The 2023 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.
ARM Board of Directors Meeting & ARM Committee Meetings (By Invitation Only)
6:45am – 9:15am Registration & Breakfast
7:45am – 8:45am Concurrent Workshops
9:00am – 6:00pm General Session, Panel Discussions, Company Presentations, Partnering
6:30pm – 9:30pm Networking Bash
6:45am – 9:15am Registration & Breakfast
7:45am – 8:45am Concurrent Workshops
9:00am – 6:00pm General Session, Panel Discussions, Company Presentations, Partnering
6:45am – 9:15am Registration & Breakfast
7:45am – 8:45am Concurrent Workshops
9:00am – 12:00pm General Session, Panel Discussions, Company Presentations, Partnering
12:00pm – 1:30pm Lunch
1:30pm Conference Closes
12:00am - 11:59pm Virtual Partnering Meetings
12:00am - 11:59pm Virtual Partnering Meetings
ON-DEMAND VIRTUAL CONTENT
Available Beginning Tuesday, October 11, 2022
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)
A DIALOG BETWEEN REGULATORY AGENCIES AND INDUSTRIES AMONG ASIAN COUNTRIES
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 APACRM, an annual conference, 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.
The latest APACRM focused on dialog with regulators based on survey results from three working groups: non-clinical, quality assessments, and environmental impact assessment settings for cell and gene therapy products (CGTs). Through panel discussions comprised of major Asian regulatory agencies, regulation differences and their underlying rationales have been more clearly defined among the participating countries and regions. The hope is that an environment of harmonization is cultivated by sharing experiences regarding products under development, and opinions on regulatory guidelines. Moreover, more proactive efforts on regulatory innovation (e.g., the necessity of new guidelines) regarding regenerative medicinal products can assist with regulatory harmonization and optimization across Asia. This would enable the rapid delivery of CGTs to more patients, thus achieving market maximization of CGTs throughout Asia. This session will explore prospects and challenges to promote harmonization and optimization of 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)
Speakers:
Ryan Chang, Advisor, Acepodia
Hiroshi Karasawa, Ph.D., Scientist, Cell Therapy Research Laboratories, Daiichi Sankyo
Masaaki Miyano, Manager, CMC Regulatory Affairs Department, Chugai Pharmaceutical
Masahiko Yasumoto, Executive Officer, Deputy Division Director, R&D Division, NanoCarrier
PRE-RECORDED VIRTUAL COMPANY PRESENTATIONS
Pre-recorded virtual company presentations will be available on-demand within the platform for all delegates to view starting October 11, 2022.
Advarra | ATMPS | Avectas | AVROBIO | Cellusion | Century Therapeutics | DiscGenics | Gamida Cell | Kytopen | Myrtelle | Qurgen | Talaris
IN-PERSON PROGRAM AND LIVESTREAM
7:00am – 9:00am | REGISTRATION AND BREAKFAST
Sponsored by KBI Biopharma
7:45am – 8:45am | CONCURRENT WORKSHOPS
7:45am - 8:45am | Oxford Biomedica Ballroom
Sponsored by Precision ADVANCE
WHAT’S NEXT FOR CELL AND GENE THERAPIES IN 2023 AND BEYOND WORKSHOP
This workshop will feature leading executives in the ATMP space who will discuss a number of topics including future technologies, the current investment environment and what innovators can do to mitigate growing market pressures. The session will also touch upon resolving manufacturing and regulatory bottlenecks as the sector moves towards commercialization, as well as the evolution of reimbursement payment models.
Chair:
Anshul Mangal, President, Precision ADVANCE and Project Farma
Speakers:
Stewart Abbot, Ph.D., Chief Scientific Officer, Turnstone Biologics
Andrew Knudten, Chief Operating Officer, Jaguar Gene Therapy
Laura Lande-Diner, Ph.D., Chief Business Officer, Satellite Bio
Robert Peters, Ph.D., Chief Scientific Officer, Ensoma
Deb Phippard, Ph.D., Chief Scientific Officer, Precision for Medicine
7:45am – 8:45am | Aviara Salon A
Sponsored by Gamma Biosciences
HOW INDUSTRY 4.0 PRINCIPLES WILL HELP DRIVE THE ADOPTION OF ADVANCED CELL THERAPIES WORKSHOP
The industry has come this far highly dependent on technologies and lessons learned from the biologics industry. How are those habits helping and hurting the progress of cell and gene therapy manufacturing? How can emerging concepts of continuous biomanufacturing, process analytical technologies (PAT), and automation help us rethink manufacturing strategies today, and for years to come? Learn from a panel of experts and industry veterans what the future will look like in advanced therapy manufacturing.
Chair:
Phil Vanek, Ph.D., Chief Technology Officer and Partner, Gamma Biosciences
Speakers:
Andy Lin, Ph.D., Vice President and Global Head of Individualized and Cell Therapy Development, Genentech
Damian Marshall, Ph.D., Vice President, Analytical Development, Resolution Therapeutics
Michael May, Ph.D., President and CEO, CCRM
Alison Moore, Ph.D., Chief Technical Officer, Allogene Therapeutics
7:45am – 8:45am | Aviara Salon B
Sponsored by Advanced Regenerative Manufacturing Institute (ARMI)
SECRETS OF SCALE: HOW THE CONVERGENCE OF SYSTEMS BIOLOGY, ENGINEERING AND NON-CONVENTIONAL REGULATORY APPROACHES ACCELERATE CELL-BASED THERAPIES TO MARKET WORKSHOP
This workshop will discuss breaking down historical barriers to scalable, consistent, cost-effective manufacturing of cell-based therapies by exploiting the convergence of systems biology, advanced process technology development, and non-conventional regulatory approaches.
Chair:
Richard McFarland, M.D., Ph.D., Chief Regulatory Officer, Advanced Regenerative Manufacturing Institute (ARMI)
Speakers:
Carter Cliff, Co-Founder and CEO, Vascugen
Esther Latres, Ph.D., Assistant Vice President of Research, JDRF International
Lloyd Rose, Ph.D., Product Manager, Warfighter Expeditionary Medicine and Treatment, U.S. Army Medical Materiel Development Activity; Program Manager, BioFabUSA
9:00am | GENERAL SESSION AND PARTNERING OPENS
WELCOME REMARKS
{UBC Ballroom}
9:00am – 9:15am
Speakers:
Tim Hunt, CEO, Alliance for Regenerative Medicine (ARM)
Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM)
9:15am – 10:15am | CONCURRENT TRACKS
PLENARY SESSION: CURRENT TRENDS AND CHALLENGES SURROUNDING GENE THERAPY COMMERCIALIZATION
{UBC Ballroom}
9:15am – 10:15am
With more and more gene therapies expected to be approved by health authorities in the coming years, we must now turn to the next major hurdle in bringing these transformative medicines to patients: commercialization. Beyond addressing the elephant in the room – price – companies must navigate early access, accelerated approval requirements, novel go-to-market challenges, and considerations of global equity, while managing the intersection of highly engaged and often demanding patient communities, physicians, payors, and governments. Hear from how the emerging leaders in this space are coping with these challenges to deliver on the promises of advanced therapies.
