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