Agenda

2021 Agenda Coming Soon!

The 2021 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 available on-demand within 24 hours of the live program time for all registrants to view. 

This year’s meeting will feature sessions and workshops covering a mix of commercialization topics related to the cell and gene therapy sector. Additionally, we will feature over 100 presentations by leading public and private companies within the sector.

Last Year's Agenda

  • Full Agenda
  • Live Streaming
  • On Demand
  • By Category
  • Day 1 - Oct. 12th
  • Day 2 - Oct. 13th
  • Day 3 - Oct. 14th
  • Day 4 - Oct. 15th
  • Day 5 - Oct. 16th

LIVE STREAMING SESSIONS WITH Q&A

PLENARY SESSION: THE REALITIES OF BRINGING GENE THERAPIES TO MARKET

8am – 9am PT / 11am – 12pm ET

Chair:

Jeff Walsh, Former Chief Financial and Strategy Officer and Current Strategic Advisor, bluebird bio

Speakers:

Jeff Ajer, EVP and Chief Commercial Officer, BioMarin
Lisa Deschamps, Chief Business Officer, AveXis, a Novartis company
Jay Newman, Head, U.S. Commercial, Spark Therapeutics
Braden Parker, SVP and General Manager, North America, Orchard Therapeutics

DISRUPTIVE TECHNOLOGIES THAT WILL CHANGE THE CELL THERAPY LANDSCAPE

12pm – 1pm PT / 3pm – 4pm ET
This panel of industry leaders will reflect on the past, present, and future to discuss how foundational technologies, new modes of operation, and changes in the talent pool are rapidly transforming the cell and gene therapy industry from futuristic promise to one of the hottest sectors in biotech.

Chair:

Michael May, Ph.D., President and CEO, CCRM

Speakers:

Dan Shoemaker, Ph.D., Chief Scientific Officer, Fate Therapeutics
Helen Tayton-Martin, Ph.D., Co-Founder and Chief Business Officer, Adaptimmune
James Trager, Ph.D., Chief Scientific Officer, Nkarta Therapeutics
Stefan Wildt, Ph.D., Head of Pharmaceutical Sciences and Translational Engine, Cell Therapies, Takeda

DURING BREAKS IN LIVE PROGRAMMING
Participate in 1×1 Partnering, Visit Virtual Exhibit Booths, View On-Demand Content

ON-DEMAND CONTENT
Available Beginning Monday, October 12, 2020

120+ COMPANY PRESENTATIONS
View the Full List

ACCELERATING BIOTHERAPEUTIC DEVELOPMENT AND PRODUCTION FOR INCREASED MARKET IMPACT OF LIFE SAVING THERAPEUTICS

Sponsored by Thermo Fisher Scientific
This panel of experts will share their insights and experience on some of today’s most pressing development challenges. The session will dive into key topics such as integrated vs. modular manufacturing approaches, ensuring security of supply, proactive approaches to regulatory compliance, and ultimately, the panel’s vision for the future.

Chair:

Susan D’Costa, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific

Speakers:

Alex Fotopoulos, Chief Technical Officer, Passage Bio

Michael Mercaldi, Ph.D., Senior Director, Downstream Process Development, Homology Medicines

Nripen Singh, Ph.D., Director, Biopharmaceutical Development, Voyager Therapeutics

ACCEPTANCE AND UPTAKE OF CELL AND GENE THERAPIES: LESSONS LEARNED AND FUTURE FOCUS

Sponsored by Evidera PPD
The first wave of transformative cell and gene therapies have launched into a healthcare landscape that arguably was not built with them in mind. Hurdles range from basic understanding to value demonstration, reimbursement and access. This session will debate lessons learned from the initial vanguard of cell and gene therapies, including building the right evidence, navigating the provider-side, achieving value-based payment, and ensuring patient uptake. Discussion will cover unanticipated challenges and critical success factors for future cell and gene therapy success.

Chair:

Eric Faulkner, VP, Precision and Transformative Medicine, Evidera; Executive Director, Genomics Biotech and Emerging Technology Institute, NAMCP

Speakers:

Shannon Baumann, VP, Access, Value and Evidence Strategy, bluebird bio

Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics

Tay Salimullah, VP, Global Head of Patient Access, AveXis, a Novartis company

ALTERNATIVE IPO STRATEGIES FOR CELL AND GENE THERAPY COMPANIES
Sponsored by Chardan
This session will center on special purpose acquisition companies (SPACs), a space heating up within the biotech world. As of August 2020, 10 biotech-focused SPACs have been successful at raising capital with many others coming down the pike. The purpose of the panel is to understand why all these biotech-focused firms are getting involved in the space, what cell and gene therapy companies need to know about this alternative to a traditional IPO if approached, and what seasoned executives of public companies should know about potentially sponsoring their own biotech focused SPAC.

Chair:

Gbola Amusa, M.D., Partner, Director of Research and Head of Healthcare Research, Chardan

Speakers:

Jonas Grossman, President, Chardan

Yvan-Claude Pierre, Partner, Cooley

Stephanie Sirota, Partner and Chief Business Officer, RTW Investments

CURRENT HOT TOPICS AND EMERGING TRENDS IN GENE THERAPY
Sponsored by Pfizer
This panel will focus on emerging gene therapy scientific breakthroughs and challenges as well as notable clinical development advancements and results. Discussion will also touch upon gene therapy manufacturing challenges and the gene therapy financing and investor landscape.

Chair:

Bob Smith, SVP, Global Gene Therapy Business, Pfizer Inc.

Speakers:

Katherine High, M.D., Visiting Professor, Rockefeller University; Co-Founder, Spark Therapeutics

Natalie Holles, President and CEO, Audentes, an Astellas company

Ken Mills, President and CEO, REGENXBIO

DOING BUSINESS IN JAPAN – AN EVOLVING MARKET: THE LATEST RISE OF COMMERCIALIZATION AND PROMOTING HARMONIZATION ON REGENERATIVE MEDICINE WITH ASIA
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)

Opening Remarks

Masayuki (Max) Nomura, Ph.D., Chair, International Affairs Committee, Forum for Innovative Regenerative Medicine (FIRM)

Update on Regulatory Aspects of Gene Therapy Products

Akira Sakurai, Ph.D., Deputy Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)

Points to Consider on the Quality of Raw Materials for the Manufacture of Cell-based Therapeutic Products

Yoji Sato, Ph.D., Head, Division of Cell-Based Therapeutic Products, National Institute of Health Sciences, Japan

Regulation in Asia, U.S., and Europe on Starting Cells for Cellular Therapy Products and Risk-based Approaches

Toshimitsu Tanaka, Member, Regulatory Harmonization Committee, Forum for Innovative Regenerative Medicine (FIRM)

mRNA PROCESS DEVELOPMENT AND MANUFACTURING ON THE ROAD TO THE CLINIC
Sponsored by Aldevron
Messenger RNA offers a promising new approach to address emerging pathogens and unmet medical needs. Shorter lead times and greater simplicity in manufacturing enable faster responses that will transform medicine. This panel will discuss the state of the art with an emphasis on CMC, manufacturing, and associated regulatory issues.

Chair:

Nate Spangler, Ph.D., Director of Innovation and Strategy, Aldevron

Speakers:

Frank DeRosa, Ph.D., Chief Technology Officer, Translate Bio

Andrew Geall, Ph.D., Chief Scientific Officer, Precision NanoSystems

Caitlyn Harvey, CMC Strategy and Project Management, Myeloid Therapeutics

Jeffrey Ulmer, Ph.D., mRNA Therapy and Vaccine Expert

NOVEL CLINICAL TRIAL DESIGNS: MORE EFFICIENT AND EFFECTIVE TESTS OF REGENERATIVE MEDICINES DURING THE PANDEMIC AND BEYOND
Sponsored by IQVIA
This panel of industry, advocacy, and academic leaders will discuss challenges faced and innovative solutions explored, in addressing the unique requirements of clinical studies in cell and gene therapy. Participants will present their first-hand experience with various novel trial designs/elements including remote endpoints/telemedicine, virtual trials, hybrid/decentralized trials, platform trials, and external comparators. Cell and gene therapy trials already incur a unique set of challenges, and COVID-19 has dramatically transformed the clinical trial landscape, highlighting and accelerating the need for new ways of thinking that are more relevant now, but which will continue to be increasingly employed going forward.

Chair:

David Cameron, Global Head of Novel Trial Design, IQVIA

Speakers:

Hearn Jay Cho, M.D., Ph.D., Chief Medical Officer, Multiple Myeloma Research Foundation

Shaji Kumar, M.D., Professor of Medicine, Division of Hematology, Mayo Clinic

Sabrina Paganoni, M.D., Ph.D., Assistant Professor, Harvard Medical School; Physician Scientist, Healey Center for ALS, Massachusetts General Hospital and Spaulding Rehabilitation Hospital

Aiman Shalabi, Pharm.D., VP R&D Cell and Gene Therapies, GSK

SAFETY AND EFFICACY CONSIDERATIONS FOR CELL AND GENE THERAPIES: FROM MANUFACTURING THROUGH DISTRIBUTION
Sponsored by Thermo Fisher Scientific
This panel discussion will cover a number of safety and efficacy factors for advanced therapies including acceptable impurity levels, dosing considerations, the impact of platforms on the potency of products, and maintaining integrity of the material from the manufacturing floor to the clinic.

Chair:

Susan D’Costa, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific

Speakers:

Justin Horvath, Director of External Manufacturing, REGENXBIO

Anne Kantardjieff, Ph.D., Senior Director, External Vector Manufacturing, bluebird bio

LIVE STREAMING SESSIONS WITH Q&A

PLENARY SESSION: THE EVOLUTION OF COMMERCIAL CELL THERAPY

8am – 9am PT / 11am – 12pm ET

Chair:

Ann Lee, Ph.D., SVP and Head of Cell Therapy Development and Operations, Bristol-Myers Squibb

Speakers:

Marco Gottardis, Ph.D., VP, Oncology Innovation, Janssen R&D
Alberto Santagostino, SVP, Head of Cell and Gene Technologies, Lonza Pharma & Biotech
Kristin Yarema, Ph.D., Chief Commercial Officer, Atara Biotherapeutics

CELL AND GENE THERAPIES FOR CHRONIC CONDITIONS

12pm – 1pm PT / 3pm – 4pm ET

Cell and gene therapies represent hope for millions of patients with chronic diseases — not just the possibility of better management, but, in some cases, the promise of a durable cure. This emerging field faces many hurdles, however, including effective delivery of therapies to patients, unique manufacturing challenges, educating regulators on the field, and ensuring market access and acceptance of value-based reimbursement policies. This panel brings together the leaders of five top companies in the sector to explore and discuss these challenges.