Chair:
Dave Lennon, Ph.D., CEO, Satellite Bio
Speakers:
Debbie Drane, Senior Vice President, Global Commercial Development and TA Strategy, CSL Behring
Christine Fox, President, Novartis Gene Therapies
Matthew Klein, M.D., Chief Operating Officer, PTC Therapeutics
Thomas Klima, Chief Commercial Officer, bluebird bio
Leslie Meltzer, Ph.D., Chief Medical Officer, Orchard Therapeutics
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
9:15am – 10:15am
| 9:15am – 9:30am | Aspen Neuroscience |
| 9:30am – 9:45am | BioCentriq |
| 9:45am – 10:00am | Encoded Therapeutics |
| 10:00am – 10:15am | BlueRock Therapeutics |
10:15am – 10:45am | MORNING BREAK
Sponsored by Accelerated Biosciences, Exothera, Minaris Regenerative Medicine and Triangle Insights Group
10:45am – 12:15pm | CONCURRENT TRACKS
EVALUATING THE QUALITY OF TISSUE ENGINEERED PRODUCTS
{UBC Ballroom}
10:45am – 11:15am
Tissue engineered products, which are complex living structures, have unique considerations with respect to characterization and release testing. This expert panel will discuss challenges associated with potency assays, stability testing, and scaling manufacturing.
Chair:
Jane Lebkowski, Ph.D., President, Regenerative Patch Technologies
Speakers:
Geoff Hamilton, Co-Founder and CEO, Stemson Therapeutics
Kevin Healy, Ph.D., Senior Vice President Regulatory Affairs, Pharmacovigilance, and Quality, Enzyvant Therapeutics
Eric Roos, Chief Business Development Officer, Aspect Biosystems
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
10:45am – 11:15am
| 10:45am – 11:00am | Cabaletta Bio |
| 11:00am – 11:15am | ExCellThera |
COMPANY PRESENTATIONS
{UBC Ballroom}
11:15am – 12:15pm
| 11:15am – 11:30am | Intellia Therapeutics |
| 11:30am – 11:45am | 4D Molecular Therapeutics |
| 11:45am – 12:00pm | Tenaya Therapeutics |
| 12:00pm – 12:15pm | PTC Therapeutics |
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
11:15am – 12:15pm
| 11:15am – 11:30am | Kyverna Therapeutics |
| 11:30am – 11:45am | Quell Therapeutics |
| 11:45am – 12:00pm | Aegle Therapeutics |
| 12:00pm – 12:15pm | Ensoma |
12:15pm – 1:15pm | LUNCH
Sponsored by Notch Therapeutics
1:15pm – 3:30pm | CONCURRENT TRACKS
THE CURRENT STATE OF INVESTMENT IN CELL AND GENE THERAPY
{UBC Ballroom}
1:15pm – 2:15pm
What is the state of investment in cell and gene therapy globally and the outlook for the rest of 2022 and beyond? This panel will discuss the availability and deployment of capital, from the perspective of venture capital, mergers and acquisitions, and contract manufacturing.
Chair:
Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer Inc.
Speakers:
Matthew Durdy, Chief Executive, Cell and Gene Therapy Catapult
Amy DuRoss, President, Cell and Gene Therapies, CSafe Global; Senior Advisor, Frazier Healthcare Partners
Albert Hwang, M.D., Managing Director, Morgan Stanley
Dmitry Kuzmin, Ph.D., Managing Partner, 4BIO Capital
Becky Pferdehirt, Ph.D., Investment Partner, Andreessen Horowitz
Josh Schimmer, M.D., Senior Managing Director, Evercore
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
1:15pm – 2:15pm
| 1:15pm – 1:30pm | CTI |
| 1:30pm – 1:45pm | Mission Bio |
| 1:45pm – 2:00pm | Sigilon Therapeutics |
| 2:00pm – 2:15pm | Ossium Health |
COMPANY PRESENTATIONS
{UBC Ballroom}
2:15pm – 3:30pm
| 2:15pm – 2:30pm | uniQure |
| 2:30pm – 2:45pm | Rocket Pharmaceuticals |
| 2:45pm – 3:00pm | Helixmith |
| 3:00pm – 3:15pm | Sernova |
| 3:15pm – 3:30pm | Homology Medicines |
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
2:15pm – 3:30pm
| 2:15pm – 2:30pm | Immusoft |
| 2:30pm – 2:45pm | Arbor Biotechnologies |
| 2:45pm – 3:00pm | Rejuvenate Bio |
| 3:00pm – 3:15pm | WuXi Advanced Therapies |
| 3:15pm – 3:30pm | Viralgen Vector Core |
3:30pm – 4:00pm | AFTERNOON BREAK
Accelerated Biosciences, Exothera, Minaris Regenerative Medicine and Triangle Insights Group
4:00pm – 6:00pm | CONCURRENT TRACKS
GENE EDITING ACCELERATES: THE FIRST GENERATION NEARS APPROVAL WHILE NEW APPROACHES PROGRESS THROUGH THE CLINIC
{UBC Ballroom}
4:00pm – 5:00pm
The first-ever approval of a CRISPR gene-editing therapy could be less than a year away, while base editing has already entered the clinic and the first in-vivo CRISPR approaches progress in clinical trials. Panelists will discuss the milestones the sector has recently reached in gene-editing, and forecast the breakthroughs of the near- and longer-term future.
Chair:
Tim Farries, Ph.D., Principal Consultant, Senior Director, Biopharma Excellence
Speakers:
Fred Chereau, President and CEO, LogicBio
Albert Seymour, Ph.D., President and CEO, Homology Medicines
Mark Shearman, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine
Devyn Smith, Ph.D., CEO, Arbor Biotechnologies
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
4:00pm – 5:00pm
| 4:00pm – 4:15pm | Athersys |
| 4:15pm – 4:30pm | AffyImmune Therapeutics |
| 4:30pm – 4:45pm | Bellicum Pharmaceuticals |
| 4:45pm – 5:00pm | Amplo Biotechnology |
COMPANY PRESENTATIONS
{UBC Ballroom}
5:00pm – 6:00pm
| 5:00pm – 5:15pm | Healios K.K. |
| 5:15pm – 5:30pm | Precision BioSciences |
| 5:30pm – 5:45pm | Senti Biosciences |
| 5:45pm – 6:00pm | Arcellx |
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
5:00pm – 6:00pm
| 5:00pm – 5:15pm | NexImmune |
| 5:15pm – 5:30pm | In8Bio |
| 5:30pm – 5:45pm | Gradalis |
| 5:45pm – 6:00pm | Lykan Bioscience |
6:00pm | PROGRAM AND PARTNERING CLOSES
6:30pm – 9:30pm | NETWORKING BASH
Sponsored by BioPharma Excellence by PharmaLex, QbD Vision and ScaleReady
7:00am – 9:00am | REGISTRATION AND BREAKFAST
Sponsored by Pall Corporation
7:45am – 8:45am | CONCURRENT WORKSHOPS
7:45am – 8:45am | Oxford Biomedica Ballroom
Sponsored by Thermo Fisher Scientific
APPROACHES TO IMPLEMENTING AUTOMATION IN CELL THERAPY MANUFACTURING WORKSHOP
The need for standardization and automation are critical drivers of innovation in cell therapy manufacturing. Implementation of an automated, closed workflow minimizes open processes and manual touchpoints, increases reproducibility, and improves traceability; this has the potential to reduce manufacturing failures and lower COGS thereby facilitating increased patient access. In this workshop, we sit down with industry experts to discuss various approaches to cGMP manufacturing of gene-modified cell therapies at industrial scale.
