Regenxbio reported a clean safety profile and functional improvement signals for its Duchenne muscular dystrophy (DMD) gene therapy as pivotal data approach, reinforcing the program’s regulatory runway. Company statements emphasized tolerability in the latest cohorts and incremental functional gains consistent with earlier-phase results. Separately, the FDA has resumed review of Capricor Therapeutics’ previously rejected DMD cell therapy after the company supplied additional clinical data, signaling agency willingness to re-evaluate cell‑based approaches with new evidence. The restart follows an initial regulatory denial and company resubmission that included updated safety and efficacy datasets. Taken together, the developments reflect active regulatory scrutiny of advanced DMD modalities—both gene and cell therapies—and underscore how fresh safety and follow-up data can reopen regulatory conversations in rare‑disease spaces.