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.
Through our research capabilities and
partnerships, we strive to excel in every step of the gene therapy process. Our
We work closely with industrial and
translational academic partners, such as the Translab and Cell Manipulation cores at Dana-Farber
Cancer Institute, to: