:00pm – 1:30pm | LUNCH
Sponsored by VGXI
1:30pm | CONFERENCE CLOSES
ON-DEMAND VIRTUAL CONTENT
Available Beginning Tuesday, October 12, 2021
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)
DOING BUSINESS IN JAPAN – AN EVOLVING MARKET WORKSHOP
Promoting Harmonization and Commercialization of Regenerative Medicines Among Asian Countries/Regions
The Asia Partnership Conference of Regenerative Medicine (APACRM), is aimed at harmonizing and optimizing regulations among Asian countries and regions. FIRM has organized the APACRM since 2018 and has closely collaborated with industrial groups such as the China Medicinal Biotech Association (CMBA) in China; Association of Biotechnology Led Enterprises (ABLE) in India; Council for Advanced Regenerative Medicine (CARM) and Strategic Center for Regenerative Medicine (SCRM) in Korea; Singapore Association of Pharmaceuticals Industries (SAPI) in Singapore; and Biotechnology and Pharmaceutical Industries Promotion Office (BPIPO) in Taiwan. This session will focus on prospects and challenges for or against regenerative medicine to promote harmonization and optimization of the regulations on regenerative medicine products among Asian countries and regions.
Chair:
Masayuki (Max) Nomura, Ph.D., Chair, International Affairs Committee, Forum for Innovative Regenerative Medicine (FIRM)
Opening Remarks:
Kozo Saiki, Director, Bio-Industry Division, Ministry of Economy, Trade and Industry (METI)
Speakers:
Tomohiro Yoneda, New Modality and External Innovation Lead, PCS and Pharmacology, Translational Medicine, Novartis Pharma K.K.
Hiroshi Karasawa, Ph.D., Scientist, Cell Therapy Research Laboratories, Daiichi Sankyo Co.
Sponsored by Charles River Laboratories
FROM CONCEPT TO COMMERCIALIZATION IN LIVING THERAPEUTICS: HINDSIGHT IS 20/20 WORKSHOP
Challenges for developing a cell therapy product evolve and shift, and reducing these risks requires experience and expertise to execute around them. From discovery through late-phase clinical trials and into commercial manufacturing, different challenges and competing priorities are ever present – from coordinating multiple CROs/CDMOs, managing clinical operations effectively, and overcoming supply chain and logistic hurdles, all while keeping in line with regulatory standards. This discussion will take a look at these and many more real-world challenges and focus on the strategies involved with mitigating risks along the way while reducing the amount of lag time involved with bringing a cell therapy product to market.
Chair:
Matthew Hewitt, Ph.D., Senior Director, Scientific Solutions Cell and Gene Therapy, Charles River Laboratories
Speakers:
Douglas Doerfler, CEO, MaxCyte
Howard Federoff, M.D., Ph.D., President and CEO, Brooklyn ImmunoTherapeutics; Senior Advisor, Aspen Neuroscience
Kristin Yarema, Ph.D., Chief Commercial Officer, Atara Biotherapeutics
Sponsored by Pfizer
BESPOKE THERAPIES FOR ULTRA-RARE DISEASE
This session will explore the recently formed National Institutes of Health (NIH)’s, Foundation for the NIH, FDA’s and the biopharmaceutical industry’s innovative public-private partnership to help individualized gene therapies reach patients despite their lack of commercial viability. Panelists will provide an overview of this initiative including how it will maximize benefits to patients, as well as explore the specific opportunities and challenges related to its success.
Chair:
Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer Inc.
Speakers:
Philip John (P.J.) Brooks, Ph.D., Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)
Gregory LaRosa, Ph.D., Vice President, Head of Scientific Research, Rare Disease Research Unit, Pfizer Inc.
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
Courtney Silverthorn, Ph.D., Associate Vice President, Research Partnerships, Foundation for the National Institutes of Health (FNIH)
PRE-RECORDED VIRTUAL COMPANY PRESENTATIONS
Pre-recorded virtual company presentations will be available on-demand within the platform for all delegates to view starting October 12, 2021. Accelerated Biosciences | Achilles Therapeutics | Adaptimmune | Advarra | AGC Biologics | AlloVir | American Gene Technologies | Amniotics | Andelyn Biosciences | ARMI | Aruvant | AskBio | Atara Biotherapeutics | Autolus | Avectas | Biogen | BrainStorm Cell Therapeutics | Cabaletta Bio | Caribou Biosciences | Carisma Therapeutics | Cellares | Cellino | CellPort Software | Cellusion | CEVEC | cGMPnow | Corning | CRC Oncology | Cryoport Systems | Cynata Therapeutics | Decibel Therapeutics | FluidForm | FUJIFILM Cellular Dynamics | G-CON Manufacturing | Gamida Cell | Genethon | Genocea Biosciences | GenSight Biologics | GPB Scientific | Healios | HemaCare | I Peace | InsightRX | Invetech | Iovance Biotherapeutics | Kytopen | Notch Therapeutics | Opsis Therapeutics | Polyplus-transfection | Precigen | Precision BioSciences | PTC Therapeutics | Regenerative Patch Technologies | Regeneus | ReNeuron | RoosterBio | SanBio | Santen Pharmaceutical | SCM Lifescience | Sernova | Sio Gene Therapies | SmartLabs | SwanBio Therapeutics | Talaris Therapeutics | Teknova | Univercells Technologies | VectorY Therapeutics | Vigene Biosciences | VisiCELL Medical | VIVEbiotechBIDEN ADMINISTRATION PLANS FOR LIFE SCIENCE INNOVATION
Chair:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Speaker:
Carrie Wolinetz, Ph.D., Deputy Director for Health and Life Sciences, White House Office of Science and Technology Policy
FEATURED FIRESIDE CHAT: FDA PERSPECTIVE
Chair:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Speaker:
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
ON-DEMAND VIRTUAL CONTENT
Available Beginning Tuesday, October 12, 2021
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)
DOING BUSINESS IN JAPAN – AN EVOLVING MARKET WORKSHOP
Promoting Harmonization and Commercialization of Regenerative Medicines Among Asian Countries/Regions
The Asia Partnership Conference of Regenerative Medicine (APACRM), is aimed at harmonizing and optimizing regulations among Asian countries and regions. FIRM has organized the APACRM since 2018 and has closely collaborated with industrial groups such as the China Medicinal Biotech Association (CMBA) in China; Association of Biotechnology Led Enterprises (ABLE) in India; Council for Advanced Regenerative Medicine (CARM) and Strategic Center for Regenerative Medicine (SCRM) in Korea; Singapore Association of Pharmaceuticals Industries (SAPI) in Singapore; and Biotechnology and Pharmaceutical Industries Promotion Office (BPIPO) in Taiwan. This session will focus on prospects and challenges for or against regenerative medicine to promote harmonization and optimization of the regulations on regenerative medicine products among Asian countries and regions.
Chair:
Masayuki (Max) Nomura, Ph.D., Chair, International Affairs Committee, Forum for Innovative Regenerative Medicine (FIRM)
Opening Remarks:
Kozo Saiki, Director, Bio-Industry Division, Ministry of Economy, Trade and Industry (METI)
Speakers:
Tomohiro Yoneda, New Modality and External Innovation Lead, PCS and Pharmacology, Translational Medicine, Novartis Pharma K.K.
Hiroshi Karasawa, Ph.D., Scientist, Cell Therapy Research Laboratories, Daiichi Sankyo Co.
Sponsored by Charles River Laboratories
FROM CONCEPT TO COMMERCIALIZATION IN LIVING THERAPEUTICS: HINDSIGHT IS 20/20 WORKSHOP
Challenges for developing a cell therapy product evolve and shift, and reducing these risks requires experience and expertise to execute around them. From discovery through late-phase clinical trials and into commercial manufacturing, different challenges and competing priorities are ever present – from coordinating multiple CROs/CDMOs, managing clinical operations effectively, and overcoming supply chain and logistic hurdles, all while keeping in line with regulatory standards. This discussion will take a look at these and many more real-world challenges and focus on the strategies involved with mitigating risks along the way while reducing the amount of lag time involved with bringing a cell therapy product to market.
