Regenxbio says Duchenne gene therapy succeeded in clinical trial
statnews.com • • business
Key Points:
- Regenxbio announced that its experimental gene therapy for Duchenne muscular dystrophy generates high levels of a miniaturized muscle protein, a key target in the disease.
- The promising results position the company to submit its therapy for FDA approval, aiming for accelerated review.
- Regenxbio aims to develop a gene therapy that is both more effective and safer than Sarepta Therapeutics’ Elevidys, which has faced safety issues including two deaths from liver failure.
- CEO Curran Simpson expressed confidence that their data meets all criteria necessary for accelerated approval by regulatory authorities.