Chair:

Devyn Smith, Ph.D., Chief Operations Officer and Head of Strategy, Sigilon Therapeutics

Speakers:

Pavan Cheruvu, M.D., CEO, Axovant Gene Therapies
Jane Lebkowski, Ph.D., President, Regenerative Patch Technologies
Emile Nuwaysir, Ph.D., President and CEO, BlueRock Therapeutics
Philip Toleikis, Ph.D., President and CEO, Sernova

DURING BREAKS IN LIVE PROGRAMMING
Participate in 1×1 Partnering, Visit Virtual Exhibit Booths, View On-Demand Content

ON-DEMAND CONTENT
Available Beginning Tuesday, October 13, 2020

EUROPEAN COMMERCIALIZATION CHALLENGES AND OUTLOOK

With 10 advanced therapy medicinal products (ATMPs) currently marketed in Europe and four withdrawn, the sector’s commercial landscape is at a turning point in Europe. So far, healthcare systems and developers have been successful in opening the way to the cell and gene therapy revolution. Treatment centers are managing patients, payers are exploring innovative ways to cover the cost for transformative therapies, and developers are inventing new ways of doing business. The next step will be to consolidate all of these successes and make them available for a significantly larger number of patients. On this panel, European business leaders will share their expectations and discuss how they are preparing to overcome these challenges.

Chair:

Christopher Vann, SVP and Chief Operating Officer, Autolus

Speakers:

Jacqueline Barry, Ph.D., Chief Clinical Officer, Cell & Gene Therapy Catapult

Nicola Heffron, SVP, Head of European Operations, bluebird bio

Emanuele Ostuni, Ph.D., Head of Europe Cell and Gene Therapy, Novartis Oncology

GMP MANUFACTURING OF CELL AND GENE THERAPIES – A 2020 PERSPECTIVE

This session will include a discussion about issues related to moving from development through to GMP manufacturing of cell and gene therapies including challenges, scalability, standards, and issues that have been raised in light of COVID-19.

Chair:

Kelvin Lee, Ph.D., Institute Director, National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)

Speakers:

Delara Motlagh, Ph.D., VP, Cell Therapy Technologies, Terumo BCT

Claudia Zylberberg, Ph.D., CEO, Akron Biotech

STATE OF PLAY IN THE VECTOR ARMS RACE: PERSPECTIVES ON THE FUTURE OF GENE DELIVERY

Join the CEOs of three leading gene therapy companies as they discuss the relative merits of AAV, lentiviral, and non-viral delivery technology – and share their views on how these platforms will help shape the future of gene therapy. Expect a lively, insightful, and thought-provoking discussion. Both Prevail Therapeutics and AVROBIO are in the clinic and have publicly shared patient data while Generation Bio is preclinical with a novel non-viral platform.

Chair:

Amber Tong, Editor, Endpoints News

Speakers:

Asa Abeliovich, M.D., Ph.D., Founder and CEO, Prevail Therapeutics

Geoff MacKay, President and CEO, AVROBIO

Geoff McDonough, M.D., President and CEO, Generation Bio

VITALITY OF THE RANGE OF INVESTING: EARLY-STAGE SEED INVESTING TO PUBLIC INVESTING
This discussion between three leaders in biotech/pharma investing will delve into the vitality of early stage seed funding to mid stage to public investing in established companies, and how these stages of the investment spectrum interact. The panel will also discuss the importance of alternative investment structures such as venture philanthropy to promote therapeutic pipelines.

Chair:

Jason Rhodes, Partner, Atlas Venture

Speakers:

Rajiv Kaul, Portfolio Manager, Fidelity Investments

Debra Miller, Founder and CEO, CureDuchenne; CEO, CureDuchenne Ventures

LIVE STREAMING SESSIONS WITH Q&A

FEATURED FIRESIDE CHAT: FDA PERSPECTIVE

8am – 9am PT / 11am – 12pm ET

Chair:

Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)

Speakers:

Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)

THE LASTING COVID-19 IMPACT ON CELL AND GENE THERAPY

12pm – 1pm PT / 3pm – 4pm ET

Chair:

Anthony Davies, Ph.D., Founder and CEO, Dark Horse Consulting

Speakers:

Wilson W. Bryan, M.D., Director, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)
Amy DuRoss, Co-Founder and CEO, Vineti
Curran Simpson, Chief Operations and Technology Officer, REGENXBIO
Timothy Schroeder, Founder and CEO, CTI Clinical Trial & Consulting

DURING BREAKS IN LIVE PROGRAMMING
Participate in 1×1 Partnering, Visit Virtual Exhibit Booths, View On-Demand Content

ON-DEMAND CONTENT
Available Beginning Wednesday, October 14, 2020

ACHIEVING DIVERSITY IN CLINICAL TRIALS

While obtaining racial diversity in clinical trials is critical to ensuring that the patients enrolled are representative of the population that will receive treatment, many clinical trials fail to achieve sufficient representation. The ARM Action for Equality (AFE) Taskforce has convened this panel to discuss the barriers, lessons learned, and opportunities to increase representation in clinical trials evaluating products for cell and gene therapy.

Chair:

Faraz Ali, CEO, Tenaya Therapeutics

Speakers:

Rear Admiral Richardae Araojo, Pharm.D., Associate Commissioner for Minority Health; Director, Office of Minority Health and Health Equity, U.S. Food and Drug Administration (FDA)

Erica Prowisor, Global Head of Patient Recruitment and Retention, IQVIA

Dara Richardson-Heron, M.D., Chief Patient Officer, Pfizer Inc.

BESPOKE THERAPIES FOR ULTRA-RARE DISEASE

This session will explore the National Institutes of Health (NIH)’s 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:

Yael Weiss, M.D., Ph.D., VP, Business Development, Ultragenyx

Speakers:

Philip J. Brooks, Ph.D., Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)

Barry Byrne, M.D., Ph.D., Professor and Associate Chair of Pediatrics; Director of the Powell Gene Therapy Center, University of Florida

Joseph Menetski, Ph.D., Associate VP of Research Partnerships, Foundation for the National Institutes of Health

Bob Smith, SVP, Global Gene Therapy Business, Pfizer Inc.

BETWEEN TWO VIRTUAL FERNS: BUILDING A SUCCESSFUL REGENERATIVE MEDICINE COMPANY

Please join us for a conversation about the key considerations for building a successful regenerative medicine company from the perspectives of an operating executive and an investor.

Speakers:

Matthew Patterson, Strategic Advisor, Audentes Therapeutics, An Astellas Company

Kush Parmar, M.D., Ph.D., Managing Partner, 5AM Ventures

THE EUROPEAN REGULATORY ENVIRONMENT FOR ATMPS – SHOULD WE EXPECT MORE OR LESS REGULATION?

This session will discuss the recent initiatives and plans that will affect the regulatory environment for advanced therapy medicinal products (ATMPs) in Europe, such as the EC Pharmaceutical Strategy, the forthcoming implementation of the new Clinical Trial Regulation, GMO requirements for gene therapies, and the European Regulatory Network Strategy to 2025. The impact of these developments on patient access to ATMPs, on the ATMP sector in Europe, and on regulatory bodies will be addressed.

Chair:

Miguel Forte, CEO, Bone Therapeutics

Speakers:

Karolina Hanslik, Senior Project Manager, EURORDIS

Ana Hidalgo-Simon, M.D., Ph.D., Head of Advanced Therapies, European Medicines Agency (EMA)

Rocio Salvador Roldan, Policy Officer, Unit B4- Medical Products, Directorate-General for Health and Food Safety (DG SANTE), European Commission

Martina Schüssler-Lenz, M.D., Chair, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA); Senior Clinical Assessor for ATMPs, Paul-Ehrlich-Institut

LIVE STREAMING SESSIONS WITH Q&A

FEATURED FIRESIDE CHAT: EMA PERSPECTIVE

8am – 9am PT / 11am – 12pm ET

Chair:

Paolo Morgese, Director, EU Market Access and Member Relations, Alliance for Regenerative Medicine (ARM)

Speakers:

Guido Rasi, M.D., Director General, European Medicines Agency (EMA)

THE EMERGENCE OF GENE EDITED CARs AND TCRs

12pm – 1pm PT / 3pm – 4pm ET

Gene editing is a powerful tool used to improve the performance of a patient’s immune cells, and to facilitate the use of allogeneic adoptive cell therapies. This panel will discuss this rapidly developing area and the implications for the future of the treatment of cancer.

Chair:

Charlie Albright, Ph.D., EVP and Chief Scientific Officer, Editas Medicine

Speakers:

Jason Fontenot, Ph.D., SVP, Cell Therapy and Head of Research, Sangamo Therapeutics
Andrew Schiermeier, Ph.D., EVP and Chief Operating Officer, Intellia Therapeutics

DURING BREAKS IN LIVE PROGRAMMING
Participate in 1×1 Partnering, Visit Virtual Exhibit Booths, View On-Demand Content

ON-DEMAND CONTENT
Available Beginning Thursday, October 15, 2020

ARM ACTION FOR EQUALITY TASK FORCE INITIATIVES

The Alliance for Regenerative Medicine (ARM) Action for Equality (AFE) Task Force is exploring what actions ARM and ARM members can take to eliminate racial inequality and address underrepresentation of Black Americans and minorities in the regenerative medicine sector. Our goal is to create a meaningful impact through various initiatives that will provide minorities with more opportunities, exposure, and mentorship in regenerative medicine. The Task Force aims to build a successful program that ARM and ARM Members are energized to pursue together.

Chair:

Emile Nuwaysir, Ph.D., President and CEO, BlueRock Therapeutics; Vice Chairman, Alliance for Regenerative Medicine (ARM)

Speakers:

Usman “Oz” Azam, M.D., President and CEO, Tmunity Therapeutics

Francesca Cook, Senior Director, Pricing and Market Access, REGENXBIO

Anshul Mangal, Founder, CEO and General Counsel, Project Farma

Courtney Utsey, Senior Manager HR Operations, Talent and Culture, Enzyvant

CELL AND GENE THERAPY PORTFOLIO STRATEGIES

This session will focus on how mid- to large-sized biotech and pharma companies develop a portfolio strategy designed to effectively progress cell and gene therapies. The panelists will discuss their respective approaches to building a robust pipeline.

Chair:

Arthur Tzianabos, Ph.D., President and CEO, Homology Medicines

Speakers:

Claudia Mitchell, Ph.D., SVP, Head of Portfolio Strategy, Astellas

Sanjaya Singh, Ph.D., VP and Global Head, Janssen BioTherapeutics, Janssen Research & Development, LLC, Janssen Pharmaceutical Companies of Johnson & Johnson

Anabella Villalobos, Ph.D., SVP, Biotherapeutics and Medicinal Sciences, Biogen

FDA WORKING WITH PATIENT GROUPS

This panel will discuss innovative ways patient groups can work with the FDA throughout the drug development and evaluation process. In this session, panelists will explore the use of real-world evidence, patient experience, and registries as replacements for more traditional methods of data collection.

Chair:

Annie Kennedy, Chief of Policy and Advocacy, Everylife Foundation for Rare Diseases

Speakers:

Ryan Fischer, SVP, Community Engagement, Parent Project Muscular Dystrophy

Monica Shah, M.D., VP and Head, Cell and Gene Therapy Center of Excellence, IQVIA

Kimberly Trant, Executive Director, Head of Patient Advocacy and Engagement, Audentes, an Astellas company

OVERCOMING CHALLENGES WITH THE DEVELOPMENT AND TRANSLATION OF A THERAPEUTIC FROM ACADEMIA TO INDUSTRY

Some of the most cutting-edge and innovative approaches to cell and gene therapy have originated from the academic setting. However, getting these treatments into the clinic can be especially challenging when the institution does not have the experience and infrastructure necessary to conduct preclinical studies, manufacture to GMP standards, and file regulatory submissions with the FDA. This panel of experts will discuss their lessons learned throughout the process and best approaches to moving a cell and gene therapeutic candidate from academia to commercialization.

