[PERSPECTIVES] Trial by "Firsts": Clinical Trial Design and Regulatory Considerations in the Development and Approval of the First AAV Gene Therapy Product in the United States

Kathleen Z. Reape1 and Katherine A. High2 Spark Therapeutics, Philadelphia, Pennsylvania 19104, USA Correspondence: kzreapegmail.com; khighaskbio.com

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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