Gene Therapy Program

Gene Therapy Program | FDA-Approved Gene Therapies

Boston Children's Hospital offers two gene therapy treatments approved by the U.S. Food and Drug Administration (FDA) — Zolgensma for spinal muscular atrophy (SMA) and KYMRIAH CAR T-cell therapy for relapsed acute lymphoblastic leukemia (ALL).

Gene therapy for SMA (Zolgensma)

We are one of the first pediatric hospitals in the nation to offer a gene therapy treatment for SMA. Zolgensma, formerly AVXS-101, prevents further muscle degeneration by replacing the defective or missing gene (SMN1) that causes SMA. Learn more about SMA gene therapy.

CAR T-cell therapy for relapsed ALL

The Dana-Farber/Boston Children's Cancer and Blood Disorders Center is a certified provider for the first FDA-approved CAR T-cell therapy called KYMRIAH for patients up to 25 years of age with relapsed or treatment-resistant B-cell acute lymphoblastic leukemia (ALL). Learn more about CAR T-cell therapy.

We are proud to serve a broad and diverse patient population that often comes to us for answers they can't find anywhere else. This top ranking is a credit and a tribute to our extraordinary and deeply committed clinicians, researchers and so many others who dedicate every day to improving and advancing the health and well-being of all children.

Sandra L. Fenwick, CEO

Connect with Boston Children's Hospital