With several first-in-class gene therapies now approved, distinct new challenges are now coming into focus. This article provides a high-level overview of the gene therapy pipeline for non-oncology rare diseases and discuss key issues impacting clinical development.
This has been a tremendous year for cell and gene therapy. In October, the FDA approved Yescarta, a chimeric antigen receptor (CAR) T-cell therapy developed by Kite Pharmaceuticals and recently acquired by Gilead Sciences, becoming only the second gene therapy to be approved in the U.S. This comes less than two months after the FDA approved Novartis’ CAR T-cell therapy, Kymriah.