Agenda

7:15am – 8:45am | Cognate Bioservices Ballroom
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)

DOING BUSINESS IN JAPAN WORKSHOP
An Evolving Market – Innovations for Industrialization Platforms and the Latest Rise of Commercialization

Chair:
Masayuki Nomura, Ph.D., GM, Business Development and Licensing Department, Healthcare R&D Center, Corporate R&D, Asahi Kasei Corporation

7:15am – 7:20am | Welcome Remarks
Speaker:
Tetsuya Tanaka, Director, Bio-Industry Division, Ministry of Economy, Trade, and Industry, The Government of Japan

7:20am – 7:35am | Evolving Market and Industrialization Platform for Innovative Regenerative Medicine in Japan
Speaker:
Yoshitsugu Shitaka, Ph.D., President, Astellas Institute for Regenerative Medicine (AIRM)

7:35am – 8:05am | Update on Regulatory Landscape of Regenerative Medicine
Speaker:
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)

8:05am – 8:25am | Japan’s First Gene Therapeutic Drug
Speaker:
Ei Yamada, Ph.D., President and CEO, AnGes

8:25am – 8:45am | CAR-T Therapy Development in Japan
Speaker:
Yoshie Tsurumaki, Japan Head, SCM CAR-T, CAR-T Supply Chain Management Group, Cell and Gene Franchise, Oncology Division, Novartis Pharma K.K.

7:15am – 8:45am | Aviara Salon A
Sponsored by Aldevron

GENE-BASED MEDICINE DEVELOPMENT WORKSHOP

7:15am – 8:00am | Session 1: It’s Never Too Soon – Considerations for Gene Therapy Manufacturing in the Early Stages of Development
The success of gene therapy has resulted in significant benefits for patients and tremendous growth in the number of programs in development. Many programs have been granted accelerated designations (RMAT, etc.) and have the potential to reach patients far faster than the traditional drug development timeline. Given the accelerated development and the complexity of gene therapy manufacturing, strategies to produce the therapy and manage the supply chain must be considered very early in the development process. This session will discuss how programs with limited time and resources can address manufacturing challenges and review some lessons learned from those in the field.

Chair:
Gregory MacMichael, Ph.D., President, CMC BioServices
Speakers:
Michelle Berg, President, GMP Nucleic Acids, Aldevron
Jeffrey Castelli, Ph.D., Chief Portfolio Officer and Head of Gene Therapy, Amicus Therapeutics
Maritza McIntyre, Ph.D., President, Advanced Therapies Partners
Susan Nichols, CEO, Falcon Therapeutics
Fraser Wright, Ph.D., Professor of Pediatrics, Center for Definitive and Curative Medicine (CDCM), Stanford Medicine

8:00am – 8:45am | Session 2: Customized Solutions for Genetic Medicines – Navigating the GMP Supplier Landscape
For most translational research units, bringing a genetic medicine to the clinic requires the support of several outside partners. Researchers need to procure custom GMP raw materials (plasmid DNA, guide RNA, Cas9 Nuclease) as well as outsourced GMP services (cell therapy manufacturing). All of these services carry a cost and significant timelines. From the end-user perspective, it is necessary to balance these costs and timelines in order to enter the clinic and begin treating patients on time. From the supplier perspective, it is important to innovate and provide clients with flexible products and services. The purpose of this panel is to bring these two perspectives together to inform the audience of best practices in outsourcing to avoid delays and best provide support to translational clients.

Chair:
Alan Trounson, Ph.D., CEO, Cartherics
Speakers:
Delfi Krishna, Ph.D., Director, Cell and Gene Therapy Platform, R&D Strategy, Portfolio and Operations, GSK
Annalisa Lattanzi, Ph.D., Translational Program Manager for Therapeutic Genome Editing, Stanford University School of Medicine – Pediatrics
Max Sellman, Client Relations Manager, Aldevron
Keith Thompson, CEO, Cell and Gene Therapy Catapult
Chy-Anh Tran, Director, Operations and cGMP Facilities, Stanford Laboratory for Cell and Gene Medicine

7:15am – 8:45am | Aviara Salon B
Sponsored by IQVIA

DEVELOPING A ROBUST EVIDENCE PACKAGE TO PREPARE FOR MARKET ENTRY WORKSHOP
When you think of bringing a new therapy to market, payers, regulators, providers, and other stakeholders are requiring robust information on effectiveness as well as the overall impact of use to understand the true value for patients and impact on the wider healthcare system. Understanding what stakeholders are looking for as part of their review process is crucial to successfully develop and communicate your value message. Careful planning during clinical development is key to develop an evidence package that is useful for regulatory filing as well as discussions with payers and providers. This panel will discuss elements and generation of this evidence package.

