Agenda

2022 Agenda Coming Soon!

The 2022 Cell & Gene Meeting on the Mesa will be delivered in a hybrid format with live programming available over the course of three days. Virtual registrants will have access to all content via livestream during the program dates. Additionally, all content will be made available on-demand within 24 hours of the live program time for all registrants to view. 

Agenda Sponsor:

Agenda Sponsor:

Last Year's Agenda

  • Full Agenda
  • On-Demand
  • Day 1 - Oct. 12
  • Day 2 - Oct. 13
  • Day 3 - Oct. 14

ON-DEMAND VIRTUAL CONTENT

Available Beginning Tuesday, October 12, 2021

  • DOING BUSINESS IN JAPAN – AN EVOLVING MARKET WORKSHOP
  • FROM CONCEPT TO COMMERCIALIZATION IN LIVING THERAPEUTICS: HINDSIGHT IS 20/20 WORKSHOP
  • BESPOKE THERAPIES FOR ULTRA-RARE DISEASE WORKSHOP

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)

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

  • WHAT’S NEXT FOR ADVANCED THERAPIES WORKSHOP
  • UNLOCKING THE POWER OF COLLABORATION TO FUEL THE INDUSTRY TRANSFORMATION FOR CELL & GENE THERAPIES WORKSHOP

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

  • DOING BUSINESS IN JAPAN – AN EVOLVING MARKET WORKSHOP
  • FROM CONCEPT TO COMMERCIALIZATION IN LIVING THERAPEUTICS: HINDSIGHT IS 20/20 WORKSHOP
  • BESPOKE THERAPIES FOR ULTRA-RARE DISEASE WORKSHOP

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)

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

  • WHAT’S NEXT FOR ADVANCED THERAPIES WORKSHOP
  • UNLOCKING THE POWER OF COLLABORATION TO FUEL THE INDUSTRY TRANSFORMATION FOR CELL & GENE THERAPIES WORKSHOP

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 – 11:00amEmendo Biotherapeutics
11:00am – 11:15amEnsoma

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

11:15am – 11:30amOxford Biomedica
11:30am – 11:45am4DMT
11:45am – 12:00pmHelixmith

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

11:15am – 11:30amOrca Bio
11:30am – 11:45amAldevron
11:45am – 12:00pmInVitria

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 – 1:30pmAffyImmune Therapeutics
1:30pm – 1:45pmAngiocrine Bioscience
1:45pm – 2:00pmArtisan Bio
2:00pm – 2:15pmCatamaran Bio

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

2:15pm – 2:30pmBlueRock Therapeutics
2:30pm – 2:45pmTaysha Gene Therapies
2:45pm – 3:00pmPassage Bio
3:00pm – 3:15pmAthersys
3:15pm – 3:30pmGinkgo Bioworks

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

2:15pm – 2:30pmRocket Pharmaceuticals
2:30pm – 2:45pmLuminary Therapeutics
2:45pm – 3:00pmSenti Biosciences
3:00pm – 3:15pmShoreline Biosciences
3:15pm – 3:30pmDiscGenics

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 – 4:15pmCellatoz Therapeutics
4:15pm – 4:30pmAspen Neuroscience
4:30pm – 4:45pmLabcorp
4:45pm – 5:00pmMirus Bio

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

5:00pm – 5:15pmAmicus Therapeutics
5:15pm – 5:30pmHomology Medicines
5:30pm – 5:45pmAVROBIO
5:45pm – 6:00pmOrchard Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

5:00pm – 5:15pmForge Biologics
5:15pm – 5:30pmLife Edit Therapeutics
5:30pm – 5:45pmTreeFrog Therapeutics
5:45pm – 6:00pmGenEdit

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 – 11:00amEmendo Biotherapeutics
11:00am – 11:15amEnsoma

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

11:15am – 11:30amOxford Biomedica
11:30am – 11:45am4DMT
11:45am – 12:00pmHelixmith

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

11:15am – 11:30amOrca Bio
11:30am – 11:45amAldevron
11:45am – 12:00pmInVitria

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 – 1:30pmAffyImmune Therapeutics
1:30pm – 1:45pmAngiocrine Bioscience
1:45pm – 2:00pmArtisan Bio
2:00pm – 2:15pmCatamaran Bio

