Beacon Gene Therapy – Clinical Holds in the AAV Space

Beacon Gene Therapy captured over 1660 assets, around 50% leverage on Adeno-Associated Viruses (AAVs), indicating that the space relies heavily on these vectors. While AAVs are less immunogenic than other viruses, this risk is not zero. A joint adverse event associated with AAV therapies is hepatotoxicity.

Despite the drawbacks associated with AAVs, an increase in new trials has not come without challenges in the AAV space. There has also been an increase in clinical holds on assets. In this piece, we will explore why trials evaluating these vectors have seen a large volume of clinical trial holds and the impact of these holds on the discontinuation of drug development.