Chair:
Betty Woo, Ph.D., Vice President and General Manager, Cell and Gene Therapy, Thermo Fisher Scientific
Speakers:
Dmitry Fradkin, Director, Automation and Systems Development, Kite Pharma
Fred Parietti, Ph.D., Co-Founder and CEO, Multiply Labs
Edwin Stone, Ph.D., Head of Future Automation, TTP
7:45am – 8:45am | Aviara Salon A
Sponsored by IQVIA
THE FUTURE OF CDMO NETWORKS IN CELL AND GENE THERAPY WORKSHOP
This workshop will feature leading executives who will discuss a number of topics surrounding CDMO networks including bottlenecks in personnel capabilities and facilities along with preclinical pipeline driving capacity and workforce requirements. The session will also touch upon downstream requirements such as clinical sites capabilities, data systems, access and diversity, and regulatory bandwidth.
Chair:
Amritha Jaishankar, Ph.D., Executive Director, IQVIA Cell and Gene Therapy Translating Center
Speakers:
Eytan Abraham, Ph.D., Vice President, Business Head of Cell, Gene and Nucleic Acids Franchises, Resilience
Abla Creasey, Ph.D., Vice President, Therapeutics Development, California Institute for Regenerative Medicine (CIRM)
Haro Hartounian, Ph.D., President and CEO, BioCentriq
Alan Moore, Chief Strategy Officer, Center for Breakthrough Medicines
Nicholas Ostrout, Ph.D., Senior Advisor, Cell and Gene Therapy Corporate Strategy, Charles River Laboratories
7:45am – 8:45am | Aviara Salon B
ACCELERATING CGTX DISCOVERY AND DEVELOPMENT THROUGH USE OF ADVANCED PHENOTYPIC MODELS AND MACHINE LEARNING TECHNOLOGIES WORKSHOP
This panel will explore the ways in which next-generation engineered in-vitro models can be combined with innovations in machine learning applications to drive more rational development of complex cell and gene therapy products. Leveraging advances in in-vitro model development (ex. engineered iPSC-derived tissues) as a starting point for discovery, AI/ML platforms can help make connections and correlations between cause and effect in CGTx development at a rapid pace, ultimately driving towards faster, cheaper, and more accurate therapeutic outputs. This model is already established in the small molecule space, and this panel will explore how this will translate to the CGTx industry.
Chair:
Adam Roose, Vice President, Cell and Gene Commercial Strategy, Adjuvant Partners
Speakers:
Isaac Bentwich, M.D., Founder and CEO, Quris Technologies
Robert Deans, Ph.D., Chief Scientific Officer, Synthego
Keith James, Ph.D., Senior Vice President, Drug Discovery, insitro
Emmanouil Metzakopian, Ph.D., Vice President, Research and Development, bit.bio
9:00am | GENERAL SESSION AND PARTNERING OPENS
9:00am – 10:15am | CONCURRENT TRACKS
UPDATE ON ARM’S GROW INTERNSHIP PROGRAM
{UBC Ballroom}
9:00am – 9:15am
Speaker:
Tim Hunt, CEO, Alliance for Regenerative Medicine (ARM)
PLENARY SESSION: CELL THERAPY COMMERCIALIZATION TODAY AND PREPARING FOR THE FUTURE
{UBC Ballroom}
9:15am – 10:15am
Over the past several years, the cell therapy industry has seen tremendous progress in bringing new cell therapies to market. Facilitating the delivery of these life-saving therapies and enabling them to address a broader range of indications for larger patient populations requires collaboration and innovation. Panelists will discuss advances in the industry, key challenges and innovative approaches to commercialization, and the value of strategic partnerships that will help drive the future of cell therapies.
Chair:
Amy Butler, Ph.D., President, Biosciences Division, Thermo Fisher Scientific
Speakers:
Diana Brainard, M.D., CEO, AlloVir
Friedrich Graf Finckenstein, M.D., Chief Medical Officer, Iovance Biotherapeutics
Cintia Piccina, Pharm.D., Chief Commercial Officer, Adaptimmune
Paul Staid, Vice President and Head of Global Supply Chain, Cell Therapy Operations, Bristol-Myers Squibb
Pascal Touchon, President and CEO, Atara Biotherapeutics
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
9:15am – 10:15am
| 9:15am – 9:30am | Forge Biologics |
| 9:30am – 9:45am | ImStem Biotechnology |
| 9:45am – 10:00am | Aldevron |
| 10:00am – 10:15am | Marken |
10:15am – 10:45am | MORNING BREAK
Sponsored by Accelerated Biosciences, Exothera, Minaris Regenerative Medicine and Triangle Insights Group
10:45am – 12:15pm | CONCURRENT TRACKS
KEYNOTE SPEAKER: TAKING CELL AND GENE THERAPY TO THE NEXT LEVEL
{UBC Ballroom}
10:45am – 11:15am
Speaker:
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
10:45am – 11:15am
| 10:45am – 11:00am | Carisma Therapeutics |
| 11:00am – 11:15am | Luminary Therapeutics |
OPPORTUNITIES FOR REGULATORY CONVERGENCE IN CELL AND GENE THERAPY
{UBC Ballroom}
11:15am – 12:15pm
Disparate requirements from one country to the next, from pre-clinical tests to manufacturing, make it difficult for developers to efficiently ensure patients across borders have access to therapies, particularly to rare disease patients whose numbers in any one geography may be very small. Representatives from the US Food and Drug Administration, European Medicines Agency, and Japanese Pharmaceuticals and Medical Devices Agency will discuss areas where regulators can and are cooperate on common standards, and what next steps are being envisioned to improve equitable access to critical CGT medicines.
Chair:
Jessie Hanrahan, Ph.D., Chief Regulatory Officer, AavantiBio
Speakers:
Patrick Celis, Ph.D., Lead Scientific Officer, ATMP Office, European Medicines Agency (EMA)
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
11:15am – 12:15pm
| 11:15am – 11:30am | Notch Therapeutics |
| 11:30am – 11:45am | Prescient Therapeutics |
| 11:45am – 12:00pm | Curate Biosciences |
| 12:00pm – 12:15pm | Capricor Therapeutics |
12:15pm – 1:15pm | LUNCH
Sponsored by Curate Biosciences
1:15pm – 2:45pm | CONCURRENT TRACKS
LESSONS LEARNED FROM THE BLUEBIRD BIO ICER ASSESSMENTS
{UBC Ballroom}
1:15pm – 1:45pm
In 2022, ICER completed its evaluation of bluebird bio’s gene therapy Zynteglo, which is approved in the U.S. for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell transfusions. This session will focus on sharing bluebird bio’s engagement with ICER throughout the assessment time period and leading up to the public meeting in July of 2022. In addition, the speakers will discuss bluebird bio’s approach to evidence generation in advance of the ICER assessment.
Chair:
Brent Rice, Senior Vice President and Chief Commercial Officer, Autolus Therapeutics
Speaker:
Clark Paramore, Vice President, Value Demonstration, bluebird bio
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
1:15pm – 1:45pm
| 1:15pm – 1:30pm | Ray Therapeutics |
| 1:30pm – 1:45pm | Coave Therapeutics |
COMPANY PRESENTATIONS
{UBC Ballroom}
1:45pm – 2:45pm
| 1:45pm – 2:00pm | REGENXBIO |
| 2:00pm – 2:15pm | Ocugen |
| 2:15pm – 2:30pm | Lineage Cell Therapeutics |
| 2:30pm – 2:45pm | AlloVir |
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
1:45pm – 2:45pm
| 1:45pm – 2:00pm | Antion Biosciences |
| 2:00pm – 2:15pm | Charles River |
| 2:15pm – 2:30pm | SparingVision |
| 2:30pm – 2:45pm | Miltenyi Biomedicine |
2:45pm – 3:15pm | AFTERNOON BREAK
Sponsored by Accelerated Biosciences, Exothera, Minaris Regenerative Medicine and Triangle Insights Group
3:15pm – 6:00pm | CONCURRENT TRACKS
NEW BUSINESS MODELS FOR MANUFACTURING INVESTMENT
{UBC Ballroom}
3:15pm – 4:15pm
Effective investment in manufacturing is key to a successful development program. Alternatives to the classic approaches of build in house vs. use a CMO are emerging. The panel will explore different models for increasing manufacturing capacity and how these can assist with pain points such as talent acquisition and retention.
