In 2017, the FDA approved the first gene therapy products in the United States, signaling a new era for gene therapy andreinforcing the continued momentum and the potential of this breakthrough approach in treating a wide range of diseases. Gene therapy research at Boston Children\’s Hospital spans multiple disease areas and integrates basic research with translational and clinical endeavors.

Boston Children\’s investigators are discovering and validating new targets, designing innovative delivery tools and vectors, conducting clinical studies and providing gene therapy products to patients. Several startups focused in bringing new gene therapy products to market have spun out of Boston Children’s. Our Gene Therapy Program, established in 2010, has become a world leader in pediatric gene therapy clinical research.

We invite you to learn more about Boston Children’s research in gene therapy and to discuss how to advance new treatments through powerful industry/academic collaborations.