Chair:

Shawn Roach, Ph.D., Specialist-Regulatory CMC, Halloran Consulting Group

Speakers:

Isabelle Riviere, Ph.D., Director, Cell Therapy and Cell Engineering Facility, Memorial Sloan Kettering Cancer Center

Anthony Ting, Ph.D., VP of Regenerative Medicine and Head of Cardiopulmonary Programs, Athersys

OPEN DAY
Participate in 1×1 Partnering, Visit Virtual Exhibit Booths, View On-Demand Content

LIVE STREAMING SESSIONS WITH Q&A

PLENARY SESSION: THE REALITIES OF BRINGING GENE THERAPIES TO MARKET

8am – 9am PT / 11am – 12pm ET

Chair:

Jeff Walsh, Former Chief Financial and Strategy Officer and Current Strategic Advisor, bluebird bio

Speakers:

Jeff Ajer, EVP and Chief Commercial Officer, BioMarin
Lisa Deschamps, Chief Business Officer, AveXis, a Novartis company
Jay Newman, Head, U.S. Commercial, Spark Therapeutics
Braden Parker, SVP and General Manager, North America, Orchard Therapeutics

DISRUPTIVE TECHNOLOGIES THAT WILL CHANGE THE CELL THERAPY LANDSCAPE

12pm – 1pm PT / 3pm – 4pm ET
This panel of industry leaders will reflect on the past, present, and future to discuss how foundational technologies, new modes of operation, and changes in the talent pool are rapidly transforming the cell and gene therapy industry from futuristic promise to one of the hottest sectors in biotech.

Chair:

Michael May, Ph.D., President and CEO, CCRM

Speakers:

Dan Shoemaker, Ph.D., Chief Scientific Officer, Fate Therapeutics
Helen Tayton-Martin, Ph.D., Co-Founder and Chief Business Officer, Adaptimmune
James Trager, Ph.D., Chief Scientific Officer, Nkarta Therapeutics
Stefan Wildt, Ph.D., Head of Pharmaceutical Sciences and Translational Engine, Cell Therapies, Takeda

LIVE STREAMING SESSIONS WITH Q&A

PLENARY SESSION: THE EVOLUTION OF COMMERCIAL CELL THERAPY

8am – 9am PT / 11am – 12pm ET

Chair:

Ann Lee, Ph.D., SVP and Head of Cell Therapy Development and Operations, Bristol-Myers Squibb

Speakers:

Marco Gottardis, Ph.D., VP, Oncology Innovation, Janssen R&D
Alberto Santagostino, SVP, Head of Cell and Gene Technologies, Lonza Pharma & Biotech
Kristin Yarema, Ph.D., Chief Commercial Officer, Atara Biotherapeutics

CELL AND GENE THERAPIES FOR CHRONIC CONDITIONS

12pm – 1pm PT / 3pm – 4pm ET

Cell and gene therapies represent hope for millions of patients with chronic diseases — not just the possibility of better management, but, in some cases, the promise of a durable cure. This emerging field faces many hurdles, however, including effective delivery of therapies to patients, unique manufacturing challenges, educating regulators on the field, and ensuring market access and acceptance of value-based reimbursement policies. This panel brings together the leaders of five top companies in the sector to explore and discuss these challenges.

Chair:

Devyn Smith, Ph.D., Chief Operations Officer and Head of Strategy, Sigilon Therapeutics

Speakers:

Pavan Cheruvu, M.D., CEO, Axovant Gene Therapies
Jane Lebkowski, Ph.D., President, Regenerative Patch Technologies
Emile Nuwaysir, Ph.D., President and CEO, BlueRock Therapeutics
Philip Toleikis, Ph.D., President and CEO, Sernova

LIVE STREAMING SESSIONS WITH Q&A

FEATURED FIRESIDE CHAT: FDA PERSPECTIVE

8am – 9am PT / 11am – 12pm ET

Chair:

Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)

Speakers:

Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)

THE LASTING COVID-19 IMPACT ON CELL AND GENE THERAPY

12pm – 1pm PT / 3pm – 4pm ET

Chair:

Anthony Davies, Ph.D., Founder and CEO, Dark Horse Consulting

Speakers:

Wilson W. Bryan, M.D., Director, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)
Amy DuRoss, Co-Founder and CEO, Vineti
Curran Simpson, Chief Operations and Technology Officer, REGENXBIO
Timothy Schroeder, Founder and CEO, CTI Clinical Trial & Consulting

LIVE STREAMING SESSIONS WITH Q&A

FEATURED FIRESIDE CHAT: EMA PERSPECTIVE

8am – 9am PT / 11am – 12pm ET

Chair:

Paolo Morgese, Director, EU Market Access and Member Relations, Alliance for Regenerative Medicine (ARM)

Speakers:

Guido Rasi, M.D., Director General, European Medicines Agency (EMA)

THE EMERGENCE OF GENE EDITED CARs AND TCRs

12pm – 1pm PT / 3pm – 4pm ET

Gene editing is a powerful tool used to improve the performance of a patient’s immune cells, and to facilitate the use of allogeneic adoptive cell therapies. This panel will discuss this rapidly developing area and the implications for the future of the treatment of cancer.

Chair:

Charlie Albright, Ph.D., EVP and Chief Scientific Officer, Editas Medicine

Speakers:

Jason Fontenot, Ph.D., SVP, Cell Therapy and Head of Research, Sangamo Therapeutics
Andrew Schiermeier, Ph.D., EVP and Chief Operating Officer, Intellia Therapeutics

ON-DEMAND CONTENT
Available Beginning Monday, October 12, 2020

120+ COMPANY PRESENTATIONS
View the Full List

ACCELERATING BIOTHERAPEUTIC DEVELOPMENT AND PRODUCTION FOR INCREASED MARKET IMPACT OF LIFE SAVING THERAPEUTICS

Sponsored by Thermo Fisher Scientific
This panel of experts will share their insights and experience on some of today’s most pressing development challenges. The session will dive into key topics such as integrated vs. modular manufacturing approaches, ensuring security of supply, proactive approaches to regulatory compliance, and ultimately, the panel’s vision for the future.

Chair:

Susan D’Costa, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific

Speakers:

Alex Fotopoulos, Chief Technical Officer, Passage Bio

Michael Mercaldi, Ph.D., Senior Director, Downstream Process Development, Homology Medicines

Nripen Singh, Ph.D., Director, Biopharmaceutical Development, Voyager Therapeutics

ACCEPTANCE AND UPTAKE OF CELL AND GENE THERAPIES: LESSONS LEARNED AND FUTURE FOCUS

Sponsored by Evidera PPD
The first wave of transformative cell and gene therapies have launched into a healthcare landscape that arguably was not built with them in mind. Hurdles range from basic understanding to value demonstration, reimbursement and access. This session will debate lessons learned from the initial vanguard of cell and gene therapies, including building the right evidence, navigating the provider-side, achieving value-based payment, and ensuring patient uptake. Discussion will cover unanticipated challenges and critical success factors for future cell and gene therapy success.

Chair:

Eric Faulkner, VP, Precision and Transformative Medicine, Evidera; Executive Director, Genomics Biotech and Emerging Technology Institute, NAMCP

Speakers:

Shannon Baumann, VP, Access, Value and Evidence Strategy, bluebird bio

Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics

Tay Salimullah, VP, Global Head of Patient Access, AveXis, a Novartis company

ALTERNATIVE IPO STRATEGIES FOR CELL AND GENE THERAPY COMPANIES
Sponsored by Chardan
This session will center on special purpose acquisition companies (SPACs), a space heating up within the biotech world. As of August 2020, 10 biotech-focused SPACs have been successful at raising capital with many others coming down the pike. The purpose of the panel is to understand why all these biotech-focused firms are getting involved in the space, what cell and gene therapy companies need to know about this alternative to a traditional IPO if approached, and what seasoned executives of public companies should know about potentially sponsoring their own biotech focused SPAC.

Chair:

Gbola Amusa, M.D., Partner, Director of Research and Head of Healthcare Research, Chardan

Speakers:

Jonas Grossman, President, Chardan

Yvan-Claude Pierre, Partner, Cooley

Stephanie Sirota, Partner and Chief Business Officer, RTW Investments

CURRENT HOT TOPICS AND EMERGING TRENDS IN GENE THERAPY
Sponsored by Pfizer
This panel will focus on emerging gene therapy scientific breakthroughs and challenges as well as notable clinical development advancements and results. Discussion will also touch upon gene therapy manufacturing challenges and the gene therapy financing and investor landscape.

Chair:

Bob Smith, SVP, Global Gene Therapy Business, Pfizer Inc.

Speakers:

Katherine High, M.D., Visiting Professor, Rockefeller University; Co-Founder, Spark Therapeutics

Natalie Holles, President and CEO, Audentes, an Astellas company

Ken Mills, President and CEO, REGENXBIO

DOING BUSINESS IN JAPAN – AN EVOLVING MARKET: THE LATEST RISE OF COMMERCIALIZATION AND PROMOTING HARMONIZATION ON REGENERATIVE MEDICINE WITH ASIA

In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)

Opening Remarks

Masayuki (Max) Nomura, Ph.D., Chair, International Affairs Committee, Forum for Innovative Regenerative Medicine (FIRM)

Update on Regulatory Aspects of Gene Therapy Products

Akira Sakurai, Ph.D., Deputy Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)

Points to Consider on the Quality of Raw Materials for the Manufacture of Cell-based Therapeutic Products

Yoji Sato, Ph.D., Head, Division of Cell-Based Therapeutic Products, National Institute of Health Sciences, Japan

Regulation in Asia, U.S., and Europe on Starting Cells for Cellular Therapy Products and Risk-based Approaches

Toshimitsu Tanaka, Member, Regulatory Harmonization Committee, Forum for Innovative Regenerative Medicine (FIRM)

mRNA PROCESS DEVELOPMENT AND MANUFACTURING ON THE ROAD TO THE CLINIC
Sponsored by Aldevron
Messenger RNA offers a promising new approach to address emerging pathogens and unmet medical needs. Shorter lead times and greater simplicity in manufacturing enable faster responses that will transform medicine. This panel will discuss the state of the art with an emphasis on CMC, manufacturing, and associated regulatory issues.

