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.

What is in-vivo gene therapy?

When it's performed in vivo (inside the body), the vector carrying the therapeutic gene is injected directly into the patient's body. Although still relatively new and often considered experimental, gene therapy is a promising treatment option for a number of diseases that don’t respond well to other therapies, including inherited conditions — such as immunodeficiencies, metabolic disorders and hematologic disorders — and certain types of cancers.

What is ex-vivo gene therapy?

When it's performed ex vivo (outside the body), the vector with the corrected gene is introduced to a sample of a patient's cells in a laboratory setting, and then transplanted back into the patient.

 

 

 

 

Our unique approach to gene therapy

The Boston Children's Hospital Gene Therapy Program is one of the leading and largest gene therapy programs in the world. Founded in 2010, our success is a result of our Program’s research and clinical expertise, combined with our ability to translate laboratory discoveries into advances in patient care. 

What makes us unique is our dedicated infrastructure. We provide a core team that focuses on gene therapy as a treatment, collaborating closely with disease experts at two of the world's leading academic medical institutions — Boston Children’s Hospital and Dana-Farber Cancer Institute. Our efficient enrollment processes allow children to move smoothly and quickly from study enrollment to treatment, and our one-of-a-kind satellite-training module supports referring physicians.

Collaborating to treat rare and difficult-to-treat conditions

Together, along with the Dana-Farber/Boston Children’s Stem Cell Transplant, Blood Disorders and Hematologic Malignancy Centers, as well as academic and pharmaceutical partners in the U.S. and abroad, we have launched and participated in numerous clinical trials. In addition, we are one of the founding members of the Transatlantic Gene Therapy Consortium (TAGTC), a collaboration that seeks to combine the expertise from a number of centers treating rare diseases with different platforms.

Our success stories

David Williams, MD, chief scientific officer, with Patricio, a participant in the ALD gene therapy clinical trial. Find out how gene therapy is making a difference for our patients and families. 


Dr. David Williams, director of the Gene Therapy Program with Patricio, who participated in the ALD Gene Therapy Clinical Trial.

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.

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