Chair:
Matthew Hewitt, Ph.D., Senior Director, Scientific Solutions Cell and Gene Therapy, Charles River Laboratories
Speakers:
Douglas Doerfler, CEO, MaxCyte
Howard Federoff, M.D., Ph.D., President and CEO, Brooklyn ImmunoTherapeutics; Senior Advisor, Aspen Neuroscience
Kristin Yarema, Ph.D., Chief Commercial Officer, Atara Biotherapeutics
Sponsored by Pfizer
BESPOKE THERAPIES FOR ULTRA-RARE DISEASE
This session will explore the recently formed National Institutes of Health (NIH)’s, Foundation for the NIH, FDA’s and the biopharmaceutical industry’s innovative public-private partnership to help individualized gene therapies reach patients despite their lack of commercial viability. Panelists will provide an overview of this initiative including how it will maximize benefits to patients, as well as explore the specific opportunities and challenges related to its success.
Chair:
Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer Inc.
Speakers:
Philip John (P.J.) Brooks, Ph.D., Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)
Gregory LaRosa, Ph.D., Vice President, Head of Scientific Research, Rare Disease Research Unit, Pfizer Inc.
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
Courtney Silverthorn, Ph.D., Associate Vice President, Research Partnerships, Foundation for the National Institutes of Health (FNIH)
PRE-RECORDED VIRTUAL COMPANY PRESENTATIONS
Pre-recorded virtual company presentations will be available on-demand within the platform for all delegates to view starting October 12, 2021.
Accelerated Biosciences | Achilles Therapeutics | Adaptimmune | Advarra | AGC Biologics | AlloVir | American Gene Technologies | Amniotics | Andelyn Biosciences | ARMI | Aruvant | AskBio | Atara Biotherapeutics | Autolus | Avectas | Biogen | BrainStorm Cell Therapeutics | Cabaletta Bio | Caribou Biosciences | Carisma Therapeutics | Cellares | Cellino | CellPort Software | Cellusion | CEVEC | cGMPnow | Corning | CRC Oncology | Cryoport Systems | Cynata Therapeutics | Decibel Therapeutics | FluidForm | FUJIFILM Cellular Dynamics | G-CON Manufacturing | Gamida Cell | Genethon | Genocea Biosciences | GenSight Biologics | GPB Scientific | Healios | HemaCare | I Peace | InsightRX | Invetech | Iovance Biotherapeutics | Kytopen | Notch Therapeutics | Opsis Therapeutics | Polyplus-transfection | Precigen | Precision BioSciences | PTC Therapeutics | Regenerative Patch Technologies | Regeneus | ReNeuron | RoosterBio | SanBio | Santen Pharmaceutical | SCM Lifescience | Sernova | Sio Gene Therapies | SmartLabs | SwanBio Therapeutics | Talaris Therapeutics | Teknova | Univercells Technologies | VectorY Therapeutics | Vigene Biosciences | VisiCELL Medical | VIVEbiotech
BIDEN ADMINISTRATION PLANS FOR LIFE SCIENCE INNOVATION
Chair:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Speaker:
Carrie Wolinetz, Ph.D., Deputy Director for Health and Life Sciences, White House Office of Science and Technology Policy
FEATURED FIRESIDE CHAT: FDA PERSPECTIVE
Chair:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Speaker:
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by KBI Biopharma
7:15am – 8:45am | CONCURRENT WORKSHOPS
7:15am – 8:45am | BlueRock Therapeutics Ballroom
Sponsored by Precision ADVANCE
WHAT’S NEXT FOR ADVANCED THERAPIES WORKSHOP
This workshop will feature leading executives in the ATMP space who will discuss a number of topics including recent advancements in clinical trials, going from treating rare diseases to prevalent conditions, and implementing next-generation gene-editing technologies. The session will also touch upon resolving manufacturing and talent bottlenecks as the sector moves towards commercialization, as well as the evolution of reimbursement payment models.
Chair:
Anshul Mangal, President, Project Farma and Precision ADVANCE
Speakers:
Phil Cyr, Senior Vice President, Customer Solutions, Precision Value & Health
Palani Palaniappan, Ph.D., Executive Vice President and Chief Technology Officer, Aruvant Sciences
Derrell Porter, M.D., Founder and CEO, Cellevolve
RA Session II, Founder, President and CEO, Taysha Gene Therapies
Claudia Zylberberg, Ph.D., Founder and Executive Chair, Akron Biotech
7:15am – 8:45am | Aviara Salon A
Sponsored by Pointellis, powered by EY
UNLOCKING THE POWER OF COLLABORATION TO FUEL THE INDUSTRY TRANSFORMATION FOR CELL & GENE THERAPIES WORKSHOP
The groundwork for seamless cell and gene therapy (CGT) care delivery needs to be laid now. There are many stakeholders involved in the complexities of the CGT treatment journey that require a radically new approach from that of traditional mass-produced therapies. A complex balancing act of multi-stakeholder processes hinge on treatment advances, product availability, and the network’s ability to meet demand. And that’s before thinking about scaling CGT delivery to more than a few thousand patients. This workshop will bring together leading experts in managing interconnected supply and logistics networks, delivering superior patient experiences, manufacturing cell and gene therapies, and building world-class digital infrastructures.
Chair:
Adlai Goldberg, Global Digital, Social and Commercial Innovation Life Sciences Leader, EY
Speakers:
Sybil Danby, Senior Vice President, Business Development and Strategy, Center for Breakthrough Medicines
Tamie Joeckel, Global Business Lead, Cell and Gene Therapy COE, ICON
Orlando Serani, Program Lead Advanced Cellular Therapies Supply Chain, Johnson & Johnson
Julia Tarasenko, Vice President, Commercial Operations, Europe and the Americas, Marken, a UPS Healthcare company
9:00am | GENERAL SESSION AND PARTNERING OPENS
WELCOME REMARKS
{AVROBIO Ballroom}
9:00am – 9:15am
Speakers:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM); Chairman, BlueRock Therapeutics
PLENARY SESSION: CELLULAR IMMUNOTHERAPY PIONEERS – LIQUID VS. SOLID TUMOR STRATEGIES
{AVROBIO Ballroom}
9:15am – 10:15am
Chair:
Usman “Oz” Azam, M.D., President and CEO, Tmunity Therapeutics
Speakers:
Kanya Rajangam, M.D., Ph.D., Chief Medical Officer, Nkarta Therapeutics
Devon Shedlock, Ph.D., Senior Vice President, Research and Development, Poseida Therapeutics
Pascal Touchon, President and CEO, Atara Biotherapeutics
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by KBI Biopharma
7:15am – 8:45am | CONCURRENT WORKSHOPS
7:15am – 8:45am | BlueRock Therapeutics Ballroom
Sponsored by Precision ADVANCE
WHAT’S NEXT FOR ADVANCED THERAPIES WORKSHOP
This workshop will feature leading executives in the ATMP space who will discuss a number of topics including recent advancements in clinical trials, going from treating rare diseases to prevalent conditions, and implementing next-generation gene-editing technologies. The session will also touch upon resolving manufacturing and talent bottlenecks as the sector moves towards commercialization, as well as the evolution of reimbursement payment models.
