Agenda

7:15am – 8:45am
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., General Manager, 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, Inc.

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
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.

Speakers:
Delfi Krishna, Ph.D., Director, Cell and Gene Therapy Platform, R&D Strategy, Portfolio and Operations, GSK
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
Sponsored by IQVIA

DEVELOPING A ROBUST EVIDENCE PACKAGE WORKSHOP

7:15am – 8:00am | Session 1: Getting Prepared – Knowing What is Expected as Part of a Robust Evidence Package
When you think of bringing a new therapy to market, payers, providers, and other stakeholders are requiring robust information on effectiveness and real product use to understand the true value for patients and 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.

Chair:
Adrian McKemey, SVP Consulting Services, IQVIA

8:00 – 8:45am | Session 2: Getting it Done – Developing a Robust Evidence Package
Having insights on expectations and requirements from main stakeholders is the initial step to develop an action plan to solve the challenges at hand. This session will show you how to get the data needed, initiatives to put in place, and how to perform the necessary analytics to demonstrate results that are understood by your target audience.

Chair:
George Smith, Senior Director, Cell and Gene Therapy Center, IQVIA

7:15am – 8:45am
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, VP and General Manager Cell Biology, Thermo Fisher Scientific
Speakers:
Nicole O’Brien, Ph.D., Senior Director, Commercial Operations, Viral Vector Services, Brammer Bio
Robert Pietrusko, SVP, Regulatory and Q&A, Voyager Therapeutics

7:15am – 8:45am
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 and Executive Director, Precision and Transformative Medicine Center of Excellence, Evidera
Speakers:
Jane Barlow, M.D., Senior Advisor, FoCUS Project, MIT Center for BioMedical Innovation/NEWDIGS
John Doyle, Dr.P.H., VP, Global Healthcare Innovation Lead, Pfizer

7:15am – 8:45am
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., General Manager, Cell and Gene Therapy Strategy, GE Healthcare

7:35am – 7:45am | Presentation 2
Speaker:
Dominic Clark, Ph.D., Global Head of Cell Therapy, HemaCare; Co-Chair, ISCT Process and Product Development Committee

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