Chair:
Susan Nichols, Chief Business Officer, ViroCell Biologics
Speakers:
Eric Blair, Chief Commercial Officer, Andelyn Biosciences
Frédéric Desdouits, Ph.D., CEO, TreeFrog Therapeutics
Behzad Mahdavi, Ph.D., Senior Vice President, Biopharma Manufacturing and Life Sciences Tools, Ginkgo Bioworks
Daniel Smith, Ph.D., Executive Director, Global Cell and Gene Therapy Portfolio, Charles River Laboratories
Arthur Tzianabos, Ph.D., Venture Partner, 5AM Ventures; Chair of the Board, Homology Medicines
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
3:15pm – 4:15pm
| 3:15pm – 3:30pm | Albumedix |
| 3:30pm – 3:45pm | Miromatrix |
| 3:45pm – 4:00pm | PBS Biotech |
| 4:00pm – 4:15pm | VERIGRAFT |
COMPANY PRESENTATIONS
{UBC Ballroom}
4:15pm – 6:00pm
| 4:15pm – 4:30pm | Orchard Therapeutics |
| 4:30pm – 4:45pm | Atara Biotherapeutics |
| 4:45pm – 5:00pm | Autolus |
| 5:00pm – 5:15pm | Poseida Therapeutics |
| 5:15pm – 5:30pm | Vor Bio |
| 5:30pm – 5:45pm | Enzyvant |
| 5:45pm – 6:00pm | ImmuneBridge |
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
4:15pm – 6:00pm
| 4:15pm – 4:30pm | Cardinal Health |
| 4:30pm – 4:45pm | ElevateBio |
| 4:45pm – 5:00pm | MaxCyte |
| 5:00pm – 5:15pm | Medpace |
| 5:15pm – 5:30pm | Terumo BCT |
| 5:30pm – 5:45pm | Cryoport Systems |
| 5:45pm – 6:00pm | Teknova |
6:00pm | PROGRAM AND PARTNERING CLOSES
7:00am – 9:00am | REGISTRATION AND BREAKFAST
Sponsored by Gift of Life
7:45am – 8:45am | CONCURRENT WORKSHOPS
7:45am – 8:45am | Oxford Biomedica Ballroom
Sponsored by SmartLabs
ADDRESSING THE PRODUCTION NEEDS OF CELL AND GENE THERAPEUTICS: NEW STRATEGIES FOR A CHANGING WORLD WORKSHOP
Join this session to hear experts debate the following industry challenges:
Chair:
Brian Taylor, Head of Biopharma Solutions, SmartLabs
Speakers:
Wolfram Carius, Pharm.D., Executive Vice President, Cell and Gene Therapy, Bayer
Justin Skoble, Ph.D., Vice President of Technical Operations, Caribou Biosciences
Sarah Yuan, Ph.D., Chief Technical Operations Officer, Sigilon Therapeutics
7:45am – 8:45am | Aviara Salon A
Sponsored by L7 Informatics
DIGITAL TRANSFORMATION: INTEGRATING AUTOMATED DATA MANAGEMENT SYSTEMS TO ADVANCE CGT MANUFACTURING AND IMPROVE PROCESS CONTROL WORKSHOP
This workshop discussion will explore ways in which advancements in data capture, data interrogation and analytics will improve advanced therapies product manufacture. Innovations in data processing will help to optimize manufacturing and process development efforts, decreasing associated time and costs. The session will explore how some of the most advanced technology companies are refining and implementing these processes.
Chair:
Trent Carrier, Ph.D., Chief Operating Officer, L7 Informatics
Speakers:
Sammy Datwani, Ph.D., Senior Vice President, Development, Synthego
Delara Motlagh, Ph.D., General Manager, Cell Therapy Technologies, Terumo BCT
Yan Zhang, Ph.D., CEO, Mission Bio
9:00am | GENERAL SESSION AND PARTNERING OPENS
9:00am – 10:45am | CONCURRENT TRACKS
ARM CLOSING STATEMENTS
{UBC Ballroom}
9:00am – 9:15am
Speaker:
Tim Hunt, CEO, Alliance for Regenerative Medicine (ARM)
SPOTLIGHT SESSION: THE PATIENT PERSPECTIVE ON CELL AND GENE THERAPY
{UBC Ballroom}
9:15am – 9:45am
Chair:
Craig Martin, CEO, Global Genes
Speaker:
Aaron Owens, Recessive Dystrophic Epidermolysis Bullosa Patient and Advocate
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
9:15am – 9:45am
| 9:15am – 9:30am | Cynata Therapeutics |
| 9:30am – 9:45am | GENETHON |
NEW DEVELOPMENTS AND ADVANCEMENTS IN THE WORLD OF PSCS
{UBC Ballroom}
9:45am – 10:45am
Chair:
Alberto Santagostino, Senior Vice President, Head of Cell and Gene Technologies, Lonza
Speakers:
Melissa Carpenter, Ph.D., Chief Scientific Officer, Regenerative Medicine, ElevateBio
Seth Ettenberg, Ph.D., President and CEO, BlueRock Therapeutics
Osvaldo Flores, Ph.D., CEO, Century Therapeutics
Xiaokui Zhang, Ph.D., Chief Scientific Officer, Aspen Neuroscience
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
9:45am – 10:45am
| 9:45am – 10:00am | MEDIPOST America |
| 10:00am – 10:15am | Xintela |
| 10:15am – 10:30am | TrakCel |
| 10:30am – 10:45am | Cellares |
10:45am – 11:00am | MORNING BREAK
Sponsored by Accelerated Biosciences, Exothera, Minaris Regenerative Medicine and Triangle Insights Group
11:00am – 12:00pm | CONCURRENT TRACKS
THE FUTURE OF GENE DELIVERY: IN SEARCH OF GAME-CHANGING IN VIVO APPLICATIONS
{UBC Ballroom}
11:00am – 12:00pm
Chair:
Faraz Ali, CEO, Tenaya Therapeutics
Speakers:
Adrian Bot, M.D., Ph.D., Chief Scientific Officer and Executive Vice President of R&D, Capstan Therapeutics
Gary Fortin, Ph.D., Chief Operating Officer, Shape Therapeutics
Philip Johnson, M.D., CEO, Interius Biotherapeutics
Yvette Leung, Head of Corporate Development, Dyno Therapeutics
Emile Nuwaysir, Ph.D., President and CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM)
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
11:00am – 12:00pm
| 11:00am – 11:15am | Versiti |
| 11:15am – 11:30am | Capsida Biotherapeutics |
| 11:30am – 11:45am | Excision BioTherapeutics |
| 11:45am – 12:00pm | VectorY |
12:00pm – 1:00pm | LUNCH
Sponsored by Theragent
1:00pm | CONFERENCE CLOSES
ON-DEMAND VIRTUAL CONTENT
Available Beginning Tuesday, October 11, 2022
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)
A DIALOG BETWEEN REGULATORY AGENCIES AND INDUSTRIES AMONG ASIAN COUNTRIES
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 APACRM, an annual conference, 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.