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

2:15pm – 2:30pmBlueRock Therapeutics
2:30pm – 2:45pmTaysha Gene Therapies
2:45pm – 3:00pmPassage Bio
3:00pm – 3:15pmAthersys
3:15pm – 3:30pmGinkgo Bioworks

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

2:15pm – 2:30pmRocket Pharmaceuticals
2:30pm – 2:45pmLuminary Therapeutics
2:45pm – 3:00pmSenti Biosciences
3:00pm – 3:15pmShoreline Biosciences
3:15pm – 3:30pmDiscGenics

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 – 4:15pmCellatoz Therapeutics
4:15pm – 4:30pmAspen Neuroscience
4:30pm – 4:45pmLabcorp
4:45pm – 5:00pmMirus Bio

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

5:00pm – 5:15pmAmicus Therapeutics
5:15pm – 5:30pmHomology Medicines
5:30pm – 5:45pmAVROBIO
5:45pm – 6:00pmOrchard Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

5:00pm – 5:15pmForge Biologics
5:15pm – 5:30pmLife Edit Therapeutics
5:30pm – 5:45pmTreeFrog Therapeutics
5:45pm – 6:00pmGenEdit

6:00pm | PROGRAM AND PARTNERING CLOSES

6:30pm – 9:30pm | NETWORKING BASH

Sponsored by Biopharma Excellence by PharmaLex, Cognate BioServices and L7 Informatics

Mesa21-Agenda-Day2

6:45am – 9:15am | REGISTRATION AND BREAKFAST

Sponsored by Triangle Insights Group

7:15am – 8:45am | CONCURRENT WORKSHOPS

  • INDUSTRIALIZATION OF CELL THERAPY MANUFACTURING – LESSONS LEARNED FROM BIOLOGICS WORKSHOP
  • DEMOCRATIZATION OF TRANSFORMATIVE CELL & GENE THERAPIES WORKSHOP
  • OVERCOMING CHALLENGES TO GENE THERAPY MANUFACTURING THROUGH STANDARDIZATION & AUTOMATION WORKSHOP

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 – 9:30amCook MyoSite
9:30am – 9:45amBioLife Solutions
9:45am – 10:00amTerumo BCT
10:00am – 10:15amBone Therapeutics
Mesa21-Agenda-Day2

6:45am – 9:15am | REGISTRATION AND BREAKFAST

Sponsored by Triangle Insights Group

7:15am – 8:45am | CONCURRENT WORKSHOPS

  • INDUSTRIALIZATION OF CELL THERAPY MANUFACTURING – LESSONS LEARNED FROM BIOLOGICS WORKSHOP
  • DEMOCRATIZATION OF TRANSFORMATIVE CELL & GENE THERAPIES WORKSHOP
  • OVERCOMING CHALLENGES TO GENE THERAPY MANUFACTURING THROUGH STANDARDIZATION & AUTOMATION WORKSHOP

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 – 9:30amCook MyoSite
9:30am – 9:45amBioLife Solutions
9:45am – 10:00amTerumo BCT
10:00am – 10:15amBone 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 – 11:00amAkron Biotech
11:00am – 11:15amMEDIPOST 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 – 1:30pmCTI 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 – 2:15pmSangamo Therapeutics
2:15pm – 2:30pmuniQure
2:30pm – 2:45pmSigilon Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

2:00pm – 2:15pmVirica Biotech
2:15pm – 2:30pmEnzyvant Therapeutics
2:30pm – 2:45pmSentien 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 – 3:30pmImmusoft
3:30pm – 3:45pmCapricor Therapeutics
3:45pm – 4:00pmPBS Biotech
4:00pm – 4:15pmL7 Informatics

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

4:15pm – 4:30pmAllogene Therapeutics
4:30pm – 4:45pmMission Bio
4:45pm – 5:00pmSynthego
5:00pm – 5:15pmMaxCyte
5:15pm – 5:30pmVor Biopharma
5:30pm – 5:45pmPoseida Therapeutics
5:45pm – 6:00pmKuur Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

4:15pm – 4:30pmSensorion
4:30pm – 4:45pmLikarda
4:45pm – 5:00pmTenaya Therapeutics
5:00pm – 5:15pmVineti
5:15pm – 5:30pmRejuvenate Bio
5:30pm – 5:45pmAvery 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 – 11:00amAkron Biotech
11:00am – 11:15amMEDIPOST 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 – 1:30pmCTI 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 – 2:15pmSangamo Therapeutics
2:15pm – 2:30pmuniQure
2:30pm – 2:45pmSigilon Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