Chair:
Anshul Mangal, President, Project Farma and Precision ADVANCE
Speakers:
Phil Cyr, Senior Vice President, Customer Solutions, Precision Value & Health
Palani Palaniappan, Ph.D., Executive Vice President and Chief Technology Officer, Aruvant Sciences
Derrell Porter, M.D., Founder and CEO, Cellevolve
RA Session II, Founder, President and CEO, Taysha Gene Therapies
Claudia Zylberberg, Ph.D., Founder and Executive Chair, Akron Biotech
7:15am – 8:45am | Aviara Salon A
Sponsored by Pointellis, powered by EY
UNLOCKING THE POWER OF COLLABORATION TO FUEL THE INDUSTRY TRANSFORMATION FOR CELL & GENE THERAPIES WORKSHOP
The groundwork for seamless cell and gene therapy (CGT) care delivery needs to be laid now. There are many stakeholders involved in the complexities of the CGT treatment journey that require a radically new approach from that of traditional mass-produced therapies. A complex balancing act of multi-stakeholder processes hinge on treatment advances, product availability, and the network’s ability to meet demand. And that’s before thinking about scaling CGT delivery to more than a few thousand patients. This workshop will bring together leading experts in managing interconnected supply and logistics networks, delivering superior patient experiences, manufacturing cell and gene therapies, and building world-class digital infrastructures.
Chair:
Adlai Goldberg, Global Digital, Social and Commercial Innovation Life Sciences Leader, EY
Speakers:
Sybil Danby, Senior Vice President, Business Development and Strategy, Center for Breakthrough Medicines
Tamie Joeckel, Global Business Lead, Cell and Gene Therapy COE, ICON
Orlando Serani, Program Lead Advanced Cellular Therapies Supply Chain, Johnson & Johnson
Julia Tarasenko, Vice President, Commercial Operations, Europe and the Americas, Marken, a UPS Healthcare company
9:00am | GENERAL SESSION AND PARTNERING OPENS
WELCOME REMARKS
{AVROBIO Ballroom}
9:00am – 9:15am
Speakers:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM); Chairman, BlueRock Therapeutics
PLENARY SESSION: CELLULAR IMMUNOTHERAPY PIONEERS – LIQUID VS. SOLID TUMOR STRATEGIES
{AVROBIO Ballroom}
9:15am – 10:15am
Chair:
Usman “Oz” Azam, M.D., President and CEO, Tmunity Therapeutics
Speakers:
Kanya Rajangam, M.D., Ph.D., Chief Medical Officer, Nkarta Therapeutics
Devon Shedlock, Ph.D., Senior Vice President, Research and Development, Poseida Therapeutics
Pascal Touchon, President and CEO, Atara Biotherapeutics
10:15am – 10:45am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
10:45am – 12:00pm | CONCURRENT TRACKS
LESSONS LEARNED: THE ROLE OF PATIENT ENGAGEMENT IN APPROVALS & PAYOR DISCUSSIONS
{AVROBIO Ballroom}
10:45am – 11:15am
In a pre-recorded video, a mother of a child living with SMA who received Zolgensma will share her story and what she believes healthcare decision makers should include in their evaluations of gene therapies. ARM’s Paige Bischoff will then lead a live discussion with Novartis’ Amy Nicole Nayar on lessons emerging gene therapy companies can learn from Novartis’ experience incorporating the patient perspective in discussions with regulators, payors, and health technology assessors.
Chair:
Paige Bischoff, Senior Vice President of Global Public Affairs, Alliance for Regenerative Medicine (ARM)
Speakers:
Katee DellaMaggiora, Mother of a child living with SMA and Cure SMA advocate
Amy Nicole Nayar, Pharm.D., Vice President of U.S. Patient Advocacy and Government Affairs, Novartis Gene Therapies
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 10:45am – 11:00am | Emendo Biotherapeutics |
| 11:00am – 11:15am | Ensoma |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 11:15am – 11:30am | Oxford Biomedica |
| 11:30am – 11:45am | 4DMT |
| 11:45am – 12:00pm | Helixmith |
12:00pm – 1:15pm | LUNCH
Sponsored by Pall Corporation
1:15pm – 3:30pm | CONCURRENT TRACKS
WHERE ARE WE ON THE JOURNEY TO CURE SICKLE CELL DISEASE?
{AVROBIO Ballroom}
1:15pm – 2:15pm
The sickle cell mutation was discovered in 1956. Unfortunately, the translation of knowledge into developing treatments has been disproportionately slow and elusive…until now. In the last five years, there has been a vast increase in the number of therapies in development and the prominence of sickle cell has grown on the agendas of professional societies and the public sector. How is this combination of attention and technology bringing us closer to a cure? Hear from leading experts on the latest cell and gene therapies aiming to address this devastating disease.
Chair:
Jill Elliott, Vice President, Cell Therapy Commercialization, Novo Nordisk
Speakers:
Edmond Chen, M.D., Vice President, Head of Clinical Development, Hematology, Editas Medicine
Debbie Drane, Senior Vice President, Global Commercial Development and TA Strategy, CSL Behring
Thomas Klima, Chief Commercial Officer, bluebird bio
Sandy Macrae, Ph.D., President and CEO, Sangamo Therapeutics
Amy Simon, M.D., Chief Medical Officer, Beam Therapeutics
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 1:15pm – 1:30pm | AffyImmune Therapeutics |
| 1:30pm – 1:45pm | Angiocrine Bioscience |
| 1:45pm – 2:00pm | Artisan Bio |
| 2:00pm – 2:15pm | Catamaran Bio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 2:15pm – 2:30pm | BlueRock Therapeutics |
| 2:30pm – 2:45pm | Taysha Gene Therapies |
| 2:45pm – 3:00pm | Passage Bio |
| 3:00pm – 3:15pm | Athersys |
| 3:15pm – 3:30pm | Ginkgo Bioworks |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 2:15pm – 2:30pm | Rocket Pharmaceuticals |
| 2:30pm – 2:45pm | Luminary Therapeutics |
| 2:45pm – 3:00pm | Senti Biosciences |
| 3:00pm – 3:15pm | Shoreline Biosciences |
| 3:15pm – 3:30pm | DiscGenics |
3:30pm – 4:00pm | AFTERNOON BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
4:00pm – 6:00pm | CONCURRENT TRACKS
CURRENT REALITIES OF MAKE VS. BUY FOR CELL & GENE THERAPY MANUFACTURING
{AVROBIO Ballroom}
4:00pm – 5:00pm
This panel of leading experts will discuss key considerations and experiences related to the Make vs. Buy decision from a variety of perspectives. Speakers will debate whether those considerations are unique to today’s environment and how they might be different in the future. This session will solicit significant audience input to shape the discussion.
Chair:
Kelvin Lee, Ph.D., Institute Director, National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)
Speakers:
Melissa Carpenter, Ph.D., Chief Scientific Officer, Regenerative Medicine, ElevateBio
Thomas Fellner, Ph.D., Vice President, Global Head of Business Development, Account Management, and Program Management, Lonza Cell & Gene Technologies
Nirupama (Rupa) Pike, Ph.D., Director, Enterprise Science and Innovation Partnerships, Global Corporate Accounts, Thermo Fisher Scientific
Joe Tarnowski, Ph.D., Senior Vice President, Cell and Gene Therapy Platform, GlaxoSmithKline
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 4:00pm – 4:15pm | Cellatoz Therapeutics |
| 4:15pm – 4:30pm | Aspen Neuroscience |
| 4:30pm – 4:45pm | Labcorp |
| 4:45pm – 5:00pm | Mirus Bio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 5:00pm – 5:15pm | Amicus Therapeutics |
| 5:15pm – 5:30pm | Homology Medicines |
| 5:30pm – 5:45pm | AVROBIO |
| 5:45pm – 6:00pm | Orchard Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 5:00pm – 5:15pm | Forge Biologics |
| 5:15pm – 5:30pm | Life Edit Therapeutics |
| 5:30pm – 5:45pm | TreeFrog Therapeutics |
| 5:45pm – 6:00pm | GenEdit |
6:00pm | PROGRAM AND PARTNERING CLOSES
6:30pm – 9:30pm | NETWORKING BASH
Sponsored by Biopharma Excellence by PharmaLex, Cognate BioServices and L7 Informatics
10:15am – 10:45am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
10:45am – 12:00pm | CONCURRENT TRACKS
LESSONS LEARNED: THE ROLE OF PATIENT ENGAGEMENT IN APPROVALS & PAYOR DISCUSSIONS
{AVROBIO Ballroom}
10:45am – 11:15am
In a pre-recorded video, a mother of a child living with SMA who received Zolgensma will share her story and what she believes healthcare decision makers should include in their evaluations of gene therapies. ARM’s Paige Bischoff will then lead a live discussion with Novartis’ Amy Nicole Nayar on lessons emerging gene therapy companies can learn from Novartis’ experience incorporating the patient perspective in discussions with regulators, payors, and health technology assessors.
