Adeno-associated virus (AAV) vectors have emerged as a leading delivery vehicle for gene therapy in the human body (ie in vivo). These viral vectors can deliver the genes for therapeutic proteins (ie transgenes) to accessible tissues in the body, and AAV vectors have generally been safe and well-tolerated in hundreds (if not thousands) of patients. One AAV product is approved in the US (Luxturna, Spark Therapeutics), and a number of other AAV gene therapy products are in late-stage clinical development. However, these products utilize 1st and 2nd generation AAV vectors that have not been designed for the treatment of any specific diseases, and they have not been customized as targeted medicines.
In contrast, 4DMT customized 3rd generation AAV vectors, and the products engineered from them, are designed to unlock the full potential of gene therapy. Through 4DMT’s Therapeutic Vector Evolution, we are able to create customized vectors solutions to overcome the hurdles identified with 1st and 2nd generation AAV vectors.