2:00pm – 2:15pmVirica Biotech
2:15pm – 2:30pmEnzyvant Therapeutics
2:30pm – 2:45pmSentien 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 – 3:30pmImmusoft
3:30pm – 3:45pmCapricor Therapeutics
3:45pm – 4:00pmPBS Biotech
4:00pm – 4:15pmL7 Informatics

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

4:15pm – 4:30pmAllogene Therapeutics
4:30pm – 4:45pmMission Bio
4:45pm – 5:00pmSynthego
5:00pm – 5:15pmMaxCyte
5:15pm – 5:30pmVor Biopharma
5:30pm – 5:45pmPoseida Therapeutics
5:45pm – 6:00pmKuur Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

4:15pm – 4:30pmSensorion
4:30pm – 4:45pmLikarda
4:45pm – 5:00pmTenaya Therapeutics
5:00pm – 5:15pmVineti
5:15pm – 5:30pmRejuvenate Bio
5:30pm – 5:45pmAvery Therapeutics

6:00pm | PROGRAM AND PARTNERING CLOSES

6:45am – 9:15am | REGISTRATION AND BREAKFAST

Sponsored by CPC

7:15am – 8:45am | MORNING WORKSHOP

  • DELIVERING THE INNOVATION OF GENE THERAPY TO PATIENTS THROUGH STRATEGIC COLLABORATIONS 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 – 9:30amMammoth Biosciences
9:30am – 9:45amStemson 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 – 10:00amEndsulin
10:00am – 10:15amArbor Biotechnologies
10:15am – 10:30amViaCyte
10:30am – 10:45amCastle 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 – 11:15amAspect Biosystems
11:15am – 11:30amTrailhead 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

  • DELIVERING THE INNOVATION OF GENE THERAPY TO PATIENTS THROUGH STRATEGIC COLLABORATIONS 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 – 9:30amMammoth Biosciences
9:30am – 9:45amStemson 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 – 10:00amEndsulin
10:00am – 10:15amArbor Biotechnologies
10:15am – 10:30amViaCyte
10:30am – 10:45amCastle 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 – 11:15amAspect Biosystems
11:15am – 11:30amTrailhead Biosystems

12:00pm – 1:30pm | LUNCH

Sponsored by VGXI

1:30pm | CONFERENCE CLOSES

ON-DEMAND VIRTUAL CONTENT

Available Beginning Tuesday, October 12, 2021

  • DOING BUSINESS IN JAPAN – AN EVOLVING MARKET WORKSHOP
  • FROM CONCEPT TO COMMERCIALIZATION IN LIVING THERAPEUTICS: HINDSIGHT IS 20/20 WORKSHOP
  • BESPOKE THERAPIES FOR ULTRA-RARE DISEASE WORKSHOP

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)

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)

ON-DEMAND VIRTUAL CONTENT

Available Beginning Tuesday, October 12, 2021

  • DOING BUSINESS IN JAPAN – AN EVOLVING MARKET WORKSHOP
  • FROM CONCEPT TO COMMERCIALIZATION IN LIVING THERAPEUTICS: HINDSIGHT IS 20/20 WORKSHOP
  • BESPOKE THERAPIES FOR ULTRA-RARE DISEASE WORKSHOP

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)

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

  • WHAT’S NEXT FOR ADVANCED THERAPIES WORKSHOP
  • UNLOCKING THE POWER OF COLLABORATION TO FUEL THE INDUSTRY TRANSFORMATION FOR CELL & GENE THERAPIES WORKSHOP

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

  • WHAT’S NEXT FOR ADVANCED THERAPIES WORKSHOP
  • UNLOCKING THE POWER OF COLLABORATION TO FUEL THE INDUSTRY TRANSFORMATION FOR CELL & GENE THERAPIES WORKSHOP

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 – 11:00amEmendo Biotherapeutics
11:00am – 11:15amEnsoma

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

11:15am – 11:30amOxford Biomedica
11:30am – 11:45am4DMT
11:45am – 12:00pmHelixmith

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

11:15am – 11:30amOrca Bio
11:30am – 11:45amAldevron
11:45am – 12:00pmInVitria

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 – 1:30pmAffyImmune Therapeutics
1:30pm – 1:45pmAngiocrine Bioscience
1:45pm – 2:00pmArtisan Bio
2:00pm – 2:15pmCatamaran Bio

