↵1 Present address: Akouos Therapeutics, Boston, Massachusetts 02210.
↵2 Present address: Asklepios BioPharmaceuticals, Philadelphia, Pennsylvania 19104; Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104.
Given the therapeutic potential of supplying a normal copy of a mutant gene to the correct target tissue, gene therapy holds extraordinary promise for the treatment of genetic disease. Like other novel classes of therapeutics however, gene therapies must overcome a range of clinical, regulatory, and manufacturing hurdles to reach regulatory approval. This paper reviews key aspects of clinical trial design, development, and evaluation of a novel primary end point, and regulatory interactions that resulted in the first approval by the U.S. Food and Drug Administration (FDA) of an adeno-associated virus (AAV) gene therapy product.
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