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.

The commitment and compassion with which we care for all children and families is matched only by the pioneering spirit of discovery and innovation that drives us to think differently, to find answers, and to build a better tomorrow for children everywhere.

Kevin B. Churchwell, President and CEO

Connect with Boston Children's Hospital