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

2:15pm – 2:30pmBlueRock Therapeutics
2:30pm – 2:45pmTaysha Gene Therapies
2:45pm – 3:00pmPassage Bio
3:00pm – 3:15pmAthersys
3:15pm – 3:30pmGinkgo Bioworks

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

2:15pm – 2:30pmRocket Pharmaceuticals
2:30pm – 2:45pmLuminary Therapeutics
2:45pm – 3:00pmSenti Biosciences
3:00pm – 3:15pmShoreline Biosciences
3:15pm – 3:30pmDiscGenics

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 – 4:15pmCellatoz Therapeutics
4:15pm – 4:30pmAspen Neuroscience
4:30pm – 4:45pmLabcorp
4:45pm – 5:00pmMirus Bio

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

5:00pm – 5:15pmAmicus Therapeutics
5:15pm – 5:30pmHomology Medicines
5:30pm – 5:45pmAVROBIO
5:45pm – 6:00pmOrchard Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

5:00pm – 5:15pmForge Biologics
5:15pm – 5:30pmLife Edit Therapeutics
5:30pm – 5:45pmTreeFrog Therapeutics
5:45pm – 6:00pmGenEdit

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 – 11:00amEmendo Biotherapeutics
11:00am – 11:15amEnsoma

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

11:15am – 11:30amOxford Biomedica
11:30am – 11:45am4DMT
11:45am – 12:00pmHelixmith

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

11:15am – 11:30amOrca Bio
11:30am – 11:45amAldevron
11:45am – 12:00pmInVitria

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 – 1:30pmAffyImmune Therapeutics
1:30pm – 1:45pmAngiocrine Bioscience
1:45pm – 2:00pmArtisan Bio
2:00pm – 2:15pmCatamaran Bio

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

2:15pm – 2:30pmBlueRock Therapeutics
2:30pm – 2:45pmTaysha Gene Therapies
2:45pm – 3:00pmPassage Bio
3:00pm – 3:15pmAthersys
3:15pm – 3:30pmGinkgo Bioworks

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

2:15pm – 2:30pmRocket Pharmaceuticals
2:30pm – 2:45pmLuminary Therapeutics
2:45pm – 3:00pmSenti Biosciences
3:00pm – 3:15pmShoreline Biosciences
3:15pm – 3:30pmDiscGenics

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 – 5:15pmAmicus Therapeutics
5:15pm – 5:30pmHomology Medicines
5:30pm – 5:45pmAVROBIO
5:45pm – 6:00pmOrchard Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

5:00pm – 5:15pmForge Biologics
5:15pm – 5:30pmLife Edit Therapeutics
5:30pm – 5:45pmTreeFrog Therapeutics
5:45pm – 6:00pmGenEdit

6:00pm | PROGRAM AND PARTNERING CLOSES

6:30pm – 9:30pm | NETWORKING BASH

Sponsored by Biopharma Excellence by PharmaLex, Cognate BioServices and L7 Informatics

Mesa21-Agenda-Day2

6:45am – 9:15am | REGISTRATION AND BREAKFAST

Sponsored by Triangle Insights Group

7:15am – 8:45am | CONCURRENT WORKSHOPS

  • INDUSTRIALIZATION OF CELL THERAPY MANUFACTURING – LESSONS LEARNED FROM BIOLOGICS WORKSHOP
  • DEMOCRATIZATION OF TRANSFORMATIVE CELL & GENE THERAPIES WORKSHOP
  • OVERCOMING CHALLENGES TO GENE THERAPY MANUFACTURING THROUGH STANDARDIZATION & AUTOMATION WORKSHOP

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 – 9:30amCook MyoSite
9:30am – 9:45amBioLife Solutions
9:45am – 10:00amTerumo BCT
10:00am – 10:15amBone Therapeutics
Mesa21-Agenda-Day2

6:45am – 9:15am | REGISTRATION AND BREAKFAST

Sponsored by Triangle Insights Group

7:15am – 8:45am | CONCURRENT WORKSHOPS

  • INDUSTRIALIZATION OF CELL THERAPY MANUFACTURING – LESSONS LEARNED FROM BIOLOGICS WORKSHOP
  • DEMOCRATIZATION OF TRANSFORMATIVE CELL & GENE THERAPIES WORKSHOP
  • OVERCOMING CHALLENGES TO GENE THERAPY MANUFACTURING THROUGH STANDARDIZATION & AUTOMATION WORKSHOP