The latest APACRM focused on dialog with regulators based on survey results from three working groups: non-clinical, quality assessments, and environmental impact assessment settings for cell and gene therapy products (CGTs). Through panel discussions comprised of major Asian regulatory agencies, regulation differences and their underlying rationales have been more clearly defined among the participating countries and regions. The hope is that an environment of harmonization is cultivated by sharing experiences regarding products under development, and opinions on regulatory guidelines. Moreover, more proactive efforts on regulatory innovation (e.g., the necessity of new guidelines) regarding regenerative medicinal products can assist with regulatory harmonization and optimization across Asia. This would enable the rapid delivery of CGTs to more patients, thus achieving market maximization of CGTs throughout Asia. This session will explore prospects and challenges to promote harmonization and optimization of 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)
Speakers:
Ryan Chang, Advisor, Acepodia
Hiroshi Karasawa, Ph.D., Scientist, Cell Therapy Research Laboratories, Daiichi Sankyo
Masaaki Miyano, Manager, CMC Regulatory Affairs Department, Chugai Pharmaceutical
Masahiko Yasumoto, Executive Officer, Deputy Division Director, R&D Division, NanoCarrier
PRE-RECORDED VIRTUAL COMPANY PRESENTATIONS
Pre-recorded virtual company presentations will be available on-demand within the platform for all delegates to view starting October 11, 2022.
Advarra | ATMPS | Avectas | AVROBIO | Cellusion | Century Therapeutics | DiscGenics | Gamida Cell | Kytopen | Myrtelle | Qurgen | Talaris
IN-PERSON PROGRAM AND LIVESTREAM
7:00am – 9:00am | REGISTRATION AND BREAKFAST
Sponsored by KBI Biopharma
7:45am – 8:45am | CONCURRENT WORKSHOPS
7:45am - 8:45am | Oxford Biomedica Ballroom
Sponsored by Precision ADVANCE
WHAT’S NEXT FOR CELL AND GENE THERAPIES IN 2023 AND BEYOND WORKSHOP
This workshop will feature leading executives in the ATMP space who will discuss a number of topics including future technologies, the current investment environment and what innovators can do to mitigate growing market pressures. The session will also touch upon resolving manufacturing and regulatory bottlenecks as the sector moves towards commercialization, as well as the evolution of reimbursement payment models.
Chair:
Anshul Mangal, President, Precision ADVANCE and Project Farma
Speakers:
Stewart Abbot, Ph.D., Chief Scientific Officer, Turnstone Biologics
Andrew Knudten, Chief Operating Officer, Jaguar Gene Therapy
Laura Lande-Diner, Ph.D., Chief Business Officer, Satellite Bio
Robert Peters, Ph.D., Chief Scientific Officer, Ensoma
Deb Phippard, Ph.D., Chief Scientific Officer, Precision for Medicine
7:45am – 8:45am | Aviara Salon A
Sponsored by Gamma Biosciences
HOW INDUSTRY 4.0 PRINCIPLES WILL HELP DRIVE THE ADOPTION OF ADVANCED CELL THERAPIES WORKSHOP
The industry has come this far highly dependent on technologies and lessons learned from the biologics industry. How are those habits helping and hurting the progress of cell and gene therapy manufacturing? How can emerging concepts of continuous biomanufacturing, process analytical technologies (PAT), and automation help us rethink manufacturing strategies today, and for years to come? Learn from a panel of experts and industry veterans what the future will look like in advanced therapy manufacturing.
Chair:
Phil Vanek, Ph.D., Chief Technology Officer and Partner, Gamma Biosciences
Speakers:
Andy Lin, Ph.D., Vice President and Global Head of Individualized and Cell Therapy Development, Genentech
Damian Marshall, Ph.D., Vice President, Analytical Development, Resolution Therapeutics
Michael May, Ph.D., President and CEO, CCRM
Alison Moore, Ph.D., Chief Technical Officer, Allogene Therapeutics
7:45am – 8:45am | Aviara Salon B
Sponsored by Advanced Regenerative Manufacturing Institute (ARMI)
SECRETS OF SCALE: HOW THE CONVERGENCE OF SYSTEMS BIOLOGY, ENGINEERING AND NON-CONVENTIONAL REGULATORY APPROACHES ACCELERATE CELL-BASED THERAPIES TO MARKET WORKSHOP
This workshop will discuss breaking down historical barriers to scalable, consistent, cost-effective manufacturing of cell-based therapies by exploiting the convergence of systems biology, advanced process technology development, and non-conventional regulatory approaches.
Chair:
Richard McFarland, M.D., Ph.D., Chief Regulatory Officer, Advanced Regenerative Manufacturing Institute (ARMI)
Speakers:
Carter Cliff, Co-Founder and CEO, Vascugen
Esther Latres, Ph.D., Assistant Vice President of Research, JDRF International
Lloyd Rose, Ph.D., Product Manager, Warfighter Expeditionary Medicine and Treatment, U.S. Army Medical Materiel Development Activity; Program Manager, BioFabUSA
9:00am | GENERAL SESSION AND PARTNERING OPENS
WELCOME REMARKS
{UBC Ballroom}
9:00am – 9:15am
Speakers:
Tim Hunt, CEO, Alliance for Regenerative Medicine (ARM)
Emile Nuwaysir, Ph.D., CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM)
9:15am – 10:15am | CONCURRENT TRACKS
PLENARY SESSION: CURRENT TRENDS AND CHALLENGES SURROUNDING GENE THERAPY COMMERCIALIZATION
{UBC Ballroom}
9:15am – 10:15am
With more and more gene therapies expected to be approved by health authorities in the coming years, we must now turn to the next major hurdle in bringing these transformative medicines to patients: commercialization. Beyond addressing the elephant in the room – price – companies must navigate early access, accelerated approval requirements, novel go-to-market challenges, and considerations of global equity, while managing the intersection of highly engaged and often demanding patient communities, physicians, payors, and governments. Hear from how the emerging leaders in this space are coping with these challenges to deliver on the promises of advanced therapies.
Chair:
Dave Lennon, Ph.D., CEO, Satellite Bio
Speakers:
Debbie Drane, Senior Vice President, Global Commercial Development and TA Strategy, CSL Behring
Christine Fox, President, Novartis Gene Therapies
Matthew Klein, M.D., Chief Operating Officer, PTC Therapeutics
Thomas Klima, Chief Commercial Officer, bluebird bio
Leslie Meltzer, Ph.D., Chief Medical Officer, Orchard Therapeutics
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
9:15am – 10:15am
| 9:15am – 9:30am | Aspen Neuroscience |
| 9:30am – 9:45am | BioCentriq |
| 9:45am – 10:00am | Encoded Therapeutics |
| 10:00am – 10:15am | BlueRock Therapeutics |
10:15am – 10:45am | MORNING BREAK
Sponsored by Accelerated Biosciences, Exothera, Minaris Regenerative Medicine and Triangle Insights Group
10:45am – 12:15pm | CONCURRENT TRACKS
EVALUATING THE QUALITY OF TISSUE ENGINEERED PRODUCTS
{UBC Ballroom}
10:45am – 11:15am
Tissue engineered products, which are complex living structures, have unique considerations with respect to characterization and release testing. This expert panel will discuss challenges associated with potency assays, stability testing, and scaling manufacturing.