Chair:

Nate Spangler, Ph.D., Director of Innovation and Strategy, Aldevron

Speakers:

Frank DeRosa, Ph.D., Chief Technology Officer, Translate Bio

Andrew Geall, Ph.D., Chief Scientific Officer, Precision NanoSystems

Caitlyn Harvey, CMC Strategy and Project Management, Myeloid Therapeutics

Jeffrey Ulmer, Ph.D., mRNA Therapy and Vaccine Expert

NOVEL CLINICAL TRIAL DESIGNS: MORE EFFICIENT AND EFFECTIVE TESTS OF REGENERATIVE MEDICINES DURING THE PANDEMIC AND BEYOND
Sponsored by IQVIA
This panel of industry, advocacy, and academic leaders will discuss challenges faced and innovative solutions explored, in addressing the unique requirements of clinical studies in cell and gene therapy. Participants will present their first-hand experience with various novel trial designs/elements including remote endpoints/telemedicine, virtual trials, hybrid/decentralized trials, platform trials, and external comparators. Cell and gene therapy trials already incur a unique set of challenges, and COVID-19 has dramatically transformed the clinical trial landscape, highlighting and accelerating the need for new ways of thinking that are more relevant now, but which will continue to be increasingly employed going forward.

Chair:

David Cameron, Global Head of Novel Trial Design, IQVIA

Speakers:

Hearn Jay Cho, M.D., Ph.D., Chief Medical Officer, Multiple Myeloma Research Foundation

Shaji Kumar, M.D., Professor of Medicine, Division of Hematology, Mayo Clinic

Sabrina Paganoni, M.D., Ph.D., Assistant Professor, Harvard Medical School; Physician Scientist, Healey Center for ALS, Massachusetts General Hospital and Spaulding Rehabilitation Hospital

Aiman Shalabi, Pharm.D., VP R&D Cell and Gene Therapies, GSK

SAFETY AND EFFICACY CONSIDERATIONS FOR CELL AND GENE THERAPIES: FROM MANUFACTURING THROUGH DISTRIBUTION
Sponsored by Thermo Fisher Scientific
This panel discussion will cover a number of safety and efficacy factors for advanced therapies including acceptable impurity levels, dosing considerations, the impact of platforms on the potency of products, and maintaining integrity of the material from the manufacturing floor to the clinic.

Chair:

Susan D’Costa, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific

Speakers:

Justin Horvath, Director of External Manufacturing, REGENXBIO

Anne Kantardjieff, Ph.D., Senior Director, External Vector Manufacturing, bluebird bio

ON-DEMAND CONTENT
Available Beginning Tuesday, October 13, 2020

EUROPEAN COMMERCIALIZATION CHALLENGES AND OUTLOOK

With 10 advanced therapy medicinal products (ATMPs) currently marketed in Europe and four withdrawn, the sector’s commercial landscape is at a turning point in Europe. So far, healthcare systems and developers have been successful in opening the way to the cell and gene therapy revolution. Treatment centers are managing patients, payers are exploring innovative ways to cover the cost for transformative therapies, and developers are inventing new ways of doing business. The next step will be to consolidate all of these successes and make them available for a significantly larger number of patients. On this panel, European business leaders will share their expectations and discuss how they are preparing to overcome these challenges.

Chair:

Christopher Vann, SVP and Chief Operating Officer, Autolus

Speakers:

Jacqueline Barry, Ph.D., Chief Clinical Officer, Cell & Gene Therapy Catapult

Nicola Heffron, SVP, Head of European Operations, bluebird bio

Emanuele Ostuni, Ph.D., Head of Europe Cell and Gene Therapy, Novartis Oncology

GMP MANUFACTURING OF CELL AND GENE THERAPIES – A 2020 PERSPECTIVE

This session will include a discussion about issues related to moving from development through to GMP manufacturing of cell and gene therapies including challenges, scalability, standards, and issues that have been raised in light of COVID-19.

Chair:

Kelvin Lee, Ph.D., Institute Director, National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)

Speakers:

Delara Motlagh, Ph.D., VP, Cell Therapy Technologies, Terumo BCT

Claudia Zylberberg, Ph.D., CEO, Akron Biotech

STATE OF PLAY IN THE VECTOR ARMS RACE: PERSPECTIVES ON THE FUTURE OF GENE DELIVERY

Join the CEOs of three leading gene therapy companies as they discuss the relative merits of AAV, lentiviral, and non-viral delivery technology – and share their views on how these platforms will help shape the future of gene therapy. Expect a lively, insightful, and thought-provoking discussion. Both Prevail Therapeutics and AVROBIO are in the clinic and have publicly shared patient data while Generation Bio is preclinical with a novel non-viral platform.

Chair:

Amber Tong, Editor, Endpoints News

Speakers:

Asa Abeliovich, M.D., Ph.D., Founder and CEO, Prevail Therapeutics

Geoff MacKay, President and CEO, AVROBIO

Geoff McDonough, M.D., President and CEO, Generation Bio

VITALITY OF THE RANGE OF INVESTING: EARLY-STAGE SEED INVESTING TO PUBLIC INVESTING
This discussion between three leaders in biotech/pharma investing will delve into the vitality of early stage seed funding to mid stage to public investing in established companies, and how these stages of the investment spectrum interact. The panel will also discuss the importance of alternative investment structures such as venture philanthropy to promote therapeutic pipelines.

Chair:

Jason Rhodes, Partner, Atlas Venture

Speakers:

Rajiv Kaul, Portfolio Manager, Fidelity Investments

Debra Miller, Founder and CEO, CureDuchenne; CEO, CureDuchenne Ventures

ON-DEMAND CONTENT
Available Beginning Wednesday, October 14, 2020

ACHIEVING DIVERSITY IN CLINICAL TRIALS

While obtaining racial diversity in clinical trials is critical to ensuring that the patients enrolled are representative of the population that will receive treatment, many clinical trials fail to achieve sufficient representation. The ARM Action for Equality (AFE) Taskforce has convened this panel to discuss the barriers, lessons learned, and opportunities to increase representation in clinical trials evaluating products for cell and gene therapy.

Chair:

Faraz Ali, CEO, Tenaya Therapeutics

Speakers:

Rear Admiral Richardae Araojo, Pharm.D., Associate Commissioner for Minority Health; Director, Office of Minority Health and Health Equity, U.S. Food and Drug Administration (FDA)

Erica Prowisor, Global Head of Patient Recruitment and Retention, IQVIA

Dara Richardson-Heron, M.D., Chief Patient Officer, Pfizer Inc.

BESPOKE THERAPIES FOR ULTRA-RARE DISEASE

This session will explore the National Institutes of Health (NIH)’s 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:

Yael Weiss, M.D., Ph.D., VP, Business Development, Ultragenyx

Speakers:

Philip J. Brooks, Ph.D., Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)

Barry Byrne, M.D., Ph.D., Professor and Associate Chair of Pediatrics; Director of the Powell Gene Therapy Center, University of Florida

Joseph Menetski, Ph.D., Associate VP of Research Partnerships, Foundation for the National Institutes of Health

Bob Smith, SVP, Global Gene Therapy Business, Pfizer Inc.

BETWEEN TWO VIRTUAL FERNS: BUILDING A SUCCESSFUL REGENERATIVE MEDICINE COMPANY

Please join us for a conversation about the key considerations for building a successful regenerative medicine company from the perspectives of an operating executive and an investor.

Speakers:

Matthew Patterson, Strategic Advisor, Audentes Therapeutics, An Astellas Company

Kush Parmar, M.D., Ph.D., Managing Partner, 5AM Ventures

THE EUROPEAN REGULATORY ENVIRONMENT FOR ATMPS – SHOULD WE EXPECT MORE OR LESS REGULATION?

This session will discuss the recent initiatives and plans that will affect the regulatory environment for advanced therapy medicinal products (ATMPs) in Europe, such as the EC Pharmaceutical Strategy, the forthcoming implementation of the new Clinical Trial Regulation, GMO requirements for gene therapies, and the European Regulatory Network Strategy to 2025. The impact of these developments on patient access to ATMPs, on the ATMP sector in Europe, and on regulatory bodies will be addressed.

Chair:

Miguel Forte, CEO, Bone Therapeutics

Speakers:

Karolina Hanslik, Senior Project Manager, EURORDIS

Ana Hidalgo-Simon, M.D., Ph.D., Head of Advanced Therapies, European Medicines Agency (EMA)

Rocio Salvador Roldan, Policy Officer, Unit B4- Medical Products, Directorate-General for Health and Food Safety (DG SANTE), European Commission

Martina Schüssler-Lenz, M.D., Chair, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA); Senior Clinical Assessor for ATMPs, Paul-Ehrlich-Institut

ON-DEMAND CONTENT
Available Beginning Thursday, October 15, 2020

ARM ACTION FOR EQUALITY TASK FORCE INITIATIVES

The Alliance for Regenerative Medicine (ARM) Action for Equality (AFE) Task Force is exploring what actions ARM and ARM members can take to eliminate racial inequality and address underrepresentation of Black Americans and minorities in the regenerative medicine sector. Our goal is to create a meaningful impact through various initiatives that will provide minorities with more opportunities, exposure, and mentorship in regenerative medicine. The Task Force aims to build a successful program that ARM and ARM Members are energized to pursue together.

Chair:

Emile Nuwaysir, Ph.D., President and CEO, BlueRock Therapeutics; Vice Chairman, Alliance for Regenerative Medicine (ARM)

Speakers:

Usman “Oz” Azam, M.D., President and CEO, Tmunity Therapeutics

Francesca Cook, Senior Director, Pricing and Market Access, REGENXBIO

Anshul Mangal, Founder, CEO and General Counsel, Project Farma

Courtney Utsey, Senior Manager HR Operations, Talent and Culture, Enzyvant

CELL AND GENE THERAPY PORTFOLIO STRATEGIES

This session will focus on how mid- to large-sized biotech and pharma companies develop a portfolio strategy designed to effectively progress cell and gene therapies. The panelists will discuss their respective approaches to building a robust pipeline.

Chair:

Arthur Tzianabos, Ph.D., President and CEO, Homology Medicines

Speakers:

Claudia Mitchell, Ph.D., SVP, Head of Portfolio Strategy, Astellas

Sanjaya Singh, Ph.D., VP and Global Head, Janssen BioTherapeutics, Janssen Research & Development, LLC, Janssen Pharmaceutical Companies of Johnson & Johnson

Anabella Villalobos, Ph.D., SVP, Biotherapeutics and Medicinal Sciences, Biogen

FDA WORKING WITH PATIENT GROUPS

This panel will discuss innovative ways patient groups can work with the FDA throughout the drug development and evaluation process. In this session, panelists will explore the use of real-world evidence, patient experience, and registries as replacements for more traditional methods of data collection.

Chair:

Annie Kennedy, Chief of Policy and Advocacy, Everylife Foundation for Rare Diseases

Speakers:

Ryan Fischer, SVP, Community Engagement, Parent Project Muscular Dystrophy

Monica Shah, M.D., VP and Head, Cell and Gene Therapy Center of Excellence, IQVIA

Kimberly Trant, Executive Director, Head of Patient Advocacy and Engagement, Audentes, an Astellas company

OVERCOMING CHALLENGES WITH THE DEVELOPMENT AND TRANSLATION OF A THERAPEUTIC FROM ACADEMIA TO INDUSTRY

Some of the most cutting-edge and innovative approaches to cell and gene therapy have originated from the academic setting. However, getting these treatments into the clinic can be especially challenging when the institution does not have the experience and infrastructure necessary to conduct preclinical studies, manufacture to GMP standards, and file regulatory submissions with the FDA. This panel of experts will discuss their lessons learned throughout the process and best approaches to moving a cell and gene therapeutic candidate from academia to commercialization.

