ARTICLE | Product Development
AAVs on the brink: Charting the clinical landscape of in vivo gene therapies
BioCentury maps the world of clinical AAV gene therapies to the companies testing them and their in vivo indications
May 18, 2019 2:23 AM UTC
Gene therapy is finally teetering on the edge of becoming a drug development mainstay, with at least nine AAV-based therapies in Phase III or II/III testing. An analysis of the AAV therapies in the clinic reveals a growing diversity of vector subtypes being used in vivo, engineering strategies gaining traction, and an expansion of the modality beyond monogenic diseases.
The 2017 approval of Luxturna voretigene neparvovec-rzyl from Spark Therapeutics Inc. as the first in vivo gene therapy broke the modality’s twenty-year logjam. ...