Chair:
Jane Lebkowski, Ph.D., President, Regenerative Patch Technologies
Speakers:
Geoff Hamilton, Co-Founder and CEO, Stemson Therapeutics
Kevin Healy, Ph.D., Senior Vice President Regulatory Affairs, Pharmacovigilance, and Quality, Enzyvant Therapeutics
Eric Roos, Chief Business Development Officer, Aspect Biosystems
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
10:45am – 11:15am
| 10:45am – 11:00am | Cabaletta Bio |
| 11:00am – 11:15am | ExCellThera |
COMPANY PRESENTATIONS
{UBC Ballroom}
11:15am – 12:15pm
| 11:15am – 11:30am | Intellia Therapeutics |
| 11:30am – 11:45am | 4D Molecular Therapeutics |
| 11:45am – 12:00pm | Tenaya Therapeutics |
| 12:00pm – 12:15pm | PTC Therapeutics |
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
11:15am – 12:15pm
| 11:15am – 11:30am | Kyverna Therapeutics |
| 11:30am – 11:45am | Quell Therapeutics |
| 11:45am – 12:00pm | Aegle Therapeutics |
| 12:00pm – 12:15pm | Ensoma |
12:15pm – 1:15pm | LUNCH
Sponsored by Notch Therapeutics
1:15pm – 3:30pm | CONCURRENT TRACKS
THE CURRENT STATE OF INVESTMENT IN CELL AND GENE THERAPY
{UBC Ballroom}
1:15pm – 2:15pm
What is the state of investment in cell and gene therapy globally and the outlook for the rest of 2022 and beyond? This panel will discuss the availability and deployment of capital, from the perspective of venture capital, mergers and acquisitions, and contract manufacturing.
Chair:
Bob Smith, Senior Vice President, Global Gene Therapy Business, Pfizer Inc.
Speakers:
Matthew Durdy, Chief Executive, Cell and Gene Therapy Catapult
Amy DuRoss, President, Cell and Gene Therapies, CSafe Global; Senior Advisor, Frazier Healthcare Partners
Albert Hwang, M.D., Managing Director, Morgan Stanley
Dmitry Kuzmin, Ph.D., Managing Partner, 4BIO Capital
Becky Pferdehirt, Ph.D., Investment Partner, Andreessen Horowitz
Josh Schimmer, M.D., Senior Managing Director, Evercore
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
1:15pm – 2:15pm
| 1:15pm – 1:30pm | CTI |
| 1:30pm – 1:45pm | Mission Bio |
| 1:45pm – 2:00pm | Sigilon Therapeutics |
| 2:00pm – 2:15pm | Ossium Health |
COMPANY PRESENTATIONS
{UBC Ballroom}
2:15pm – 3:30pm
| 2:15pm – 2:30pm | uniQure |
| 2:30pm – 2:45pm | Rocket Pharmaceuticals |
| 2:45pm – 3:00pm | Helixmith |
| 3:00pm – 3:15pm | Sernova |
| 3:15pm – 3:30pm | Homology Medicines |
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
2:15pm – 3:30pm
| 2:15pm – 2:30pm | Immusoft |
| 2:30pm – 2:45pm | Arbor Biotechnologies |
| 2:45pm – 3:00pm | Rejuvenate Bio |
| 3:00pm – 3:15pm | WuXi Advanced Therapies |
| 3:15pm – 3:30pm | Viralgen Vector Core |
3:30pm – 4:00pm | AFTERNOON BREAK
Accelerated Biosciences, Exothera, Minaris Regenerative Medicine and Triangle Insights Group
4:00pm – 6:00pm | CONCURRENT TRACKS
GENE EDITING ACCELERATES: THE FIRST GENERATION NEARS APPROVAL WHILE NEW APPROACHES PROGRESS THROUGH THE CLINIC
{UBC Ballroom}
4:00pm – 5:00pm
The first-ever approval of a CRISPR gene-editing therapy could be less than a year away, while base editing has already entered the clinic and the first in-vivo CRISPR approaches progress in clinical trials. Panelists will discuss the milestones the sector has recently reached in gene-editing, and forecast the breakthroughs of the near- and longer-term future.
Chair:
Tim Farries, Ph.D., Principal Consultant, Senior Director, Biopharma Excellence
Speakers:
Fred Chereau, President and CEO, LogicBio
Albert Seymour, Ph.D., President and CEO, Homology Medicines
Mark Shearman, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine
Devyn Smith, Ph.D., CEO, Arbor Biotechnologies
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
4:00pm – 5:00pm
| 4:00pm – 4:15pm | Athersys |
| 4:15pm – 4:30pm | AffyImmune Therapeutics |
| 4:30pm – 4:45pm | Bellicum Pharmaceuticals |
| 4:45pm – 5:00pm | Amplo Biotechnology |
COMPANY PRESENTATIONS
{UBC Ballroom}
5:00pm – 6:00pm
| 5:00pm – 5:15pm | Healios K.K. |
| 5:15pm – 5:30pm | Precision BioSciences |
| 5:30pm – 5:45pm | Senti Biosciences |
| 5:45pm – 6:00pm | Arcellx |
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
5:00pm – 6:00pm
| 5:00pm – 5:15pm | NexImmune |
| 5:15pm – 5:30pm | In8Bio |
| 5:30pm – 5:45pm | Gradalis |
| 5:45pm – 6:00pm | Lykan Bioscience |
6:00pm | PROGRAM AND PARTNERING CLOSES
6:30pm – 9:30pm | NETWORKING BASH
Sponsored by BioPharma Excellence by PharmaLex, QbD Vision and ScaleReady
7:00am – 9:00am | REGISTRATION AND BREAKFAST
Sponsored by Pall Corporation
7:45am – 8:45am | CONCURRENT WORKSHOPS
7:45am – 8:45am | Oxford Biomedica Ballroom
Sponsored by Thermo Fisher Scientific
APPROACHES TO IMPLEMENTING AUTOMATION IN CELL THERAPY MANUFACTURING WORKSHOP
The need for standardization and automation are critical drivers of innovation in cell therapy manufacturing. Implementation of an automated, closed workflow minimizes open processes and manual touchpoints, increases reproducibility, and improves traceability; this has the potential to reduce manufacturing failures and lower COGS thereby facilitating increased patient access. In this workshop, we sit down with industry experts to discuss various approaches to cGMP manufacturing of gene-modified cell therapies at industrial scale.
Chair:
Betty Woo, Ph.D., Vice President and General Manager, Cell and Gene Therapy, Thermo Fisher Scientific
Speakers:
Dmitry Fradkin, Director, Automation and Systems Development, Kite Pharma
Fred Parietti, Ph.D., Co-Founder and CEO, Multiply Labs
Edwin Stone, Ph.D., Head of Future Automation, TTP
7:45am – 8:45am | Aviara Salon A
Sponsored by IQVIA
THE FUTURE OF CDMO NETWORKS IN CELL AND GENE THERAPY WORKSHOP
This workshop will feature leading executives who will discuss a number of topics surrounding CDMO networks including bottlenecks in personnel capabilities and facilities along with preclinical pipeline driving capacity and workforce requirements. The session will also touch upon downstream requirements such as clinical sites capabilities, data systems, access and diversity, and regulatory bandwidth.
