Aviara Salon A Workshops

AVIARA SALON A LIVESTREAM AGENDA

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

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

8:45am | LIVESTREAM ENDS FOR THE DAY IN AVIARA SALON A

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

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

8:45am | LIVESTREAM ENDS IN AVIARA SALON A

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