Chair:

Shawn Roach, Ph.D., Specialist-Regulatory CMC, Halloran Consulting Group

Speakers:

Isabelle Riviere, Ph.D., Director, Cell Therapy and Cell Engineering Facility, Memorial Sloan Kettering Cancer Center

Anthony Ting, Ph.D., VP of Regenerative Medicine and Head of Cardiopulmonary Programs, Athersys

REGULATION & POLICY

FEATURED FIRESIDE CHAT: FDA PERSPECTIVE

8am – 9am PT / 11am – 12pm ET

Chair:

Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)

Speakers:

Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)

FEATURED FIRESIDE CHAT: EMA PERSPECTIVE

8am – 9am PT / 11am – 12pm ET

Chair:

Paolo Morgese, Director, EU Market Access and Member Relations, Alliance for Regenerative Medicine (ARM)

Speakers:

Guido Rasi, M.D., Director General, European Medicines Agency (EMA)

BESPOKE THERAPIES FOR ULTRA-RARE DISEASE

This session will explore the National Institutes of Health (NIH)’s 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:

Yael Weiss, M.D., Ph.D., VP, Business Development, Ultragenyx

Speakers:

Philip J. Brooks, Ph.D., Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)

Barry Byrne, M.D., Ph.D., Professor and Associate Chair of Pediatrics; Director of the Powell Gene Therapy Center, University of Florida

Joseph Menetski, Ph.D., Associate VP of Research Partnerships, Foundation for the National Institutes of Health

Bob Smith, SVP, Global Gene Therapy Business, Pfizer Inc.

FDA WORKING WITH PATIENT GROUPS

This panel will discuss innovative ways patient groups can work with the FDA throughout the drug development and evaluation process. In this session, panelists will explore the use of real-world evidence, patient experience, and registries as replacements for more traditional methods of data collection.

Chair:

Annie Kennedy, Chief of Policy and Advocacy, Everylife Foundation for Rare Diseases

Speakers:

Ryan Fischer, SVP, Community Engagement, Parent Project Muscular Dystrophy

Monica Shah, M.D., VP and Head, Cell and Gene Therapy Center of Excellence, IQVIA

Kimberly Trant, Executive Director, Head of Patient Advocacy and Engagement, Audentes, an Astellas company

INTERNATIONAL SESSIONS

DOING BUSINESS IN JAPAN – AN EVOLVING MARKET: THE LATEST RISE OF COMMERCIALIZATION AND PROMOTING HARMONIZATION ON REGENERATIVE MEDICINE WITH ASIA
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)

Opening Remarks

Masayuki (Max) Nomura, Ph.D., Chair, International Affairs Committee, Forum for Innovative Regenerative Medicine (FIRM)

Update on Regulatory Aspects of Gene Therapy Products

Akira Sakurai, Ph.D., Deputy Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)

Points to Consider on the Quality of Raw Materials for the Manufacture of Cell-based Therapeutic Products

Yoji Sato, Ph.D., Head, Division of Cell-Based Therapeutic Products, National Institute of Health Sciences, Japan

Regulation in Asia, U.S., and Europe on Starting Cells for Cellular Therapy Products and Risk-based Approaches

Toshimitsu Tanaka, Member, Regulatory Harmonization Committee, Forum for Innovative Regenerative Medicine (FIRM)

EUROPEAN COMMERCIALIZATION CHALLENGES AND OUTLOOK

With 10 advanced therapy medicinal products (ATMPs) currently marketed in Europe and four withdrawn, the sector’s commercial landscape is at a turning point in Europe. So far, healthcare systems and developers have been successful in opening the way to the cell and gene therapy revolution. Treatment centers are managing patients, payers are exploring innovative ways to cover the cost for transformative therapies, and developers are inventing new ways of doing business. The next step will be to consolidate all of these successes and make them available for a significantly larger number of patients. On this panel, European business leaders will share their expectations and discuss how they are preparing to overcome these challenges.

Chair:

Christopher Vann, SVP and Chief Operating Officer, Autolus

Speakers:

Jacqueline Barry, Ph.D., Chief Clinical Officer, Cell & Gene Therapy Catapult

Nicola Heffron, SVP, Head of European Operations, bluebird bio

Emanuele Ostuni, Ph.D., Head of Europe Cell and Gene Therapy, Novartis Oncology

THE EUROPEAN REGULATORY ENVIRONMENT FOR ATMPS – SHOULD WE EXPECT MORE OR LESS REGULATION?

This session will discuss the recent initiatives and plans that will affect the regulatory environment for advanced therapy medicinal products (ATMPs) in Europe, such as the EC Pharmaceutical Strategy, the forthcoming implementation of the new Clinical Trial Regulation, GMO requirements for gene therapies, and the European Regulatory Network Strategy to 2025. The impact of these developments on patient access to ATMPs, on the ATMP sector in Europe, and on regulatory bodies will be addressed.

Chair:

Miguel Forte, CEO, Bone Therapeutics

Speakers:

Karolina Hanslik, Senior Project Manager, EURORDIS

Ana Hidalgo-Simon, M.D., Ph.D., Head of Advanced Therapies, European Medicines Agency (EMA)

Rocio Salvador Roldan, Policy Officer, Unit B4- Medical Products, Directorate-General for Health and Food Safety (DG SANTE), European Commission

Martina Schüssler-Lenz, M.D., Chair, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA); Senior Clinical Assessor for ATMPs, Paul-Ehrlich-Institut

COMMERCIALIZATION & PRODUCT DEVELOPMENT

PLENARY SESSION: THE REALITIES OF BRINGING GENE THERAPIES TO MARKET

8am – 9am PT / 11am – 12pm ET

Chair:

Jeff Walsh, Former Chief Financial and Strategy Officer and Current Strategic Advisor, bluebird bio

Speakers:

Jeff Ajer, EVP and Chief Commercial Officer, BioMarin
Lisa Deschamps, Chief Business Officer, AveXis, a Novartis company
Jay Newman, Head, U.S. Commercial, Spark Therapeutics
Braden Parker, SVP and General Manager, North America, Orchard Therapeutics

PLENARY SESSION: THE EVOLUTION OF COMMERCIAL CELL THERAPY

8am – 9am PT / 11am – 12pm ET

Chair:

Ann Lee, Ph.D., SVP and Head of Cell Therapy Development and Operations, Bristol-Myers Squibb

Speakers:

Marco Gottardis, Ph.D., VP, Oncology Innovation, Janssen R&D
Alberto Santagostino, SVP, Head of Cell and Gene Technologies, Lonza Pharma & Biotech
Kristin Yarema, Ph.D., Chief Commercial Officer, Atara Biotherapeutics

ACCEPTANCE AND UPTAKE OF CELL AND GENE THERAPIES: LESSONS LEARNED AND FUTURE FOCUS

Sponsored by Evidera PPD
The first wave of transformative cell and gene therapies have launched into a healthcare landscape that arguably was not built with them in mind. Hurdles range from basic understanding to value demonstration, reimbursement and access. This session will debate lessons learned from the initial vanguard of cell and gene therapies, including building the right evidence, navigating the provider-side, achieving value-based payment, and ensuring patient uptake. Discussion will cover unanticipated challenges and critical success factors for future cell and gene therapy success.

Chair:

Eric Faulkner, VP, Precision and Transformative Medicine, Evidera; Executive Director, Genomics Biotech and Emerging Technology Institute, NAMCP

Speakers:

Shannon Baumann, VP, Access, Value and Evidence Strategy, bluebird bio

Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics

Tay Salimullah, VP, Global Head of Patient Access, AveXis, a Novartis company

BETWEEN TWO VIRTUAL FERNS: BUILDING A SUCCESSFUL REGENERATIVE MEDICINE COMPANY

Please join us for a conversation about the key considerations for building a successful regenerative medicine company from the perspectives of an operating executive and an investor.

Speakers:

Matthew Patterson, Strategic Advisor, Audentes Therapeutics, An Astellas Company

Kush Parmar, M.D., Ph.D., Managing Partner, 5AM Ventures

INVESTING IN CELL & GENE THERAPY

ALTERNATIVE IPO STRATEGIES FOR CELL AND GENE THERAPY COMPANIES
Sponsored by Chardan
This session will center on special purpose acquisition companies (SPACs), a space heating up within the biotech world. As of August 2020, 10 biotech-focused SPACs have been successful at raising capital with many others coming down the pike. The purpose of the panel is to understand why all these biotech-focused firms are getting involved in the space, what cell and gene therapy companies need to know about this alternative to a traditional IPO if approached, and what seasoned executives of public companies should know about potentially sponsoring their own biotech focused SPAC.

Chair:

Gbola Amusa, M.D., Partner, Director of Research and Head of Healthcare Research, Chardan

Speakers:

Jonas Grossman, President, Chardan

Yvan-Claude Pierre, Partner, Cooley

Stephanie Sirota, Partner and Chief Business Officer, RTW Investments

VITALITY OF THE RANGE OF INVESTING: EARLY-STAGE SEED INVESTING TO PUBLIC INVESTING
This discussion between three leaders in biotech/pharma investing will delve into the vitality of early stage seed funding to mid stage to public investing in established companies, and how these stages of the investment spectrum interact. The panel will also discuss the importance of alternative investment structures such as venture philanthropy to promote therapeutic pipelines.

Chair:

Jason Rhodes, Partner, Atlas Venture

Speakers:

Rajiv Kaul, Portfolio Manager, Fidelity Investments

Debra Miller, Founder and CEO, CureDuchenne; CEO, CureDuchenne Ventures

TECHNOLOGY & MANUFACTURING

DISRUPTIVE TECHNOLOGIES THAT WILL CHANGE THE CELL THERAPY LANDSCAPE

12pm – 1pm PT / 3pm – 4pm ET
This panel of industry leaders will reflect on the past, present, and future to discuss how foundational technologies, new modes of operation, and changes in the talent pool are rapidly transforming the cell and gene therapy industry from futuristic promise to one of the hottest sectors in biotech.

Chair:

Michael May, Ph.D., President and CEO, CCRM

Speakers:

Dan Shoemaker, Ph.D., Chief Scientific Officer, Fate Therapeutics
Helen Tayton-Martin, Ph.D., Co-Founder and Chief Business Officer, Adaptimmune
James Trager, Ph.D., Chief Scientific Officer, Nkarta Therapeutics
Stefan Wildt, Ph.D., Head of Pharmaceutical Sciences and Translational Engine, Cell Therapies, Takeda

CELL AND GENE THERAPIES FOR CHRONIC CONDITIONS

12pm – 1pm PT / 3pm – 4pm ET

Cell and gene therapies represent hope for millions of patients with chronic diseases — not just the possibility of better management, but, in some cases, the promise of a durable cure. This emerging field faces many hurdles, however, including effective delivery of therapies to patients, unique manufacturing challenges, educating regulators on the field, and ensuring market access and acceptance of value-based reimbursement policies. This panel brings together the leaders of five top companies in the sector to explore and discuss these challenges.