Chair:
Amritha Jaishankar, Ph.D., Executive Director, IQVIA Cell and Gene Therapy Translating Center
Speakers:
Eytan Abraham, Ph.D., Vice President, Business Head of Cell, Gene and Nucleic Acids Franchises, Resilience
Abla Creasey, Ph.D., Vice President, Therapeutics Development, California Institute for Regenerative Medicine (CIRM)
Haro Hartounian, Ph.D., President and CEO, BioCentriq
Alan Moore, Chief Strategy Officer, Center for Breakthrough Medicines
Nicholas Ostrout, Ph.D., Senior Advisor, Cell and Gene Therapy Corporate Strategy, Charles River Laboratories
7:45am – 8:45am | Aviara Salon B
ACCELERATING CGTX DISCOVERY AND DEVELOPMENT THROUGH USE OF ADVANCED PHENOTYPIC MODELS AND MACHINE LEARNING TECHNOLOGIES WORKSHOP
This panel will explore the ways in which next-generation engineered in-vitro models can be combined with innovations in machine learning applications to drive more rational development of complex cell and gene therapy products. Leveraging advances in in-vitro model development (ex. engineered iPSC-derived tissues) as a starting point for discovery, AI/ML platforms can help make connections and correlations between cause and effect in CGTx development at a rapid pace, ultimately driving towards faster, cheaper, and more accurate therapeutic outputs. This model is already established in the small molecule space, and this panel will explore how this will translate to the CGTx industry.
Chair:
Adam Roose, Vice President, Cell and Gene Commercial Strategy, Adjuvant Partners
Speakers:
Isaac Bentwich, M.D., Founder and CEO, Quris Technologies
Robert Deans, Ph.D., Chief Scientific Officer, Synthego
Keith James, Ph.D., Senior Vice President, Drug Discovery, insitro
Emmanouil Metzakopian, Ph.D., Vice President, Research and Development, bit.bio
9:00am | GENERAL SESSION AND PARTNERING OPENS
9:00am – 10:15am | CONCURRENT TRACKS
UPDATE ON ARM’S GROW INTERNSHIP PROGRAM
{UBC Ballroom}
9:00am – 9:15am
Speaker:
Tim Hunt, CEO, Alliance for Regenerative Medicine (ARM)
PLENARY SESSION: CELL THERAPY COMMERCIALIZATION TODAY AND PREPARING FOR THE FUTURE
{UBC Ballroom}
9:15am – 10:15am
Over the past several years, the cell therapy industry has seen tremendous progress in bringing new cell therapies to market. Facilitating the delivery of these life-saving therapies and enabling them to address a broader range of indications for larger patient populations requires collaboration and innovation. Panelists will discuss advances in the industry, key challenges and innovative approaches to commercialization, and the value of strategic partnerships that will help drive the future of cell therapies.
Chair:
Amy Butler, Ph.D., President, Biosciences Division, Thermo Fisher Scientific
Speakers:
Diana Brainard, M.D., CEO, AlloVir
Friedrich Graf Finckenstein, M.D., Chief Medical Officer, Iovance Biotherapeutics
Cintia Piccina, Pharm.D., Chief Commercial Officer, Adaptimmune
Paul Staid, Vice President and Head of Global Supply Chain, Cell Therapy Operations, Bristol-Myers Squibb
Pascal Touchon, President and CEO, Atara Biotherapeutics
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
9:15am – 10:15am
| 9:15am – 9:30am | Forge Biologics |
| 9:30am – 9:45am | ImStem Biotechnology |
| 9:45am – 10:00am | Aldevron |
| 10:00am – 10:15am | Marken |
10:15am – 10:45am | MORNING BREAK
Sponsored by Accelerated Biosciences, Exothera, Minaris Regenerative Medicine and Triangle Insights Group
10:45am – 12:15pm | CONCURRENT TRACKS
KEYNOTE SPEAKER: TAKING CELL AND GENE THERAPY TO THE NEXT LEVEL
{UBC Ballroom}
10:45am – 11:15am
Speaker:
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
10:45am – 11:15am
| 10:45am – 11:00am | Carisma Therapeutics |
| 11:00am – 11:15am | Luminary Therapeutics |
OPPORTUNITIES FOR REGULATORY CONVERGENCE IN CELL AND GENE THERAPY
{UBC Ballroom}
11:15am – 12:15pm
Disparate requirements from one country to the next, from pre-clinical tests to manufacturing, make it difficult for developers to efficiently ensure patients across borders have access to therapies, particularly to rare disease patients whose numbers in any one geography may be very small. Representatives from the US Food and Drug Administration, European Medicines Agency, and Japanese Pharmaceuticals and Medical Devices Agency will discuss areas where regulators can and are cooperate on common standards, and what next steps are being envisioned to improve equitable access to critical CGT medicines.
Chair:
Jessie Hanrahan, Ph.D., Chief Regulatory Officer, AavantiBio
Speakers:
Patrick Celis, Ph.D., Lead Scientific Officer, ATMP Office, European Medicines Agency (EMA)
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
11:15am – 12:15pm
| 11:15am – 11:30am | Notch Therapeutics |
| 11:30am – 11:45am | Prescient Therapeutics |
| 11:45am – 12:00pm | Curate Biosciences |
| 12:00pm – 12:15pm | Capricor Therapeutics |
12:15pm – 1:15pm | LUNCH
Sponsored by Curate Biosciences
1:15pm – 2:45pm | CONCURRENT TRACKS
LESSONS LEARNED FROM THE BLUEBIRD BIO ICER ASSESSMENTS
{UBC Ballroom}
1:15pm – 1:45pm
In 2022, ICER completed its evaluation of bluebird bio’s gene therapy Zynteglo, which is approved in the U.S. for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell transfusions. This session will focus on sharing bluebird bio’s engagement with ICER throughout the assessment time period and leading up to the public meeting in July of 2022. In addition, the speakers will discuss bluebird bio’s approach to evidence generation in advance of the ICER assessment.
Chair:
Brent Rice, Senior Vice President and Chief Commercial Officer, Autolus Therapeutics
Speaker:
Clark Paramore, Vice President, Value Demonstration, bluebird bio
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
1:15pm – 1:45pm
| 1:15pm – 1:30pm | Ray Therapeutics |
| 1:30pm – 1:45pm | Coave Therapeutics |
COMPANY PRESENTATIONS
{UBC Ballroom}
1:45pm – 2:45pm
| 1:45pm – 2:00pm | REGENXBIO |
| 2:00pm – 2:15pm | Ocugen |
| 2:15pm – 2:30pm | Lineage Cell Therapeutics |
| 2:30pm – 2:45pm | AlloVir |
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
1:45pm – 2:45pm
| 1:45pm – 2:00pm | Antion Biosciences |
| 2:00pm – 2:15pm | Charles River |
| 2:15pm – 2:30pm | SparingVision |
| 2:30pm – 2:45pm | Miltenyi Biomedicine |
2:45pm – 3:15pm | AFTERNOON BREAK
Sponsored by Accelerated Biosciences, Exothera, Minaris Regenerative Medicine and Triangle Insights Group
3:15pm – 6:00pm | CONCURRENT TRACKS
NEW BUSINESS MODELS FOR MANUFACTURING INVESTMENT
{UBC Ballroom}
3:15pm – 4:15pm
Effective investment in manufacturing is key to a successful development program. Alternatives to the classic approaches of build in house vs. use a CMO are emerging. The panel will explore different models for increasing manufacturing capacity and how these can assist with pain points such as talent acquisition and retention.