Chair:
Paige Bischoff, Senior Vice President of Global Public Affairs, Alliance for Regenerative Medicine (ARM)
Speakers:
Katee DellaMaggiora, Mother of a child living with SMA and Cure SMA advocate
Amy Nicole Nayar, Pharm.D., Vice President of U.S. Patient Advocacy and Government Affairs, Novartis Gene Therapies
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 10:45am – 11:00am | Emendo Biotherapeutics |
| 11:00am – 11:15am | Ensoma |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 11:15am – 11:30am | Oxford Biomedica |
| 11:30am – 11:45am | 4DMT |
| 11:45am – 12:00pm | Helixmith |
12:00pm – 1:15pm | LUNCH
Sponsored by Pall Corporation
1:15pm – 3:30pm | CONCURRENT TRACKS
WHERE ARE WE ON THE JOURNEY TO CURE SICKLE CELL DISEASE?
{AVROBIO Ballroom}
1:15pm – 2:15pm
The sickle cell mutation was discovered in 1956. Unfortunately, the translation of knowledge into developing treatments has been disproportionately slow and elusive…until now. In the last five years, there has been a vast increase in the number of therapies in development and the prominence of sickle cell has grown on the agendas of professional societies and the public sector. How is this combination of attention and technology bringing us closer to a cure? Hear from leading experts on the latest cell and gene therapies aiming to address this devastating disease.
Chair:
Jill Elliott, Vice President, Cell Therapy Commercialization, Novo Nordisk
Speakers:
Edmond Chen, M.D., Vice President, Head of Clinical Development, Hematology, Editas Medicine
Debbie Drane, Senior Vice President, Global Commercial Development and TA Strategy, CSL Behring
Thomas Klima, Chief Commercial Officer, bluebird bio
Sandy Macrae, Ph.D., President and CEO, Sangamo Therapeutics
Amy Simon, M.D., Chief Medical Officer, Beam Therapeutics
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 1:15pm – 1:30pm | AffyImmune Therapeutics |
| 1:30pm – 1:45pm | Angiocrine Bioscience |
| 1:45pm – 2:00pm | Artisan Bio |
| 2:00pm – 2:15pm | Catamaran Bio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 2:15pm – 2:30pm | BlueRock Therapeutics |
| 2:30pm – 2:45pm | Taysha Gene Therapies |
| 2:45pm – 3:00pm | Passage Bio |
| 3:00pm – 3:15pm | Athersys |
| 3:15pm – 3:30pm | Ginkgo Bioworks |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 2:15pm – 2:30pm | Rocket Pharmaceuticals |
| 2:30pm – 2:45pm | Luminary Therapeutics |
| 2:45pm – 3:00pm | Senti Biosciences |
| 3:00pm – 3:15pm | Shoreline Biosciences |
| 3:15pm – 3:30pm | DiscGenics |
3:30pm – 4:00pm | AFTERNOON BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
4:00pm – 6:00pm | CONCURRENT TRACKS
CURRENT REALITIES OF MAKE VS. BUY FOR CELL & GENE THERAPY MANUFACTURING
{AVROBIO Ballroom}
4:00pm – 5:00pm
This panel of leading experts will discuss key considerations and experiences related to the Make vs. Buy decision from a variety of perspectives. Speakers will debate whether those considerations are unique to today’s environment and how they might be different in the future. This session will solicit significant audience input to shape the discussion.
Chair:
Kelvin Lee, Ph.D., Institute Director, National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)
Speakers:
Melissa Carpenter, Ph.D., Chief Scientific Officer, Regenerative Medicine, ElevateBio
Thomas Fellner, Ph.D., Vice President, Global Head of Business Development, Account Management, and Program Management, Lonza Cell & Gene Technologies
Nirupama (Rupa) Pike, Ph.D., Director, Enterprise Science and Innovation Partnerships, Global Corporate Accounts, Thermo Fisher Scientific
Joe Tarnowski, Ph.D., Senior Vice President, Cell and Gene Therapy Platform, GlaxoSmithKline
COMPANY PRESENTATIONS {BlueRock Therapeutics Ballroom}
| 4:00pm – 4:15pm | Cellatoz Therapeutics |
| 4:15pm – 4:30pm | Aspen Neuroscience |
| 4:30pm – 4:45pm | Labcorp |
| 4:45pm – 5:00pm | Mirus Bio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 5:00pm – 5:15pm | Amicus Therapeutics |
| 5:15pm – 5:30pm | Homology Medicines |
| 5:30pm – 5:45pm | AVROBIO |
| 5:45pm – 6:00pm | Orchard Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 5:00pm – 5:15pm | Forge Biologics |
| 5:15pm – 5:30pm | Life Edit Therapeutics |
| 5:30pm – 5:45pm | TreeFrog Therapeutics |
| 5:45pm – 6:00pm | GenEdit |
6:00pm | PROGRAM AND PARTNERING CLOSES
6:30pm – 9:30pm | NETWORKING BASH
Sponsored by Biopharma Excellence by PharmaLex, Cognate BioServices and L7 Informatics
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by Triangle Insights Group
7:15am – 8:45am | CONCURRENT WORKSHOPS
7:15am – 8:45am | BlueRock Therapeutics Ballroom
Sponsored by Thermo Fisher Scientific
INDUSTRIALIZATION OF CELL THERAPY MANUFACTURING – LESSONS LEARNED FROM BIOLOGICS WORKSHOP
The transformative impact that cell therapies have is evident from the observed efficacy of FDA-approved therapies to date. However, as an increasing number of drugs move from early discovery to commercialization, manufacturing innovations have moved to the forefront of the discussion. We have reached an inflection point that requires us to move from small scale, open workflows to robust, closed and automated manufacturing processes. In this session we will discuss and reflect on the various lessons that we have learned from biologics manufacturing and how they can be applied to advance the industrialization of the cell therapy manufacturing process. Join this session to hear from experts on:
- Innovation in “fit for purpose” closed cell therapy manufacturing equipment
- Controlling critical process parameters with digital integration utilizing a distributed control system (Delta V)
- Supply chain considerations
- Characterization and QC analytics for standardization
Chair:
Betty Woo, Ph.D., Vice President and General Manager, Cell and Gene Therapy, Thermo Fisher Scientific
Speakers:
Jerry Cacia, Chief Technical Officer, Graphite Bio
Andy Lin, Ph.D., Principal Technical Development Leader, Pharma Technical Development, Genentech
Sophia Sharp Donaldson, Executive Director, Supply Chain, Global Strategic Sourcing and Procurement, Kite, a Gilead company
7:15am – 8:45am | Aviara Salon A
Sponsored by IQVIA
DEMOCRATIZATION OF TRANSFORMATIVE CELL & GENE THERAPIES WORKSHOP
Recent years have seen dramatic success with gene therapy, even apparent cures for serious medical conditions (e.g., spinomuscular atrophy, sickle cell disease). Yet, for several of the diseases being cured with gene therapy, the vast majority of patients who could potentially benefit reside in low- and middle-income countries (LMIC), where such therapies are not available. The reasons underlying inaccessibility are multifold, and both near-term and long-term solutions need to be devised and implemented by the regenerative medicine industry. Near and next-term solutions come from disparate areas from utilizing information technology with patient databases/registries, to improving efficiencies and cost of manufacturing and distribution. The challenges of gene therapy implementation in LMIC span several functional areas including education and communication, regulatory, clinical preparedness, and technical / logistical aspects. Looking at the longer-range and worldwide aspirations, one initiative called the Global Gene Therapy Initiative (GGTI) includes diverse stakeholders covering broad areas of expertise from all sectors. The GGTI has set an aspirational goal of launching Phase I gene therapy clinical trials in each of two LMIC, Uganda and India, by 2024.
