Nippon Shinyaku Co., Ltd.

With 48-week data from a Phase II open-label trial in Duchenne muscular dystrophy (DMD) showing an advancement in clinical benefit beyond what a 24-week readout showed last year, Wave Life Sciences sa

It’s a big year for Regenxbio. The company has a potential commercial launch of its gene therapy program for MPS II (Hunter syndrome), a Biologics License Application for its Duchenne muscular dystrop

One-third of all the regenerative medicine advanced therapy (RMAT) designations granted by the US Food and Drug Administration’s Center for Biologics Evaluation and Research were awarded in fiscal yea

While the FDA is facing a disrupted and uncharted future, drug development offers a few certainties, including that oncology and neuroscience still dominate the novel agents seeking approval. But the