Chair:
Susan Nichols, Chief Business Officer, ViroCell Biologics
Speakers:
Eric Blair, Chief Commercial Officer, Andelyn Biosciences
Frédéric Desdouits, Ph.D., CEO, TreeFrog Therapeutics
Behzad Mahdavi, Ph.D., Senior Vice President, Biopharma Manufacturing and Life Sciences Tools, Ginkgo Bioworks
Daniel Smith, Ph.D., Executive Director, Global Cell and Gene Therapy Portfolio, Charles River Laboratories
Arthur Tzianabos, Ph.D., Venture Partner, 5AM Ventures; Chair of the Board, Homology Medicines
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
3:15pm – 4:15pm
| 3:15pm – 3:30pm | Albumedix |
| 3:30pm – 3:45pm | Miromatrix |
| 3:45pm – 4:00pm | PBS Biotech |
| 4:00pm – 4:15pm | VERIGRAFT |
COMPANY PRESENTATIONS
{UBC Ballroom}
4:15pm – 6:00pm
| 4:15pm – 4:30pm | Orchard Therapeutics |
| 4:30pm – 4:45pm | Atara Biotherapeutics |
| 4:45pm – 5:00pm | Autolus |
| 5:00pm – 5:15pm | Poseida Therapeutics |
| 5:15pm – 5:30pm | Vor Bio |
| 5:30pm – 5:45pm | Enzyvant |
| 5:45pm – 6:00pm | ImmuneBridge |
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
4:15pm – 6:00pm
| 4:15pm – 4:30pm | Cardinal Health |
| 4:30pm – 4:45pm | ElevateBio |
| 4:45pm – 5:00pm | MaxCyte |
| 5:00pm – 5:15pm | Medpace |
| 5:15pm – 5:30pm | Terumo BCT |
| 5:30pm – 5:45pm | Cryoport Systems |
| 5:45pm – 6:00pm | Teknova |
6:00pm | PROGRAM AND PARTNERING CLOSES
7:00am – 9:00am | REGISTRATION AND BREAKFAST
Sponsored by Gift of Life
7:45am – 8:45am | CONCURRENT WORKSHOPS
7:45am – 8:45am | Oxford Biomedica Ballroom
Sponsored by SmartLabs
ADDRESSING THE PRODUCTION NEEDS OF CELL AND GENE THERAPEUTICS: NEW STRATEGIES FOR A CHANGING WORLD WORKSHOP
Join this session to hear experts debate the following industry challenges:
Chair:
Brian Taylor, Head of Biopharma Solutions, SmartLabs
Speakers:
Wolfram Carius, Pharm.D., Executive Vice President, Cell and Gene Therapy, Bayer
Justin Skoble, Ph.D., Vice President of Technical Operations, Caribou Biosciences
Sarah Yuan, Ph.D., Chief Technical Operations Officer, Sigilon Therapeutics
7:45am – 8:45am | Aviara Salon A
Sponsored by L7 Informatics
DIGITAL TRANSFORMATION: INTEGRATING AUTOMATED DATA MANAGEMENT SYSTEMS TO ADVANCE CGT MANUFACTURING AND IMPROVE PROCESS CONTROL WORKSHOP
This workshop discussion will explore ways in which advancements in data capture, data interrogation and analytics will improve advanced therapies product manufacture. Innovations in data processing will help to optimize manufacturing and process development efforts, decreasing associated time and costs. The session will explore how some of the most advanced technology companies are refining and implementing these processes.
Chair:
Trent Carrier, Ph.D., Chief Operating Officer, L7 Informatics
Speakers:
Sammy Datwani, Ph.D., Senior Vice President, Development, Synthego
Delara Motlagh, Ph.D., General Manager, Cell Therapy Technologies, Terumo BCT
Yan Zhang, Ph.D., CEO, Mission Bio
9:00am | GENERAL SESSION AND PARTNERING OPENS
9:00am – 10:45am | CONCURRENT TRACKS
ARM CLOSING STATEMENTS
{UBC Ballroom}
9:00am – 9:15am
Speaker:
Tim Hunt, CEO, Alliance for Regenerative Medicine (ARM)
SPOTLIGHT SESSION: THE PATIENT PERSPECTIVE ON CELL AND GENE THERAPY
{UBC Ballroom}
9:15am – 9:45am
Chair:
Craig Martin, CEO, Global Genes
Speaker:
Aaron Owens, Recessive Dystrophic Epidermolysis Bullosa Patient and Advocate
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
9:15am – 9:45am
| 9:15am – 9:30am | Cynata Therapeutics |
| 9:30am – 9:45am | GENETHON |
NEW DEVELOPMENTS AND ADVANCEMENTS IN THE WORLD OF PSCS
{UBC Ballroom}
9:45am – 10:45am
Chair:
Alberto Santagostino, Senior Vice President, Head of Cell and Gene Technologies, Lonza
Speakers:
Melissa Carpenter, Ph.D., Chief Scientific Officer, Regenerative Medicine, ElevateBio
Seth Ettenberg, Ph.D., President and CEO, BlueRock Therapeutics
Osvaldo Flores, Ph.D., CEO, Century Therapeutics
Xiaokui Zhang, Ph.D., Chief Scientific Officer, Aspen Neuroscience
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
9:45am – 10:45am
| 9:45am – 10:00am | MEDIPOST America |
| 10:00am – 10:15am | Xintela |
| 10:15am – 10:30am | TrakCel |
| 10:30am – 10:45am | Cellares |
10:45am – 11:00am | MORNING BREAK
Sponsored by Accelerated Biosciences, Exothera, Minaris Regenerative Medicine and Triangle Insights Group
11:00am – 12:00pm | CONCURRENT TRACKS
THE FUTURE OF GENE DELIVERY: IN SEARCH OF GAME-CHANGING IN VIVO APPLICATIONS
{UBC Ballroom}
11:00am – 12:00pm
Chair:
Faraz Ali, CEO, Tenaya Therapeutics
Speakers:
Adrian Bot, M.D., Ph.D., Chief Scientific Officer and Executive Vice President of R&D, Capstan Therapeutics
Gary Fortin, Ph.D., Chief Operating Officer, Shape Therapeutics
Philip Johnson, M.D., CEO, Interius Biotherapeutics
Yvette Leung, Head of Corporate Development, Dyno Therapeutics
Emile Nuwaysir, Ph.D., President and CEO, Ensoma; Chairman, Alliance for Regenerative Medicine (ARM)
COMPANY PRESENTATIONS
{Oxford Biomedica Ballroom}
11:00am – 12:00pm
| 11:00am – 11:15am | Versiti |
| 11:15am – 11:30am | Capsida Biotherapeutics |
| 11:30am – 11:45am | Excision BioTherapeutics |
| 11:45am – 12:00pm | VectorY |
12:00pm – 1:00pm | LUNCH
Sponsored by Theragent
1:00pm | CONFERENCE CLOSES
The Cell & Gene Meeting on the Mesa is the sector’s foremost annual conference bringing together senior executives and top decision-makers in the industry to advance cutting-edge research into cures. Tackling the commercialization hurdles facing the cell and gene therapy sector today, this meeting covers a wide range of topics from clinical trial design to alternative payment models to scale-up and supply chain platforms for advanced therapies. The program features expert-led panels, extensive partnering capabilities, exclusive networking opportunities, and 100+ dedicated presentations by the leading publicly traded and privately held companies in the space. Attracting over 2,000 attendees – over 20% of which are C-level executives – this conference enables key partnerships through more than 5,000 one-on-one meetings while highlighting the significant clinical and commercial progress in the field.
October 10-12, 2023
Park Hyatt Aviara
Carlsbad, CA