Chair:
Eugene Brandon, Ph.D., Head, Cell and Gene Therapy Translation, IQVIA
Speakers:
Jennifer Adair, Ph.D., Associate Professor, Clinical Research Division, Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center
Boro Dropulic, Ph.D., Executive Director, Caring Cross
Eric Faulkner, Vice President, Global Real World Evidence, Novartis Gene Therapies
Mike McCullough, Chief Information Officer, National Marrow Donor Program (NMDP)/Be The Match
7:15am – 8:45am | Aviara Salon B
Sponsored by Cell One Partners
OVERCOMING CHALLENGES TO GENE THERAPY MANUFACTURING THROUGH STANDARDIZATION & AUTOMATION WORKSHOP
Gene therapy clinical trials pose interesting challenges in GMP manufacturing. Plasmids and vectors often cannot be produced by the same vendors, may have different stabilities, and may require different storage conditions. Furthermore, some gene therapies require additional processing of the vector with target cells ex vivo. This expert-led session will explore innovations and best practices that are shortening timelines, lowering costs, and standardizing processes when manufacturing gene therapies.
Chair:
Anthony Gringeri, Ph.D., Strategic Advisor, Cell One Partners
Speakers:
David Backer, Chief Commercial Officer, Oxford Biomedica
Michelle Berg, President, GMP Nucleic Acids, Aldevron
Fabian Gerlinghaus, Co-Founder and CEO, Cellares
Judith Koliwer, Ph.D., Lead Consultant Software, Körber Business Area Pharma
9:00am | GENERAL SESSION AND PARTNERING OPENS
OVERVIEW OF ARM’S INITIATIVES
{AVROBIO Ballroom}
9:00am – 9:15am
Speaker:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
9:15am – 10:15am | CONCURRENT TRACKS
THE PAST, PRESENT & FUTURE OF GENE DELIVERY
{AVROBIO Ballroom}
9:15am – 10:15am
Gene therapy holds promise for treating and, possibly, curing a wide range of diseases, including cancer, cystic fibrosis, heart disease, and diabetes. Since the first FDA approved gene therapy trial more than 30 years ago, the advances in gene therapy have rapidly evolved. However, challenges to delivering these life-saving therapies persist, many of which are bespoke to patients. Answers may be provided in technology, automation, and scientific advances.
Chair:
Timothy Schroeder, Chairman and CEO, CTI Clinical Trial & Consulting Services
Speakers:
Patrick Boyle, Ph.D., Head of Codebase, Ginkgo Bioworks
Emma McBurney, Vice President, Business Development, Biogen
Leslie Meltzer, Ph.D., Chief Medical Officer, Orchard Therapeutics
Peter Nell, Ph.D., Chief Business Officer and Head of Therapeutics, Mammoth Biosciences
Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM); Chairman, BlueRock Therapeutics
Devyn Smith, Ph.D., CEO, Arbor Biotechnologies
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:15am – 9:30am | Cook MyoSite |
| 9:30am – 9:45am | BioLife Solutions |
| 9:45am – 10:00am | Terumo BCT |
| 10:00am – 10:15am | Bone Therapeutics |
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by Triangle Insights Group
7:15am – 8:45am | CONCURRENT WORKSHOPS
7:15am – 8:45am | BlueRock Therapeutics Ballroom
Sponsored by Thermo Fisher Scientific
INDUSTRIALIZATION OF CELL THERAPY MANUFACTURING – LESSONS LEARNED FROM BIOLOGICS WORKSHOP
The transformative impact that cell therapies have is evident from the observed efficacy of FDA-approved therapies to date. However, as an increasing number of drugs move from early discovery to commercialization, manufacturing innovations have moved to the forefront of the discussion. We have reached an inflection point that requires us to move from small scale, open workflows to robust, closed and automated manufacturing processes. In this session we will discuss and reflect on the various lessons that we have learned from biologics manufacturing and how they can be applied to advance the industrialization of the cell therapy manufacturing process. Join this session to hear from experts on:
- Innovation in “fit for purpose” closed cell therapy manufacturing equipment
- Controlling critical process parameters with digital integration utilizing a distributed control system (Delta V)
- Supply chain considerations
- Characterization and QC analytics for standardization
Chair:
Betty Woo, Ph.D., Vice President and General Manager, Cell and Gene Therapy, Thermo Fisher Scientific
Speakers:
Jerry Cacia, Chief Technical Officer, Graphite Bio
Andy Lin, Ph.D., Principal Technical Development Leader, Pharma Technical Development, Genentech
Sophia Sharp Donaldson, Executive Director, Supply Chain, Global Strategic Sourcing and Procurement, Kite, a Gilead company
7:15am – 8:45am | Aviara Salon A
Sponsored by IQVIA
DEMOCRATIZATION OF TRANSFORMATIVE CELL & GENE THERAPIES WORKSHOP
Recent years have seen dramatic success with gene therapy, even apparent cures for serious medical conditions (e.g., spinomuscular atrophy, sickle cell disease). Yet, for several of the diseases being cured with gene therapy, the vast majority of patients who could potentially benefit reside in low- and middle-income countries (LMIC), where such therapies are not available. The reasons underlying inaccessibility are multifold, and both near-term and long-term solutions need to be devised and implemented by the regenerative medicine industry. Near and next-term solutions come from disparate areas from utilizing information technology with patient databases/registries, to improving efficiencies and cost of manufacturing and distribution. The challenges of gene therapy implementation in LMIC span several functional areas including education and communication, regulatory, clinical preparedness, and technical / logistical aspects. Looking at the longer-range and worldwide aspirations, one initiative called the Global Gene Therapy Initiative (GGTI) includes diverse stakeholders covering broad areas of expertise from all sectors. The GGTI has set an aspirational goal of launching Phase I gene therapy clinical trials in each of two LMIC, Uganda and India, by 2024.
Chair:
Eugene Brandon, Ph.D., Head, Cell and Gene Therapy Translation, IQVIA
Speakers:
Jennifer Adair, Ph.D., Associate Professor, Clinical Research Division, Stem Cell and Gene Therapy Program, Fred Hutchinson Cancer Research Center
Boro Dropulic, Ph.D., Executive Director, Caring Cross
Eric Faulkner, Vice President, Global Real World Evidence, Novartis Gene Therapies
Mike McCullough, Chief Information Officer, National Marrow Donor Program (NMDP)/Be The Match
7:15am – 8:45am | Aviara Salon B
Sponsored by Cell One Partners
OVERCOMING CHALLENGES TO GENE THERAPY MANUFACTURING THROUGH STANDARDIZATION & AUTOMATION WORKSHOP
Gene therapy clinical trials pose interesting challenges in GMP manufacturing. Plasmids and vectors often cannot be produced by the same vendors, may have different stabilities, and may require different storage conditions. Furthermore, some gene therapies require additional processing of the vector with target cells ex vivo. This expert-led session will explore innovations and best practices that are shortening timelines, lowering costs, and standardizing processes when manufacturing gene therapies.
