Bionest

FDA Issues Set of Guidelines on Gene Therapy

Back in December, the U.S. Food and Drug Administration (FDA) granted its first approval for a gene therapy product, Spark Therapeutics’ Luxturna. According to the Alliance for Regenerative Medicine, as many as 50 more such treatments — addressing a diverse range of health problems from genetic, autoimmune, and heart diseases, to cancer and HIV/AIDS —...

Bionest

First Up For The New FDA RMAT Designation: Gene Therapeutics for Hemophilia

We recently wrote about the U.S. Food and Drug Administration’s (FDA) creation of a Regenerative Medicine Advanced Therapy (RMAT) designation in an effort to speed up the approval process for certain types of treatments, including gene therapies. The agency has specifically chosen gene therapy for hemophilia as its first focus, due to the considerable effort...

Bionest

Gene Therapy for Inherited Retinal Disease Achieves a Historic Milestone

This has been an amazing year in the world of drug development, with three new therapeutic classes marking historic gains. We first saw Novartis’ FDA approval for the first CAR-T immunotherapy. Then Alnylam’s RNAi treatment achieved Phase 3 success, the first such for that class of therapeutics. Most recently, Spark Therapeutics received a unanimous recommendation...

Bionest

Gene Therapy: Coming of Age Perhaps, but Who Will Pay?

New technologies tend to go through cycles of interest and attention. Initial excitement about the potential of a technological advance too often builds to over-blown hype, followed by a quick reversal of interest in the aftermath of a big disappointment. But if continued research later produces truly encouraging results, interest can build again as time...