Chair:

Devyn Smith, Ph.D., Chief Operations Officer and Head of Strategy, Sigilon Therapeutics

Speakers:

Pavan Cheruvu, M.D., CEO, Axovant Gene Therapies
Jane Lebkowski, Ph.D., President, Regenerative Patch Technologies
Emile Nuwaysir, Ph.D., President and CEO, BlueRock Therapeutics
Philip Toleikis, Ph.D., President and CEO, Sernova

THE LASTING COVID-19 IMPACT ON CELL AND GENE THERAPY

12pm – 1pm PT / 3pm – 4pm ET

Chair:

Anthony Davies, Ph.D., Founder and CEO, Dark Horse Consulting

Speakers:

Wilson W. Bryan, M.D., Director, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)
Amy DuRoss, Co-Founder and CEO, Vineti
Curran Simpson, Chief Operations and Technology Officer, REGENXBIO
Timothy Schroeder, Founder and CEO, CTI Clinical Trial & Consulting

THE EMERGENCE OF GENE EDITED CARs AND TCRs

Gene editing is a powerful tool used to improve the performance of a patient’s immune cells, and to facilitate the use of allogeneic adoptive cell therapies. This panel will discuss this rapidly developing area and the implications for the future of the treatment of cancer.

12pm – 1pm PT / 3pm – 4pm ET

Chair:

Charlie Albright, Ph.D., EVP and Chief Scientific Officer, Editas Medicine

Speakers:

Jason Fontenot, Ph.D., SVP, Cell Therapy and Head of Research, Sangamo Therapeutics
Andrew Schiermeier, Ph.D., EVP and Chief Operating Officer, Intellia Therapeutics

ACCELERATING BIOTHERAPEUTIC DEVELOPMENT AND PRODUCTION FOR INCREASED MARKET IMPACT OF LIFE SAVING THERAPEUTICS

Sponsored by Thermo Fisher Scientific
This panel of experts will share their insights and experience on some of today’s most pressing development challenges. The session will dive into key topics such as integrated vs. modular manufacturing approaches, ensuring security of supply, proactive approaches to regulatory compliance, and ultimately, the panel’s vision for the future.

Chair:

Susan D’Costa, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific

Speakers:

Alex Fotopoulos, Chief Technical Officer, Passage Bio

Michael Mercaldi, Ph.D., Senior Director, Downstream Process Development, Homology Medicines

Nripen Singh, Ph.D., Director, Biopharmaceutical Development, Voyager Therapeutics

ACHIEVING DIVERSITY IN CLINICAL TRIALS

While obtaining racial diversity in clinical trials is critical to ensuring that the patients enrolled are representative of the population that will receive treatment, many clinical trials fail to achieve sufficient representation. The ARM Action for Equality (AFE) Taskforce has convened this panel to discuss the barriers, lessons learned, and opportunities to increase representation in clinical trials evaluating products for cell and gene therapy.

Chair:

Chair:

Faraz Ali, CEO, Tenaya Therapeutics

Speakers:

Rear Admiral Richardae Araojo, Pharm.D., Associate Commissioner for Minority Health; Director, Office of Minority Health and Health Equity, U.S. Food and Drug Administration (FDA)

Erica Prowisor, Global Head of Patient Recruitment and Retention, IQVIA

Dara Richardson-Heron, M.D., Chief Patient Officer, Pfizer Inc.

ARM ACTION FOR EQUALITY TASK FORCE INITIATIVES

The Alliance for Regenerative Medicine (ARM) Action for Equality (AFE) Task Force is exploring what actions ARM and ARM members can take to eliminate racial inequality and address underrepresentation of Black Americans and minorities in the regenerative medicine sector. Our goal is to create a meaningful impact through various initiatives that will provide minorities with more opportunities, exposure, and mentorship in regenerative medicine. The Task Force aims to build a successful program that ARM and ARM Members are energized to pursue together.

Chair:

Emile Nuwaysir, Ph.D., President and CEO, BlueRock Therapeutics; Vice Chairman, Alliance for Regenerative Medicine (ARM)

Speakers:

Usman “Oz” Azam, M.D., President and CEO, Tmunity Therapeutics

Francesca Cook, Senior Director, Pricing and Market Access, REGENXBIO

Anshul Mangal, Founder, CEO and General Counsel, Project Farma

Courtney Utsey, Senior Manager HR Operations, Talent and Culture, Enzyvant

CELL AND GENE THERAPY PORTFOLIO STRATEGIES

This session will focus on how mid- to large-sized biotech and pharma companies develop a portfolio strategy designed to effectively progress cell and gene therapies. The panelists will discuss their respective approaches to building a robust pipeline.

Chair:

Arthur Tzianabos, Ph.D., President and CEO, Homology Medicines

Speakers:

Claudia Mitchell, Ph.D., SVP, Head of Portfolio Strategy, Astellas

Sanjaya Singh, Ph.D., VP and Global Head, Janssen BioTherapeutics, Janssen Research & Development, LLC, Janssen Pharmaceutical Companies of Johnson & Johnson

Anabella Villalobos, Ph.D., SVP, Biotherapeutics and Medicinal Sciences, Biogen

CURRENT HOT TOPICS AND EMERGING TRENDS IN GENE THERAPY
Sponsored by Pfizer
This panel will focus on emerging gene therapy scientific breakthroughs and challenges as well as notable clinical development advancements and results. Discussion will also touch upon gene therapy manufacturing challenges and the gene therapy financing and investor landscape.

Chair:

Bob Smith, SVP, Global Gene Therapy Business, Pfizer Inc.

Speakers:

Katherine High, M.D., Visiting Professor, Rockefeller University; Co-Founder, Spark Therapeutics

Natalie Holles, President and CEO, Audentes, an Astellas company

Ken Mills, President and CEO, REGENXBIO

GMP MANUFACTURING OF CELL AND GENE THERAPIES – A 2020 PERSPECTIVE

This session will include a discussion about issues related to moving from development through to GMP manufacturing of cell and gene therapies including challenges, scalability, standards, and issues that have been raised in light of COVID-19.

Chair:

Kelvin Lee, Ph.D., Institute Director, National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)

Speakers:

Delara Motlagh, Ph.D., VP, Cell Therapy Technologies, Terumo BCT

Claudia Zylberberg, Ph.D., CEO, Akron Biotech

mRNA PROCESS DEVELOPMENT AND MANUFACTURING ON THE ROAD TO THE CLINIC
Sponsored by Aldevron
Messenger RNA offers a promising new approach to address emerging pathogens and unmet medical needs. Shorter lead times and greater simplicity in manufacturing enable faster responses that will transform medicine. This panel will discuss the state of the art with an emphasis on CMC, manufacturing, and associated regulatory issues.

Chair:

Nate Spangler, Ph.D., Director of Innovation and Strategy, Aldevron

Speakers:

Frank DeRosa, Ph.D., Chief Technology Officer, Translate Bio

Andrew Geall, Ph.D., Chief Scientific Officer, Precision NanoSystems

Caitlyn Harvey, CMC Strategy and Project Management, Myeloid Therapeutics

Jeffrey Ulmer, Ph.D., mRNA Therapy and Vaccine Expert

NOVEL CLINICAL TRIAL DESIGNS: MORE EFFICIENT AND EFFECTIVE TESTS OF REGENERATIVE MEDICINES DURING THE PANDEMIC AND BEYOND
Sponsored by IQVIA
This panel of industry, advocacy, and academic leaders will discuss challenges faced and innovative solutions explored, in addressing the unique requirements of clinical studies in cell and gene therapy. Participants will present their first-hand experience with various novel trial designs/elements including remote endpoints/telemedicine, virtual trials, hybrid/decentralized trials, platform trials, and external comparators. Cell and gene therapy trials already incur a unique set of challenges, and COVID-19 has dramatically transformed the clinical trial landscape, highlighting and accelerating the need for new ways of thinking that are more relevant now, but which will continue to be increasingly employed going forward.

Chair:

David Cameron, Global Head of Novel Trial Design, IQVIA

Speakers:

Hearn Jay Cho, M.D., Ph.D., Chief Medical Officer, Multiple Myeloma Research Foundation

Shaji Kumar, M.D., Professor of Medicine, Division of Hematology, Mayo Clinic

Sabrina Paganoni, M.D., Ph.D., Assistant Professor, Harvard Medical School; Physician Scientist, Healey Center for ALS, Massachusetts General Hospital and Spaulding Rehabilitation Hospital

Aiman Shalabi, Pharm.D., VP R&D Cell and Gene Therapies, GSK

OVERCOMING CHALLENGES WITH THE DEVELOPMENT AND TRANSLATION OF A THERAPEUTIC FROM ACADEMIA TO INDUSTRY

Some of the most cutting-edge and innovative approaches to cell and gene therapy have originated from the academic setting. However, getting these treatments into the clinic can be especially challenging when the institution does not have the experience and infrastructure necessary to conduct preclinical studies, manufacture to GMP standards, and file regulatory submissions with the FDA. This panel of experts will discuss their lessons learned throughout the process and best approaches to moving a cell and gene therapeutic candidate from academia to commercialization.

Chair:

Shawn Roach, Ph.D., Specialist-Regulatory CMC, Halloran Consulting Group

Speakers:

Isabelle Riviere, Ph.D., Director, Cell Therapy and Cell Engineering Facility, Memorial Sloan Kettering Cancer Center

Anthony Ting, Ph.D., VP of Regenerative Medicine and Head of Cardiopulmonary Programs, Athersys

SAFETY AND EFFICACY CONSIDERATIONS FOR CELL AND GENE THERAPIES: FROM MANUFACTURING THROUGH DISTRIBUTION
Sponsored by Thermo Fisher Scientific
This panel discussion will cover a number of safety and efficacy factors for advanced therapies including acceptable impurity levels, dosing considerations, the impact of platforms on the potency of products, and maintaining integrity of the material from the manufacturing floor to the clinic.

Chair:

Susan D’Costa, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific

Speakers:

Justin Horvath, Director of External Manufacturing, REGENXBIO

Anne Kantardjieff, Ph.D., Senior Director, External Vector Manufacturing, bluebird bio

STATE OF PLAY IN THE VECTOR ARMS RACE: PERSPECTIVES ON THE FUTURE OF GENE DELIVERY

Join the CEOs of three leading gene therapy companies as they discuss the relative merits of AAV, lentiviral, and non-viral delivery technology – and share their views on how these platforms will help shape the future of gene therapy. Expect a lively, insightful, and thought-provoking discussion. Both Prevail Therapeutics and AVROBIO are in the clinic and have publicly shared patient data while Generation Bio is preclinical with a novel non-viral platform.

