Sarepta Therapeutics is refusing to pull its gene therapy Elevidys (delandistrogene moxeparvovec), despite a request from the FDA.

Industry experts have provided insight into how oncology clinical trials can be designed for success from the outset and rendered as patient-centric as possible.

Ocugen has dosed the first subject in its Phase II/III GARDian3 trial of modifier gene therapy candidate, OCU410ST.