Chair:
Anthony Gringeri, Ph.D., Strategic Advisor, Cell One Partners
Speakers:
David Backer, Chief Commercial Officer, Oxford Biomedica
Michelle Berg, President, GMP Nucleic Acids, Aldevron
Fabian Gerlinghaus, Co-Founder and CEO, Cellares
Judith Koliwer, Ph.D., Lead Consultant Software, Körber Business Area Pharma
9:00am | GENERAL SESSION AND PARTNERING OPENS
OVERVIEW OF ARM’S INITIATIVES
{AVROBIO Ballroom}
9:00am – 9:15am
Speaker:
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
9:15am – 10:15am | CONCURRENT TRACKS
THE PAST, PRESENT & FUTURE OF GENE DELIVERY
{AVROBIO Ballroom}
9:15am – 10:15am
Gene therapy holds promise for treating and, possibly, curing a wide range of diseases, including cancer, cystic fibrosis, heart disease, and diabetes. Since the first FDA approved gene therapy trial more than 30 years ago, the advances in gene therapy have rapidly evolved. However, challenges to delivering these life-saving therapies persist, many of which are bespoke to patients. Answers may be provided in technology, automation, and scientific advances.
Chair:
Timothy Schroeder, Chairman and CEO, CTI Clinical Trial & Consulting Services
Speakers:
Patrick Boyle, Ph.D., Head of Codebase, Ginkgo Bioworks
Emma McBurney, Vice President, Business Development, Biogen
Leslie Meltzer, Ph.D., Chief Medical Officer, Orchard Therapeutics
Peter Nell, Ph.D., Chief Business Officer and Head of Therapeutics, Mammoth Biosciences
Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM); Chairman, BlueRock Therapeutics
Devyn Smith, Ph.D., CEO, Arbor Biotechnologies
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:15am – 9:30am | Cook MyoSite |
| 9:30am – 9:45am | BioLife Solutions |
| 9:45am – 10:00am | Terumo BCT |
| 10:00am – 10:15am | Bone Therapeutics |
10:15am – 10:45am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
10:45am – 12:00pm | CONCURRENT TRACKS
INNOVATIVE REGULATORY PATHWAYS FOR NEXT-GENERATION TREATMENTS
{AVROBIO Ballroom}
10:45am – 11:15am
Cell and gene therapies are complex and the current regulations around them are also multifaceted – and constantly evolving. Regulatory agencies need to be involved throughout the development process to keep in lockstep and build their insights into the program. Ultimately, the goal is to create and execute an effective and efficient strategy that leaves nothing to chance. This session will feature discussion between leading developers of innovative therapies sharing insights about their key regulatory considerations and pathways to market.
Chair:
Mark Lane, Ph.D., Vice President, Development Consulting and Scientific Affairs, Biopharma Excellence by PharmaLex
Speakers:
Jennifer Dittman, Vice President, Regulatory Affairs, Generation Bio
Rachelle Jacques, CEO, Enzyvant
Jeff Ross, Ph.D., CEO, Miromatrix
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 10:45am – 11:00am | Akron Biotech |
| 11:00am – 11:15am | MEDIPOST America |
STRATEGIES FOR MANAGING ACCESS & REIMBURSEMENT CHALLENGES
{AVROBIO Ballroom}
11:15am – 12:00pm
Chair:
Katy Spink, Ph.D., Chief Operating Officer and Managing Partner, Dark Horse Consulting Group
Speakers:
Eric Faulkner, Vice President, Global Real World Evidence, Novartis Gene Therapies
Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics
Kristin Wolff, Vice President, Global Policy Strategy and U.S. Government Payer, bluebird bio
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 11:15am – 11:30am | AGTC |
| 11:30am – 11:45am | ConeSight Therapeutics |
| 11:45am – 12:00pm | SparingVision |
12:00pm – 1:15pm | LUNCH
Sponsored by Yposkesi
1:15pm – 2:45pm | CONCURRENT TRACKS
UPDATE ON THE ARM FOUNDATION FOR CELL & GENE MEDICINE
{AVROBIO Ballroom}
1:15pm – 1:25pm
Speakers:
Brett Kopelan, Chairman of the Board, ARM Foundation for Cell & Gene Medicine; Executive Director, debra of America
Martha Rook, Ph.D., Treasurer, ARM Foundation for Cell & Gene Medicine; Chief Technical Operations Officer, Sigilon Therapeutics
COMPANY PRESENTATION
{BlueRock Therapeutics Ballroom}
| 1:15pm – 1:30pm | CTI Clinical Trial & Consulting |
THE INFORMATION PATIENTS WANT ABOUT REGENERATIVE MEDICINE
{AVROBIO Ballroom}
1:25pm – 2:00pm
Chair:
Brett Kopelan, Chairman of the Board, ARM Foundation for Cell & Gene Medicine; Executive Director, debra of America
Speakers:
Leonard Valentino, M.D., President and CEO, National Hemophilia Foundation
Ellen Wagner, Special Projects Coordinator, Parent Project Muscular Dystrophy (PPMD)
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 1:30pm – 1:45pm | ElevateBio |
| 1:45pm – 2:00pm | GentiBio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 2:00pm – 2:15pm | Sangamo Therapeutics |
| 2:15pm – 2:30pm | uniQure |
| 2:30pm – 2:45pm | Sigilon Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 2:00pm – 2:15pm | Virica Biotech |
| 2:15pm – 2:30pm | Enzyvant Therapeutics |
| 2:30pm – 2:45pm | Sentien Biotechnologies |
2:45pm – 3:15pm | AFTERNOON BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
3:15pm – 6:00pm | CONCURRENT TRACKS
PERSONALIZING CARDIOVASCULAR THERAPIES TO ADDRESS THE UNDERLYING CAUSES OF DISEASE
{AVROBIO Ballroom}
3:15pm – 4:15pm
Cell and gene therapies are some of the most cutting-edge innovations in cardiovascular (CV) medicine and represent the next frontier of this therapeutic area. Even though CV cell and gene therapies have great promise to significantly improve clinical outcomes, it is still an emerging field, with a history of clinical trials yielding mixed results. Some of the key challenges in developing these therapies include understanding the true mechanisms of action, identifying the most effective cell types, dosing, CV clinical targets, and routes and frequency of administration. Other important questions about CV cell and gene therapy clinical development focus on how best to design clinical trials, what target populations to focus on, and what clinical development paradigms to use to successfully advance the field. This session includes leaders in the field who have already grappled with many of these questions, and have successfully addressed them to launch CV clinical development programs.
Chair:
Monica Shah, M.D., Vice President and Head, Cell and Gene Therapy Center of Excellence, IQVIA
Speakers:
Faraz Ali, CEO, Tenaya Therapeutics
Joachim Fruebis, Ph.D., Chief Development Officer, BlueRock Therapeutics
Jordan Lancaster, Ph.D., Co-founder and CEO, Avery Therapeutics
William “B.J.” Lehmann, Interim CEO, Athersys
Michael Scott, Ph.D., Vice President, Cell Therapy Medical Devices, Novo Nordisk
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 3:15pm – 3:30pm | Immusoft |
| 3:30pm – 3:45pm | Capricor Therapeutics |
| 3:45pm – 4:00pm | PBS Biotech |
| 4:00pm – 4:15pm | L7 Informatics |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 4:15pm – 4:30pm | Allogene Therapeutics |
| 4:30pm – 4:45pm | Mission Bio |
| 4:45pm – 5:00pm | Synthego |
| 5:00pm – 5:15pm | MaxCyte |
| 5:15pm – 5:30pm | Vor Biopharma |
| 5:30pm – 5:45pm | Poseida Therapeutics |
| 5:45pm – 6:00pm | Kuur Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 4:15pm – 4:30pm | Sensorion |
| 4:30pm – 4:45pm | Likarda |
| 4:45pm – 5:00pm | Tenaya Therapeutics |
| 5:00pm – 5:15pm | Vineti |
| 5:15pm – 5:30pm | Rejuvenate Bio |
| 5:30pm – 5:45pm | Avery Therapeutics |
6:00pm | PROGRAM AND PARTNERING CLOSES
10:15am – 10:45am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
10:45am – 12:00pm | CONCURRENT TRACKS
INNOVATIVE REGULATORY PATHWAYS FOR NEXT-GENERATION TREATMENTS
{AVROBIO Ballroom}
10:45am – 11:15am
Cell and gene therapies are complex and the current regulations around them are also multifaceted – and constantly evolving. Regulatory agencies need to be involved throughout the development process to keep in lockstep and build their insights into the program. Ultimately, the goal is to create and execute an effective and efficient strategy that leaves nothing to chance. This session will feature discussion between leading developers of innovative therapies sharing insights about their key regulatory considerations and pathways to market.