Chair:

Amber Tong, Editor, Endpoints News

Speakers:

Asa Abeliovich, M.D., Ph.D., Founder and CEO, Prevail Therapeutics

Geoff MacKay, President and CEO, AVROBIO

Geoff McDonough, M.D., President and CEO, Generation Bio

LIVE STREAMING SESSIONS WITH Q&A

PLENARY SESSION: THE REALITIES OF BRINGING GENE THERAPIES TO MARKET

8am – 9am PT / 11am – 12pm ET

Chair:

Jeff Walsh, Former Chief Financial and Strategy Officer and Current Strategic Advisor, bluebird bio

Speakers:

Jeff Ajer, EVP and Chief Commercial Officer, BioMarin
Lisa Deschamps, Chief Business Officer, AveXis, a Novartis company
Jay Newman, Head, U.S. Commercial, Spark Therapeutics
Braden Parker, SVP and General Manager, North America, Orchard Therapeutics

DISRUPTIVE TECHNOLOGIES THAT WILL CHANGE THE CELL THERAPY LANDSCAPE

12pm – 1pm PT / 3pm – 4pm ET
This panel of industry leaders will reflect on the past, present, and future to discuss how foundational technologies, new modes of operation, and changes in the talent pool are rapidly transforming the cell and gene therapy industry from futuristic promise to one of the hottest sectors in biotech.

Chair:

Michael May, Ph.D., President and CEO, CCRM

Speakers:

Dan Shoemaker, Ph.D., Chief Scientific Officer, Fate Therapeutics
Helen Tayton-Martin, Ph.D., Co-Founder and Chief Business Officer, Adaptimmune
James Trager, Ph.D., Chief Scientific Officer, Nkarta Therapeutics
Stefan Wildt, Ph.D., Head of Pharmaceutical Sciences and Translational Engine, Cell Therapies, Takeda

DURING BREAKS IN LIVE PROGRAMMING
Participate in 1×1 Partnering, Visit Virtual Exhibit Booths, View On-Demand Content

ON-DEMAND CONTENT
Available Beginning Monday, October 12, 2020

120+ COMPANY PRESENTATIONS
View the Full List

ACCELERATING BIOTHERAPEUTIC DEVELOPMENT AND PRODUCTION FOR INCREASED MARKET IMPACT OF LIFE SAVING THERAPEUTICS

Sponsored by Thermo Fisher Scientific
This panel of experts will share their insights and experience on some of today’s most pressing development challenges. The session will dive into key topics such as integrated vs. modular manufacturing approaches, ensuring security of supply, proactive approaches to regulatory compliance, and ultimately, the panel’s vision for the future.

Chair:

Susan D’Costa, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific

Speakers:

Alex Fotopoulos, Chief Technical Officer, Passage Bio

Michael Mercaldi, Ph.D., Senior Director, Downstream Process Development, Homology Medicines

Nripen Singh, Ph.D., Director, Biopharmaceutical Development, Voyager Therapeutics

ACCEPTANCE AND UPTAKE OF CELL AND GENE THERAPIES: LESSONS LEARNED AND FUTURE FOCUS

Sponsored by Evidera PPD
The first wave of transformative cell and gene therapies have launched into a healthcare landscape that arguably was not built with them in mind. Hurdles range from basic understanding to value demonstration, reimbursement and access. This session will debate lessons learned from the initial vanguard of cell and gene therapies, including building the right evidence, navigating the provider-side, achieving value-based payment, and ensuring patient uptake. Discussion will cover unanticipated challenges and critical success factors for future cell and gene therapy success.

Chair:

Eric Faulkner, VP, Precision and Transformative Medicine, Evidera; Executive Director, Genomics Biotech and Emerging Technology Institute, NAMCP

Speakers:

Shannon Baumann, VP, Access, Value and Evidence Strategy, bluebird bio

Sarah Pitluck, Head, Global Pricing and Reimbursement, Spark Therapeutics

Tay Salimullah, VP, Global Head of Patient Access, AveXis, a Novartis company

ALTERNATIVE IPO STRATEGIES FOR CELL AND GENE THERAPY COMPANIES
Sponsored by Chardan
This session will center on special purpose acquisition companies (SPACs), a space heating up within the biotech world. As of August 2020, 10 biotech-focused SPACs have been successful at raising capital with many others coming down the pike. The purpose of the panel is to understand why all these biotech-focused firms are getting involved in the space, what cell and gene therapy companies need to know about this alternative to a traditional IPO if approached, and what seasoned executives of public companies should know about potentially sponsoring their own biotech focused SPAC.

Chair:

Gbola Amusa, M.D., Partner, Director of Research and Head of Healthcare Research, Chardan

Speakers:

Jonas Grossman, President, Chardan

Yvan-Claude Pierre, Partner, Cooley

Stephanie Sirota, Partner and Chief Business Officer, RTW Investments

CURRENT HOT TOPICS AND EMERGING TRENDS IN GENE THERAPY
Sponsored by Pfizer
This panel will focus on emerging gene therapy scientific breakthroughs and challenges as well as notable clinical development advancements and results. Discussion will also touch upon gene therapy manufacturing challenges and the gene therapy financing and investor landscape.

Chair:

Bob Smith, SVP, Global Gene Therapy Business, Pfizer Inc.

Speakers:

Katherine High, M.D., Visiting Professor, Rockefeller University; Co-Founder, Spark Therapeutics

Natalie Holles, President and CEO, Audentes, an Astellas company

Ken Mills, President and CEO, REGENXBIO

DOING BUSINESS IN JAPAN – AN EVOLVING MARKET: THE LATEST RISE OF COMMERCIALIZATION AND PROMOTING HARMONIZATION ON REGENERATIVE MEDICINE WITH ASIA

In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)

Opening Remarks

Masayuki (Max) Nomura, Ph.D., Chair, International Affairs Committee, Forum for Innovative Regenerative Medicine (FIRM)

Update on Regulatory Aspects of Gene Therapy Products

Akira Sakurai, Ph.D., Deputy Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)

Points to Consider on the Quality of Raw Materials for the Manufacture of Cell-based Therapeutic Products

Yoji Sato, Ph.D., Head, Division of Cell-Based Therapeutic Products, National Institute of Health Sciences, Japan

Regulation in Asia, U.S., and Europe on Starting Cells for Cellular Therapy Products and Risk-based Approaches

Toshimitsu Tanaka, Member, Regulatory Harmonization Committee, Forum for Innovative Regenerative Medicine (FIRM)

mRNA PROCESS DEVELOPMENT AND MANUFACTURING ON THE ROAD TO THE CLINIC
Sponsored by Aldevron
Messenger RNA offers a promising new approach to address emerging pathogens and unmet medical needs. Shorter lead times and greater simplicity in manufacturing enable faster responses that will transform medicine. This panel will discuss the state of the art with an emphasis on CMC, manufacturing, and associated regulatory issues.

Chair:

Nate Spangler, Ph.D., Director of Innovation and Strategy, Aldevron

Speakers:

Frank DeRosa, Ph.D., Chief Technology Officer, Translate Bio

Andrew Geall, Ph.D., Chief Scientific Officer, Precision NanoSystems

Caitlyn Harvey, CMC Strategy and Project Management, Myeloid Therapeutics

Jeffrey Ulmer, Ph.D., mRNA Therapy and Vaccine Expert

NOVEL CLINICAL TRIAL DESIGNS: MORE EFFICIENT AND EFFECTIVE TESTS OF REGENERATIVE MEDICINES DURING THE PANDEMIC AND BEYOND
Sponsored by IQVIA
This panel of industry, advocacy, and academic leaders will discuss challenges faced and innovative solutions explored, in addressing the unique requirements of clinical studies in cell and gene therapy. Participants will present their first-hand experience with various novel trial designs/elements including remote endpoints/telemedicine, virtual trials, hybrid/decentralized trials, platform trials, and external comparators. Cell and gene therapy trials already incur a unique set of challenges, and COVID-19 has dramatically transformed the clinical trial landscape, highlighting and accelerating the need for new ways of thinking that are more relevant now, but which will continue to be increasingly employed going forward.

Chair:

David Cameron, Global Head of Novel Trial Design, IQVIA

Speakers:

Hearn Jay Cho, M.D., Ph.D., Chief Medical Officer, Multiple Myeloma Research Foundation

Shaji Kumar, M.D., Professor of Medicine, Division of Hematology, Mayo Clinic

Sabrina Paganoni, M.D., Ph.D., Assistant Professor, Harvard Medical School; Physician Scientist, Healey Center for ALS, Massachusetts General Hospital and Spaulding Rehabilitation Hospital

Aiman Shalabi, Pharm.D., VP R&D Cell and Gene Therapies, GSK

SAFETY AND EFFICACY CONSIDERATIONS FOR CELL AND GENE THERAPIES: FROM MANUFACTURING THROUGH DISTRIBUTION
Sponsored by Thermo Fisher Scientific
This panel discussion will cover a number of safety and efficacy factors for advanced therapies including acceptable impurity levels, dosing considerations, the impact of platforms on the potency of products, and maintaining integrity of the material from the manufacturing floor to the clinic.

Chair:

Susan D’Costa, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific

Speakers:

Justin Horvath, Director of External Manufacturing, REGENXBIO

Anne Kantardjieff, Ph.D., Senior Director, External Vector Manufacturing, bluebird bio

LIVE STREAMING SESSIONS WITH Q&A

PLENARY SESSION: THE EVOLUTION OF COMMERCIAL CELL THERAPY

8am – 9am PT / 11am – 12pm ET

Chair:

Ann Lee, Ph.D., SVP and Head of Cell Therapy Development and Operations, Bristol-Myers Squibb

Speakers:

Marco Gottardis, Ph.D., VP, Oncology Innovation, Janssen R&D
Alberto Santagostino, SVP, Head of Cell and Gene Technologies, Lonza Pharma & Biotech
Kristin Yarema, Ph.D., Chief Commercial Officer, Atara Biotherapeutics

CELL AND GENE THERAPIES FOR CHRONIC CONDITIONS

12pm – 1pm PT / 3pm – 4pm ET

Cell and gene therapies represent hope for millions of patients with chronic diseases — not just the possibility of better management, but, in some cases, the promise of a durable cure. This emerging field faces many hurdles, however, including effective delivery of therapies to patients, unique manufacturing challenges, educating regulators on the field, and ensuring market access and acceptance of value-based reimbursement policies. This panel brings together the leaders of five top companies in the sector to explore and discuss these challenges.

Chair:

Devyn Smith, Ph.D., Chief Operations Officer and Head of Strategy, Sigilon Therapeutics

Speakers:

Pavan Cheruvu, M.D., CEO, Axovant Gene Therapies
Jane Lebkowski, Ph.D., President, Regenerative Patch Technologies
Emile Nuwaysir, Ph.D., President and CEO, BlueRock Therapeutics
Philip Toleikis, Ph.D., President and CEO, Sernova

DURING BREAKS IN LIVE PROGRAMMING
Participate in 1×1 Partnering, Visit Virtual Exhibit Booths, View On-Demand Content

ON-DEMAND CONTENT
Available Beginning Tuesday, October 13, 2020

EUROPEAN COMMERCIALIZATION CHALLENGES AND OUTLOOK

With 10 advanced therapy medicinal products (ATMPs) currently marketed in Europe and four withdrawn, the sector’s commercial landscape is at a turning point in Europe. So far, healthcare systems and developers have been successful in opening the way to the cell and gene therapy revolution. Treatment centers are managing patients, payers are exploring innovative ways to cover the cost for transformative therapies, and developers are inventing new ways of doing business. The next step will be to consolidate all of these successes and make them available for a significantly larger number of patients. On this panel, European business leaders will share their expectations and discuss how they are preparing to overcome these challenges.

