Hematopoietic Stem Cell Gene Therapy

Overview

Ex-vivo gene therapy approach that corrects or adds functional genes into hematopoietic stem cells to treat blood disorders, immunodeficiencies, and neurodegenerative diseases; three products approved as of 2026 with durable benefits lasting 10-15+ years. Hematopoietic stem cell gene therapy has become a frontline treatment for rare genetic disorders including immunodeficiencies, neurodegenerative disorders, and blood disorders, because corrected stem cells can reconstitute the entire hematopoietic system and deliver enzymes locally to sites such as the CNS. Three hematopoietic stem cell gene therapy products had been approved as of 2026 from the San Raffaele/Telethon research program: treatments for ADA-SCID, metachromatic leukodystrophy, and Wiskott-Aldrich syndrome. (Segment 1 (00:00-05:00))