Chair:
Mark Lane, Ph.D., Vice President, Development Consulting and Scientific Affairs, Biopharma Excellence by PharmaLex
Speakers:
Jennifer Dittman, Vice President, Regulatory Affairs, Generation Bio
Rachelle Jacques, CEO, Enzyvant
Jeff Ross, Ph.D., CEO, Miromatrix
COMPANY PRESENTATIONS {BlueRock Therapeutics Ballroom}
| 10:45am – 11:00am | Akron Biotech |
| 11:00am – 11:15am | MEDIPOST America |
STRATEGIES FOR MANAGING ACCESS & REIMBURSEMENT CHALLENGES
{AVROBIO Ballroom}
11:15am – 12:00pm
Chair:
Katy Spink, Ph.D., Chief Operating Officer and Managing Partner, Dark Horse Consulting Group
Speakers:
Eric Faulkner, Vice President, Global Real World Evidence, Novartis Gene Therapies
Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics
Kristin Wolff, Vice President, Global Policy Strategy and U.S. Government Payer, bluebird bio
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 11:15am – 11:30am | AGTC |
| 11:30am – 11:45am | ConeSight Therapeutics |
| 11:45am – 12:00pm | SparingVision |
12:00pm – 1:15pm | LUNCH
Sponsored by Yposkesi
1:15pm – 2:45pm | CONCURRENT TRACKS
UPDATE ON THE ARM FOUNDATION FOR CELL & GENE MEDICINE
{AVROBIO Ballroom}
1:15pm – 1:25pm
Speakers:
Brett Kopelan, Chairman of the Board, ARM Foundation for Cell & Gene Medicine; Executive Director, debra of America
Martha Rook, Ph.D., Treasurer, ARM Foundation for Cell & Gene Medicine; Chief Technical Operations Officer, Sigilon Therapeutics
COMPANY PRESENTATION {BlueRock Therapeutics Ballroom}
| 1:15pm – 1:30pm | CTI Clinical Trial & Consulting |
THE INFORMATION PATIENTS WANT ABOUT REGENERATIVE MEDICINE
{AVROBIO Ballroom}
1:25pm – 2:00pm
Chair:
Brett Kopelan, Chairman of the Board, ARM Foundation for Cell & Gene Medicine; Executive Director, debra of America
Speakers:
Leonard Valentino, M.D., President and CEO, National Hemophilia Foundation
Ellen Wagner, Special Projects Coordinator, Parent Project Muscular Dystrophy (PPMD)
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 1:30pm – 1:45pm | ElevateBio |
| 1:45pm – 2:00pm | GentiBio |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 2:00pm – 2:15pm | Sangamo Therapeutics |
| 2:15pm – 2:30pm | uniQure |
| 2:30pm – 2:45pm | Sigilon Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 2:00pm – 2:15pm | Virica Biotech |
| 2:15pm – 2:30pm | Enzyvant Therapeutics |
| 2:30pm – 2:45pm | Sentien Biotechnologies |
2:45pm – 3:15pm | AFTERNOON BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
3:15pm – 6:00pm | CONCURRENT TRACKS
PERSONALIZING CARDIOVASCULAR THERAPIES TO ADDRESS THE UNDERLYING CAUSES OF DISEASE
{AVROBIO Ballroom}
3:15pm – 4:15pm
Cell and gene therapies are some of the most cutting-edge innovations in cardiovascular (CV) medicine and represent the next frontier of this therapeutic area. Even though CV cell and gene therapies have great promise to significantly improve clinical outcomes, it is still an emerging field, with a history of clinical trials yielding mixed results. Some of the key challenges in developing these therapies include understanding the true mechanisms of action, identifying the most effective cell types, dosing, CV clinical targets, and routes and frequency of administration. Other important questions about CV cell and gene therapy clinical development focus on how best to design clinical trials, what target populations to focus on, and what clinical development paradigms to use to successfully advance the field. This session includes leaders in the field who have already grappled with many of these questions, and have successfully addressed them to launch CV clinical development programs.
Chair:
Monica Shah, M.D., Vice President and Head, Cell and Gene Therapy Center of Excellence, IQVIA
Speakers:
Faraz Ali, CEO, Tenaya Therapeutics
Joachim Fruebis, Ph.D., Chief Development Officer, BlueRock Therapeutics
Jordan Lancaster, Ph.D., Co-founder and CEO, Avery Therapeutics
William “B.J.” Lehmann, Interim CEO, Athersys
Michael Scott, Ph.D., Vice President, Cell Therapy Medical Devices, Novo Nordisk
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 3:15pm – 3:30pm | Immusoft |
| 3:30pm – 3:45pm | Capricor Therapeutics |
| 3:45pm – 4:00pm | PBS Biotech |
| 4:00pm – 4:15pm | L7 Informatics |
COMPANY PRESENTATIONS
{AVROBIO Ballroom}
| 4:15pm – 4:30pm | Allogene Therapeutics |
| 4:30pm – 4:45pm | Mission Bio |
| 4:45pm – 5:00pm | Synthego |
| 5:00pm – 5:15pm | MaxCyte |
| 5:15pm – 5:30pm | Vor Biopharma |
| 5:30pm – 5:45pm | Poseida Therapeutics |
| 5:45pm – 6:00pm | Kuur Therapeutics |
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 4:15pm – 4:30pm | Sensorion |
| 4:30pm – 4:45pm | Likarda |
| 4:45pm – 5:00pm | Tenaya Therapeutics |
| 5:00pm – 5:15pm | Vineti |
| 5:15pm – 5:30pm | Rejuvenate Bio |
| 5:30pm – 5:45pm | Avery Therapeutics |
6:00pm | PROGRAM AND PARTNERING CLOSES
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by CPC
7:15am – 8:45am | MORNING WORKSHOP
7:15am – 8:45am | Aviara Salon B
Sponsored by CSL Behring
DELIVERING THE INNOVATION OF GENE THERAPY TO PATIENTS THROUGH STRATEGIC COLLABORATIONS WORKSHOP
This workshop will explore case studies where strategic collaborations have been the pathway of choice for gene therapy innovators to ensure an optimal route to commercialization. Leading executives in the gene therapy sector will discuss the rationale of these strategic partnerships along with key learnings for successful implementation.