Chair:

Christopher Vann, SVP and Chief Operating Officer, Autolus

Speakers:

Jacqueline Barry, Ph.D., Chief Clinical Officer, Cell & Gene Therapy Catapult

Nicola Heffron, SVP, Head of European Operations, bluebird bio

Emanuele Ostuni, Ph.D., Head of Europe Cell and Gene Therapy, Novartis Oncology

GMP MANUFACTURING OF CELL AND GENE THERAPIES – A 2020 PERSPECTIVE

This session will include a discussion about issues related to moving from development through to GMP manufacturing of cell and gene therapies including challenges, scalability, standards, and issues that have been raised in light of COVID-19.

Chair:

Kelvin Lee, Ph.D., Institute Director, National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)

Speakers:

Delara Motlagh, Ph.D., VP, Cell Therapy Technologies, Terumo BCT

Claudia Zylberberg, Ph.D., CEO, Akron Biotech

STATE OF PLAY IN THE VECTOR ARMS RACE: PERSPECTIVES ON THE FUTURE OF GENE DELIVERY

Join the CEOs of three leading gene therapy companies as they discuss the relative merits of AAV, lentiviral, and non-viral delivery technology – and share their views on how these platforms will help shape the future of gene therapy. Expect a lively, insightful, and thought-provoking discussion. Both Prevail Therapeutics and AVROBIO are in the clinic and have publicly shared patient data while Generation Bio is preclinical with a novel non-viral platform.

Chair:

Amber Tong, Editor, Endpoints News

Speakers:

Asa Abeliovich, M.D., Ph.D., Founder and CEO, Prevail Therapeutics

Geoff MacKay, President and CEO, AVROBIO

Geoff McDonough, M.D., President and CEO, Generation Bio

VITALITY OF THE RANGE OF INVESTING: EARLY-STAGE SEED INVESTING TO PUBLIC INVESTING
This discussion between three leaders in biotech/pharma investing will delve into the vitality of early stage seed funding to mid stage to public investing in established companies, and how these stages of the investment spectrum interact. The panel will also discuss the importance of alternative investment structures such as venture philanthropy to promote therapeutic pipelines.

Chair:

Jason Rhodes, Partner, Atlas Venture

Speakers:

Rajiv Kaul, Portfolio Manager, Fidelity Investments

Debra Miller, Founder and CEO, CureDuchenne; CEO, CureDuchenne Ventures

LIVE STREAMING SESSIONS WITH Q&A

FEATURED FIRESIDE CHAT: FDA PERSPECTIVE

8am – 9am PT / 11am – 12pm ET

Chair:

Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)

Speakers:

Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)

THE LASTING COVID-19 IMPACT ON CELL AND GENE THERAPY

12pm – 1pm PT / 3pm – 4pm ET

Chair:

Anthony Davies, Ph.D., Founder and CEO, Dark Horse Consulting

Speakers:

Wilson W. Bryan, M.D., Director, Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)
Amy DuRoss, Co-Founder and CEO, Vineti
Curran Simpson, Chief Operations and Technology Officer, REGENXBIO
Timothy Schroeder, Founder and CEO, CTI Clinical Trial & Consulting

DURING BREAKS IN LIVE PROGRAMMING
Participate in 1×1 Partnering, Visit Virtual Exhibit Booths, View On-Demand Content

ON-DEMAND CONTENT
Available Beginning Wednesday, October 14, 2020

ACHIEVING DIVERSITY IN CLINICAL TRIALS

While obtaining racial diversity in clinical trials is critical to ensuring that the patients enrolled are representative of the population that will receive treatment, many clinical trials fail to achieve sufficient representation. The ARM Action for Equality (AFE) Taskforce has convened this panel to discuss the barriers, lessons learned, and opportunities to increase representation in clinical trials evaluating products for cell and gene therapy.

Chair:

Faraz Ali, CEO, Tenaya Therapeutics

Speakers:

Rear Admiral Richardae Araojo, Pharm.D., Associate Commissioner for Minority Health; Director, Office of Minority Health and Health Equity, U.S. Food and Drug Administration (FDA)

Erica Prowisor, Global Head of Patient Recruitment and Retention, IQVIA

Dara Richardson-Heron, M.D., Chief Patient Officer, Pfizer Inc.

BESPOKE THERAPIES FOR ULTRA-RARE DISEASE

This session will explore the National Institutes of Health (NIH)’s 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:

Yael Weiss, M.D., Ph.D., VP, Business Development, Ultragenyx

Speakers:

Philip J. Brooks, Ph.D., Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)

Barry Byrne, M.D., Ph.D., Professor and Associate Chair of Pediatrics; Director of the Powell Gene Therapy Center, University of Florida

Joseph Menetski, Ph.D., Associate VP of Research Partnerships, Foundation for the National Institutes of Health

Bob Smith, SVP, Global Gene Therapy Business, Pfizer Inc.

BETWEEN TWO VIRTUAL FERNS: BUILDING A SUCCESSFUL REGENERATIVE MEDICINE COMPANY

Please join us for a conversation about the key considerations for building a successful regenerative medicine company from the perspectives of an operating executive and an investor.

Speakers:

Matthew Patterson, Strategic Advisor, Audentes Therapeutics, An Astellas Company

Kush Parmar, M.D., Ph.D., Managing Partner, 5AM Ventures

THE EUROPEAN REGULATORY ENVIRONMENT FOR ATMPS – SHOULD WE EXPECT MORE OR LESS REGULATION?

This session will discuss the recent initiatives and plans that will affect the regulatory environment for advanced therapy medicinal products (ATMPs) in Europe, such as the EC Pharmaceutical Strategy, the forthcoming implementation of the new Clinical Trial Regulation, GMO requirements for gene therapies, and the European Regulatory Network Strategy to 2025. The impact of these developments on patient access to ATMPs, on the ATMP sector in Europe, and on regulatory bodies will be addressed.

Chair:

Miguel Forte, CEO, Bone Therapeutics

Speakers:

Karolina Hanslik, Senior Project Manager, EURORDIS

Ana Hidalgo-Simon, M.D., Ph.D., Head of Advanced Therapies, European Medicines Agency (EMA)

Rocio Salvador Roldan, Policy Officer, Unit B4- Medical Products, Directorate-General for Health and Food Safety (DG SANTE), European Commission

Martina Schüssler-Lenz, M.D., Chair, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA); Senior Clinical Assessor for ATMPs, Paul-Ehrlich-Institut

LIVE STREAMING SESSIONS WITH Q&A

FEATURED FIRESIDE CHAT: EMA PERSPECTIVE

8am – 9am PT / 11am – 12pm ET

Chair:

Paolo Morgese, Director, EU Market Access and Member Relations, Alliance for Regenerative Medicine (ARM)

Speakers:

Guido Rasi, M.D., Director General, European Medicines Agency (EMA)

THE EMERGENCE OF GENE EDITED CARs AND TCRs

Gene editing is a powerful tool used to improve the performance of a patient’s immune cells, and to facilitate the use of allogeneic adoptive cell therapies. This panel will discuss this rapidly developing area and the implications for the future of the treatment of cancer.

12pm – 1pm PT / 3pm – 4pm ET

Chair:

Charlie Albright, Ph.D., EVP and Chief Scientific Officer, Editas Medicine

Speakers:

Jason Fontenot, Ph.D., SVP, Cell Therapy and Head of Research, Sangamo Therapeutics
Andrew Schiermeier, Ph.D., EVP and Chief Operating Officer, Intellia Therapeutics

DURING BREAKS IN LIVE PROGRAMMING
Participate in 1×1 Partnering, Visit Virtual Exhibit Booths, View On-Demand Content

ON-DEMAND CONTENT
Available Beginning Thursday, October 15, 2020

ARM ACTION FOR EQUALITY TASK FORCE INITIATIVES

The Alliance for Regenerative Medicine (ARM) Action for Equality (AFE) Task Force is exploring what actions ARM and ARM members can take to eliminate racial inequality and address underrepresentation of Black Americans and minorities in the regenerative medicine sector. Our goal is to create a meaningful impact through various initiatives that will provide minorities with more opportunities, exposure, and mentorship in regenerative medicine. The Task Force aims to build a successful program that ARM and ARM Members are energized to pursue together.

Chair:

Emile Nuwaysir, Ph.D., President and CEO, BlueRock Therapeutics; Vice Chairman, Alliance for Regenerative Medicine (ARM)

Speakers:

Usman “Oz” Azam, M.D., President and CEO, Tmunity Therapeutics

Francesca Cook, Senior Director, Pricing and Market Access, REGENXBIO

Anshul Mangal, Founder, CEO and General Counsel, Project Farma

Courtney Utsey, Senior Manager HR Operations, Talent and Culture, Enzyvant

CELL AND GENE THERAPY PORTFOLIO STRATEGIES

This session will focus on how mid- to large-sized biotech and pharma companies develop a portfolio strategy designed to effectively progress cell and gene therapies. The panelists will discuss their respective approaches to building a robust pipeline.

Chair:

Arthur Tzianabos, Ph.D., President and CEO, Homology Medicines

Speakers:

Claudia Mitchell, Ph.D., SVP, Head of Portfolio Strategy, Astellas

Sanjaya Singh, Ph.D., VP and Global Head, Janssen BioTherapeutics, Janssen Research & Development, LLC, Janssen Pharmaceutical Companies of Johnson & Johnson

Anabella Villalobos, Ph.D., SVP, Biotherapeutics and Medicinal Sciences, Biogen

FDA WORKING WITH PATIENT GROUPS

This panel will discuss innovative ways patient groups can work with the FDA throughout the drug development and evaluation process. In this session, panelists will explore the use of real-world evidence, patient experience, and registries as replacements for more traditional methods of data collection.

Chair:

Annie Kennedy, Chief of Policy and Advocacy, Everylife Foundation for Rare Diseases

Speakers:

Ryan Fischer, SVP, Community Engagement, Parent Project Muscular Dystrophy

Monica Shah, M.D., VP and Head, Cell and Gene Therapy Center of Excellence, IQVIA

Kimberly Trant, Executive Director, Head of Patient Advocacy and Engagement, Audentes, an Astellas company

OVERCOMING CHALLENGES WITH THE DEVELOPMENT AND TRANSLATION OF A THERAPEUTIC FROM ACADEMIA TO INDUSTRY

Some of the most cutting-edge and innovative approaches to cell and gene therapy have originated from the academic setting. However, getting these treatments into the clinic can be especially challenging when the institution does not have the experience and infrastructure necessary to conduct preclinical studies, manufacture to GMP standards, and file regulatory submissions with the FDA. This panel of experts will discuss their lessons learned throughout the process and best approaches to moving a cell and gene therapeutic candidate from academia to commercialization.

Chair:

Shawn Roach, Ph.D., Specialist-Regulatory CMC, Halloran Consulting Group

Speakers:

Isabelle Riviere, Ph.D., Director, Cell Therapy and Cell Engineering Facility, Memorial Sloan Kettering Cancer Center

Anthony Ting, Ph.D., VP of Regenerative Medicine and Head of Cardiopulmonary Programs, Athersys

OPEN DAY
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