• Gene Therapy Clinical Trials

    Dana-Farber/Boston Children’s is a global leader in pediatric gene therapy clinical research. Launched by Dana-Farber/Boston Children’s President David Williams, MD, and now under the direction of Alessandra Biffi, MD, our program conducts preclinical and translational research on gene therapy techniques for a number of diseases.

    Collaborating 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 United States and abroad, the program has launched and participated in numerous gene therapy clinical trials.

    As a founding member of the Trans Atlantic Gene Therapy Consortium, we collaborate with partners in the United Kingdom, France, and Italy to conduct international trials of gene therapy for a growing variety of genetic immunodeficiencies. We also offer clinical trials for metabolic disorders, hematologic malignancies, and non-malignant blood disorders. Gene therapy trials for children with other diseases are under development. Our portfolio of clinical trials includes both trials initiated by our own scientists and trials sponsored by pharmaceutical partners.

    Our Gene Therapy Trials Open for Accrual

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