ScripAtsena Therapeutics thinks it can be a success story in the gene therapy field, where some companies have recently stumbled, with the closure of a funding round that it plans to use especially for its
ScripWith 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
In VivoIt’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
Pink SheetOne-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