Agenda Details

The Cutting-Edge of Stem Cell Genetic Innovation

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Friday, May 31, 2024 08:00 AM - 09:00 AM

Room 211

From FIH to Clinical Acceleration

Concurrent Session

Chair

Bruce Levine, PhD, University of Pennsylvania, United States

Speakers

Alice Bertaina, MD, PhD, Stanford Medicine, United States, "Pioneering Ex Vivo Graft Manipulation: Advancing the Frontiers of Stem Cell Therapy"
Susan Prockop, MD, Boston Children's Hospital, United States, "TBC"

Panelist:

Jaap Jan Boelens, MD, PhD, Memorial Sloan Kettering Cancer Center, United States,

In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase gene into the T cells of two children with severe combined immunodeficiency (ADA- SCID). At the time, this was a dream to be able to successfully genetically correct an enzymatic defect to treat disease. In 2016, a version of this therapy using a different viral vector was approved in Europe. Several gene modified hematopoietic stem cell products are now approved to treat rare diseases Such as ADA-SCID, cerebral adrenal leukodystrophy, metachromatic leukodystrophy and hemoglobinopathies including sickle cell disease and beta thalassemia. One of these products is the first to use CRISPR gene editing. What were the scientific, technical, and clinical hurdles overcome to bring these therapies to regulatory approval? What are the next therapies on the horizon and how should we think about developing therapies and models for access to more patients?

Speakers/Moderators

Bruce Levine
Barbara and Edward Netter Professor in Cancer Gene Therapy
University of Pennsylvania
Chair

Susan Prockop
Associate Director, Clinical and Translational Research
Boston Children's Hospital
Speaker

Alice Bertaina
Professor of Pediatrics
Stanford University
Speaker

Jaap Jan Boelens
Chief Transplantation and Cellular Therapies
Memorial Sloan Kettering Cancer Center
Panelist

Session Details