Session Chairs: Francois Gianelli, TreeFrog Therapeutics, Deep Shah, Sana Biotechnology, and Christopher Storbeck, Health Canada

The field of Cell and Gene Therapy has witnessed tremendous growth in recent years with the approval of many CAR T cell and AAV-based Gene therapy products. Stem cell therapeutic products on the other hand, with their tremendous potential for treatment of many diseases due to their inherent properties of self renewal, differentiation potential and tissue regeneration and repair, have been lagging behind in their development. This may be in part be due to numerous challenges regarding their therapeutic use in the clinic, including the risk of immunological rejection, tumorigenesis, and achieving precise control of their characteristics.

The recent approval of a stem cell-based Gene editing product for Sickle Cell Disease and Beta Thalassemia may signal the beginnings of an upward trend for advancement of stem cell therapeutic products. This trend will be explored in this session with a look to promising products emerging in the stem cell therapeutic space.

Session Speakers:

Ensuring Product Comparability in Cell Therapy: A Risk-Based Approach to Managing Multifactorial Changes

Patty Sachamitr, BlueRock Therapeutics LP

Transparency-Driven Partnerships for Shared Success – iPSC Journey

Pedro Castanho Vaz, Catalent, Inc

Introduction to CASGEVY®, The First CRISPR-Cas9 Based Commercially Approved Therapy For SCD and TDT

Irina Kadiyala, Vertex Pharmaceuticals Incorporated

Additional Panelist:

Kathleen Francissen, Genentech, a Member of the Roche Group

Francois Gianelli

Appearances