US Food and Drug Administration Commissioner Marty Makary said he’s trying to persuade Vinay Prasad, the agency’s former head ...

Sarepta Therapeutics (NASDAQ:SRPT) has hired a lobbying firm with close ties to the Trump administration as regulatory ...

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

Extend transfection complex formation time by up to 3 hours, reduce complex volume by >50% and maintain high titers and full ...

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development ...

Leveraging Northway Biotech’s 20+ years of CDMO expertise, Diorasis Therapeutics is advancing AAV gene therapy for glaucoma ...

The Food & Drug Administration is investigating the death of an 8‑year‑old following Elevidys gene therapy for Duchenne ...

Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that have attracted attention from regulators ...

Michael Carducci, M.D., Professor, Johns Hopkins Kimmel Cancer Center Jan. 01, 2009 -- Question: What is the difference between gene therapy and immunotherapy, and can either or both be combined ...

The first gene therapy product approved by the Food and Drug Administration (a treatment for a form of leukemia, approved last summer) costs hundreds of thousands of dollars for each infusion.