Welcome Remarks:
George Smith, Ph.D., Senior Director, Cell and Gene Therapy Center, IQVIA
Chair:
Adrian McKemey, Ph.D., SVP, Consulting Services, IQVIA
Speakers:
Stewart Abbot, Ph.D., Chief Operating and Scientific Officer, Adicet Bio
John Doyle, Dr.P.H., VP, Global Healthcare Innovation Lead, Pfizer
Manuel Duenas, VP, Market Access, Atara Biotherapeutics
Felix Hsu, SVP and Global Head of Advanced Therapies, WuXi AppTec
Chris McClain, VP, Sales and New Business Development, Be The Match BioTherapies
Aiman Shalabi, VP, R&D, Cell and Gene Therapies, GlaxoSmithKline

7:15am – 8:45am | Cognate Bioservices Ballroom
Sponsored by Thermo Fisher Scientific

IDENTIFYING PITFALLS WITHIN GENE THERAPY DEVELOPMENT AND HOW TO OVERCOME THEM WORKSHOP
This session will cover recent advancements in gene therapy with insights from gene therapy developers and CDMOs on common pitfalls and how to avoid and overcome them. Join this interactive session to learn about their strategies and lessons learned. Topics discussed will range from insourcing vs. outsourcing, building reliable and scalable processes, and their vision for the future of gene therapy.

Chair:
Amy Butler, Ph.D., VP and GM Cell Biology, Thermo Fisher Scientific
Speakers:
Timothy Miller, Ph.D., Co-Founder, President, and Chief Scientific Officer, Abeona Therapeutics
Nicole O’Brien, Ph.D., Senior Director, Technical Program Design, Viral Vector Services, Thermo Fisher Scientific
Robert Pietrusko, Pharm.D., SVP, Regulatory Affairs and Quality Assurance, Voyager Therapeutics
Jeff Walsh, Chief Strategy Officer, bluebird bio
Ran Zheng, Chief Technical Officer, Orchard Therapeutics

7:15am – 8:45am | Aviara Salon A
Sponsored by Evidera PPD

REAL WORLD EVIDENCE TO DRIVE ACCEPTANCE AND UPTAKE OF CELL AND GENE THERAPY: LESSONS AND BEST PRACTICES WORKSHOP
Cell and gene therapies offer transformative or curative potential, but you must prove it. Real world evidence (RWE) is a foundational tool to address uncertainty around asset value across the product lifecycle, from early development to expanded access. This is especially true in the case of therapies whose value proposition hinges on magnitude and duration of effect. Global regulatory agencies and payers are also weighing in and shifting RWE from “nice to know” to “got to have”, solidifying RWE as an essential to new cell and gene therapy strategy. Despite this momentum in RWE use, methods, stakeholder expectations, and “what good looks like” are still evolving. This workshop will discuss what RWE is, when and how to use it, and consider evolving lessons and best practices for cell and gene therapies.

Chair:
Eric Faulkner, VP, Real World Value and Strategy; Executive Director, Precision and Transformative Medicine Center of Excellence, Evidera
Speakers:
Ramesh Arjunji, Ph.D., Senior Director, Health Economics and Outcomes, AveXis
Jane F. Barlow, M.D., Senior Advisor, MIT Center for BioMedical Innovation/NEWDIGS
Francesca Cook, Senior Director, Pricing and Market Access, REGENXBIO
Bhash Parasuraman, Ph.D., VP, Patient and Health Impact, Rare Disease Business Unit, Pfizer

7:15am – 8:45am | Aviara Salon B
Sponsored by World Courier

ALIGNING CLINICAL, MANUFACTURING, AND LOGISTICS PLATFORMS TO TREAT PATIENTS AT SCALE WORKSHOP
For advanced therapies to become a true industry, developers need to align the three platforms of manufacturing (closed and automated process), clinical (safe and efficacious) and logistics (seamless connection to patients). Without this alignment, companies risk creating a life changing therapy that can’t be made, or manufacturing a therapy that can’t be delivered. This workshop will look to understand the challenges in coordinating development across these platforms, providing lessons learned to help companies create therapies that can easily scale into globally viable commercial therapies.

7:15am – 7:25am | Welcome Remarks
Chair:
Simon Ellison, Cell and Gene Therapy Service Director, World Courier

7:25am – 7:35am | Presentation 1
Speaker:
Phil Vanek, Ph.D., GM, Cell and Gene Therapy Strategy, GE Healthcare

7:35am – 7:45am | Presentation 2
Speaker:
Dominic Clarke, Ph.D., Global Head of Cell Therapy, HemaCare; Co-Chair, Process and Product Development Committee, International Society for Cell and Gene Therapies (ISCT)

7:45am – 7:55am | Presentation 3
Speaker:
Jacqueline Barry, Ph.D., Chief Clinical Officer, Cell and Gene Therapy Catapult

7:55am – 8:45am | Moderated Discussion with All Speakers