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 – 9:30amCook MyoSite
9:30am – 9:45amBioLife Solutions
9:45am – 10:00amTerumo BCT
10:00am – 10:15amBone 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 – 11:00amAkron Biotech
11:00am – 11:15amMEDIPOST 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 – 1:30pmCTI 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 – 2:15pmSangamo Therapeutics
2:15pm – 2:30pmuniQure
2:30pm – 2:45pmSigilon Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

2:00pm – 2:15pmVirica Biotech
2:15pm – 2:30pmEnzyvant Therapeutics
2:30pm – 2:45pmSentien 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 – 3:30pmImmusoft
3:30pm – 3:45pmCapricor Therapeutics
3:45pm – 4:00pmPBS Biotech
4:00pm – 4:15pmL7 Informatics

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

4:15pm – 4:30pmAllogene Therapeutics
4:30pm – 4:45pmMission Bio
4:45pm – 5:00pmSynthego
5:00pm – 5:15pmMaxCyte
5:15pm – 5:30pmVor Biopharma
5:30pm – 5:45pmPoseida Therapeutics
5:45pm – 6:00pmKuur Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

4:15pm – 4:30pmSensorion
4:30pm – 4:45pmLikarda
4:45pm – 5:00pmTenaya Therapeutics
5:00pm – 5:15pmVineti
5:15pm – 5:30pmRejuvenate Bio
5:30pm – 5:45pmAvery 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 – 2:15pmSangamo Therapeutics
2:15pm – 2:30pmuniQure
2:30pm – 2:45pmSigilon Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

2:00pm – 2:15pmVirica Biotech
2:15pm – 2:30pmEnzyvant Therapeutics
2:30pm – 2:45pmSentien 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 – 3:30pmImmusoft
3:30pm – 3:45pmCapricor Therapeutics
3:45pm – 4:00pmPBS Biotech
4:00pm – 4:15pmL7 Informatics

COMPANY PRESENTATIONS
{AVROBIO Ballroom}

4:15pm – 4:30pmAllogene Therapeutics
4:30pm – 4:45pmMission Bio
4:45pm – 5:00pmSynthego
5:00pm – 5:15pmMaxCyte
5:15pm – 5:30pmVor Biopharma
5:30pm – 5:45pmPoseida Therapeutics
5:45pm – 6:00pmKuur Therapeutics

COMPANY PRESENTATIONS
{BlueRock Therapeutics Ballroom}

4:15pm – 4:30pmSensorion
4:30pm – 4:45pmLikarda
4:45pm – 5:00pmTenaya Therapeutics
5:00pm – 5:15pmVineti
5:15pm – 5:30pmRejuvenate Bio
5:30pm – 5:45pmAvery Therapeutics

6:00pm | PROGRAM AND PARTNERING CLOSES

6:45am – 9:15am | REGISTRATION AND BREAKFAST

Sponsored by CPC

7:15am – 8:45am | MORNING WORKSHOP

  • DELIVERING THE INNOVATION OF GENE THERAPY TO PATIENTS THROUGH STRATEGIC COLLABORATIONS 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 – 9:30amMammoth Biosciences
9:30am – 9:45amStemson 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 – 10:00amEndsulin
10:00am – 10:15amArbor Biotechnologies
10:15am – 10:30amViaCyte
10:30am – 10:45amCastle 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 – 11:15amAspect Biosystems
11:15am – 11:30amTrailhead 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

  • DELIVERING THE INNOVATION OF GENE THERAPY TO PATIENTS THROUGH STRATEGIC COLLABORATIONS 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 – 9:30amMammoth Biosciences
9:30am – 9:45amStemson 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 – 10:00amEndsulin
10:00am – 10:15amArbor Biotechnologies
10:15am – 10:30amViaCyte
10:30am – 10:45amCastle 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 – 11:15amAspect Biosystems
11:15am – 11:30amTrailhead Biosystems

12:00pm – 1:30pm | LUNCH

Sponsored by VGXI

1:30pm | CONFERENCE CLOSES

By using this website you agree to accept our Privacy Policy and Terms & Conditions