• Gene Therapy Research

    Gene Therapy

    Phone: 617-632-5064

    From the first attempt at modifying human DNA in the early 1980s… to the first clinical trials based on genetic engineering of the mid 1990s… to the first commercialization of a genetically engineered cell product in 2002… gene therapy has moved from being a pioneering research field to becoming one of the most technologically advanced and promising clinical realities for the treatment of a wide spectrum of diseases and tumors.

    Founded in 2010, our Gene Therapy Program has been at the forefront of the fight against rare genetic disorders affecting children. The success of our gene therapy clinical trials is due to our program’s unique combination of basic research and clinical care – and our ability to translate what we learn at the laboratory bench to the bedside care of the patients we treat.

    Our Gene Therapy Basic Research

    Through our research capabilities and partnerships, we strive to excel in every step of the gene therapy process. Our research includes:

    • basic studies on the causes of genetic diseases
    • gene/target identification
    • vector selection and development
    • preclinical validation and development
    • monitoring the efficacy and safety of genetic modification
    • development of new strategies for genetic engineering
    • identification of novel clinical applications

    Our Gene Therapy Translational Research Activities

    We work closely with industrial and translational academic partners, such as the Translab and Cell Manipulation cores at Dana-Farber Cancer Institute, to:

    • refine vector delivery delineate cell manufacturing requirements
    • conduct patient cell transduction and prepare gene corrected patient cells for infusion

    Learn more