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.