Gene Therapy Program

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.

Entering a new decade

After steady improvements in technique, gene therapy represents a major shift in medicine, offering cures for conditions not previously treatable and for diseases often demanding heroic efforts. Since our founding a decade ago, we have treated more than 70 patients from 20 countries through 19 gene therapy clinical trials.

As the Gene Therapy Program enters its second decade, our team is optimistically embracing new gene-correction technologies such as gene editing and gene silencing. We feel fortunate to be at the forefront of this field with our patients and families providing motivation and purpose.