Hemophilia and Gene Therapy Basics

ASGCT and the Hemophilia Federation of America (HFA) partnered to produce a series of patient education webinars. Mark Kay, PhD of Stanford University School of Medicine explains the basics of gene therapy to the hemophilia patient community and how gene therapy may be used to treat hemophilia.

All Webinars

Cytokine Release Syndrome

Marco Davila, MD, PhD, of the H. Lee Moffitt Cancer Center and Research Institute, and Marcela Maus, MD, PhD, of the Massachusetts General Hospital Cancer Center and Harvard University, explained the diagnostic criteria, various grading schemes, and the risks of cytokine release syndrome.

Back to Basics Handling CART Cell Products

Dr. Isabelle Riviere and Dr. Xiuyan Wang of the Michael G. Harris Cell Therapy and Cell Engineering Facility at Memorial Sloan Kettering Cancer Center addressed CAR T cell products manufacturing and regulatory considerations as well as handling procedures in the context of academic centers.

Gene Therapy Basics

Dr. Mark Kay of Stanford University School of Medicine explains basic concepts in hemophilia gene therapy. This webinar is meant to help patients understand how gene therapy might impact their treatment and possible advancement to come.

Gene Therapy in Bleeding Disorders

Dr. Kay discusses the latest advances in gene therapy for bleeding disorders, specifically for hemophilia. He provides a brief history of previous human gene therapy trials for hemophilia and discusses the differences between trials. Dr. Kay also addresses potential challenges of gene therapy for hemophilia.


ASGCT Policy Summit

November 4 – 6 | Washington D.C.