Although AAV gene therapy is new to CAH, it’s not new. A number of AAV gene therapies have been approved by the FDA.
Gene therapies have also been studied extensively in clinical trials for adults with hemophilia.
In total, gene therapies have been used to treat more than 3,500 people around the world, and more patients are being treated with gene therapy every day. There are currently 100+ active AAV gene therapy trials for a variety of diseases.
Long-term safety information for both the approved gene therapies and those still in development is being collected continuously. The American Society of Gene and Cell Therapy has additional information about gene therapies on its website (asgct.org/genetherapy101/).