Adeno-Associated Virus: Enabling Genomic Medicine

AAV is the leading vector for in vivo gene therapy. Recent landmark regulatory approvals of AAV-based therapeutics have only bolstered enthusiasm as well as the continued investment for the development of AAV therapeutics to address diseases with high unmet medical need. Since the first rAAV-based therapy approved by the FDA, application of gene therapies utilizing rAAV vectors has exploded and active clinical programs are being fast-tracked for clinical approval for congestive heart failure, hemophilia A and B, retinal disease, X-linked myotubular myopathy, and other indications.

Take an in-depth look at some of the factors to consider with AAV research and development:

• The History of AAV
• Clinical Applications of AAV
• AAV Biology: Structure and Function of AAV Vectors
• Manufacturing and Quantification of Fully Potent AAV