Chair:
Debbie Drane, Senior Vice President, Global Commercial Development and TA Strategy, CSL Behring
Speakers:
Lawrence Kau, Global Commercial Strategy Lead, Spark Therapeutics
Josh Leeman, Ph.D., Vice President, Business Development, Search and Evaluation, uniQure
Adam Simpson, Ph.D., Head, Gene Therapies BD&L, Novartis Institutes for BioMedical Research
9:00am | GENERAL SESSION AND PARTNERING OPENS
ARM GROW INTERNSHIP UPDATE {AVROBIO Ballroom}
9:00am – 9:15am
Speakers:
Ifeoluwa Awoleye, ARM GROW Intern, Prevail Therapeutics
Rashida Dujue-Jackson, AFE Program Director, Alliance for Regenerative Medicine (ARM)
Rachelle Jacques, CEO, Enzyvant
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Jamila Ritter, ARM GROW Intern, Amicus Therapeutics
9:15am – 10:45am | CONCURRENT TRACKS
SYNTHETIC BIOLOGY APPLICATIONS IN CELL & GENE THERAPY
{AVROBIO Ballroom}
9:15am – 9:45am
Chair:
Robert Deans, Ph.D., Chief Scientific Officer, Synthego
Speakers:
Timothy Lu, M.D., Ph.D., Co-founder and CEO, Senti Biosciences
Ali Nahvi, Ph.D., Head of New Therapeutic Modalities, Spark Therapeutics
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:15am – 9:30am | Mammoth Biosciences |
| 9:30am – 9:45am | Stemson Therapeutics |
WHAT CELL & GENE THERAPY INVESTORS ARE LOOKING FOR NOW
{AVROBIO Ballroom}
9:45am – 10:45am
Chair:
Phil Vanek, Ph.D., Chief Technology Officer, Gamma Biosciences
Speakers:
Gbola Amusa, M.D., Partner and Chief Scientific Officer, Chardan
Chris Garabedian, Chairman and CEO, Xontogeny
Ed Hurwitz, Managing Director, MPM Capital
Deborah Palestrant, Ph.D., Partner and Head of 4:59, 5AM Ventures
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:45am – 10:00am | Endsulin |
| 10:00am – 10:15am | Arbor Biotechnologies |
| 10:15am – 10:30am | ViaCyte |
| 10:30am – 10:45am | Castle Creek Biosciences |
10:45am – 11:00am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
11:00am – 12:00pm | CONCURRENT TRACKS
DOSING & REDOSING IN GENE THERAPY
{AVROBIO Ballroom}
11:00am – 12:00pm
Since the earliest days of gene therapy, dosing has been a key pivotal issue to balance with tremendous benefit of this technology with its inherent risks. Today, understanding considerations of dosing across diverse patient populations for which we intend treatment is much improved, but still requires empiric trials. With a considerable number of key questions still to be answered, our goal should be to collectively and comprehensively explore issues in dosing, and ultimately, re-dosing of patients. Are we doing enough as an industry to confidently employ dosing strategies for gene therapies?
Chair:
Chris Mason, M.D., Ph.D., Chief Scientific Officer, AVROBIO
Speakers:
Eric Crombez, M.D., Chief Medical Officer, Ultragenyx Gene Therapy
Peter Francis, M.D., Ph.D., Chief Scientific Officer, Ophthalmology Therapeutic Area Head, 4D Molecular Therapeutics
Timothy Miller, Ph.D., CEO, President and Co-Founder, Forge Biologics
Nicole Paulk, Ph.D., Professor, AAV Gene Therapy, University of California, San Francisco
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 11:00am – 11:15am | Aspect Biosystems |
| 11:15am – 11:30am | Trailhead Biosystems |
12:00pm – 1:30pm | LUNCH
Sponsored by VGXI
1:30pm | CONFERENCE CLOSES
6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by CPC
7:15am – 8:45am | MORNING WORKSHOP
7:15am – 8:45am | Aviara Salon B
Sponsored by CSL Behring
DELIVERING THE INNOVATION OF GENE THERAPY TO PATIENTS THROUGH STRATEGIC COLLABORATIONS WORKSHOP
This workshop will explore case studies where strategic collaborations have been the pathway of choice for gene therapy innovators to ensure an optimal route to commercialization. Leading executives in the gene therapy sector will discuss the rationale of these strategic partnerships along with key learnings for successful implementation.
Chair:
Debbie Drane, Senior Vice President, Global Commercial Development and TA Strategy, CSL Behring
Speakers:
Lawrence Kau, Global Commercial Strategy Lead, Spark Therapeutics
Josh Leeman, Ph.D., Vice President, Business Development, Search and Evaluation, uniQure
Adam Simpson, Ph.D., Head, Gene Therapies BD&L, Novartis Institutes for BioMedical Research
9:00am | GENERAL SESSION AND PARTNERING OPENS
ARM GROW INTERNSHIP UPDATE {AVROBIO Ballroom}
9:00am – 9:15am
Speakers:
Ifeoluwa Awoleye, ARM GROW Intern, Prevail Therapeutics
Rashida Dujue-Jackson, AFE Program Director, Alliance for Regenerative Medicine (ARM)
Rachelle Jacques, CEO, Enzyvant
Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)
Jamila Ritter, ARM GROW Intern, Amicus Therapeutics
9:15am – 10:45am | CONCURRENT TRACKS
SYNTHETIC BIOLOGY APPLICATIONS IN CELL & GENE THERAPY
{AVROBIO Ballroom}
9:15am – 9:45am
Chair:
Robert Deans, Ph.D., Chief Scientific Officer, Synthego
Speakers:
Timothy Lu, M.D., Ph.D., Co-founder and CEO, Senti Biosciences
Ali Nahvi, Ph.D., Head of New Therapeutic Modalities, Spark Therapeutics
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:15am – 9:30am | Mammoth Biosciences |
| 9:30am – 9:45am | Stemson Therapeutics |
WHAT CELL & GENE THERAPY INVESTORS ARE LOOKING FOR NOW
{AVROBIO Ballroom}
9:45am – 10:45am
Chair:
Phil Vanek, Ph.D., Chief Technology Officer, Gamma Biosciences
Speakers:
Gbola Amusa, M.D., Partner and Chief Scientific Officer, Chardan
Chris Garabedian, Chairman and CEO, Xontogeny
Ed Hurwitz, Managing Director, MPM Capital
Deborah Palestrant, Ph.D., Partner and Head of 4:59, 5AM Ventures
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 9:45am – 10:00am | Endsulin |
| 10:00am – 10:15am | Arbor Biotechnologies |
| 10:15am – 10:30am | ViaCyte |
| 10:30am – 10:45am | Castle Creek Biosciences |
10:45am – 11:00am | MORNING BREAK
Sponsored by Bone Therapeutics, Cytotheryx, Invetech and POMS
11:00am – 12:00pm | CONCURRENT TRACKS
DOSING & REDOSING IN GENE THERAPY
{AVROBIO Ballroom}
11:00am – 12:00pm
Since the earliest days of gene therapy, dosing has been a key pivotal issue to balance with tremendous benefit of this technology with its inherent risks. Today, understanding considerations of dosing across diverse patient populations for which we intend treatment is much improved, but still requires empiric trials. With a considerable number of key questions still to be answered, our goal should be to collectively and comprehensively explore issues in dosing, and ultimately, re-dosing of patients. Are we doing enough as an industry to confidently employ dosing strategies for gene therapies?
Chair:
Chris Mason, M.D., Ph.D., Chief Scientific Officer, AVROBIO
Speakers:
Eric Crombez, M.D., Chief Medical Officer, Ultragenyx Gene Therapy
Peter Francis, M.D., Ph.D., Chief Scientific Officer, Ophthalmology Therapeutic Area Head, 4D Molecular Therapeutics
Timothy Miller, Ph.D., CEO, President and Co-Founder, Forge Biologics
Nicole Paulk, Ph.D., Professor, AAV Gene Therapy, University of California, San Francisco
COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}
| 11:00am – 11:15am | Aspect Biosystems |
| 11:15am – 11:30am | Trailhead Biosystems |
12:00pm – 1:30pm | LUNCH
Sponsored by VGXI
1:30pm | CONFERENCE CLOSES
The Cell & Gene Meeting on the Mesa is the sector’s foremost annual conference bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures. Tackling the commercialization hurdles facing the cell and gene therapy sector today, this meeting covers a wide range of topics from clinical trial design to alternative payment models to scale-up and supply chain platforms for advanced therapies. The program features expert-led panels, extensive partnering capabilities, exclusive networking opportunities, and 100+ dedicated presentations by the leading publicly traded and privately held companies in the space. Attracting over 1,200 attendees – over 20% of which are C-level executives – this conference enables key partnerships through more than 3,000 one-on-one meetings while highlighting the significant clinical and commercial progress in the field.
October 12-14, 2021
Carlsbad, CA
