The Gene Therapy Program is at the forefront of the science and translation of adeno-associated virus (AAV) vectors for the development of human and animal therapeutics.
This program represents our ambitious commitment to developing cutting-edge vector technologies. Despite tremendous progress in AAV research in the last 25 years and its continued clinical success, there is a significant opportunity to improve the performance of the AAV platform for gene therapy and to successfully apply it to a broader range of therapeutic applications.
Our diversified approach focuses on delivering advanced vectors for traditional gene therapy applications as well as vectors tailored specifically for genome editing. The design of these next-generation AAV vectors is guided by experimental results from studies on the basic biology of AAV that leverage our world-class resources in molecular, structural, cell, and computational biology.