Dyne Therapeutics completed a positive pre-BLA meeting with FDA for z-rostudirsen and is on track to file its BLA in Q2 2026, targeting a Q1 2027 launch for the exon-51 DMD therapy. It’s the most advanced of at least five muscular dystrophy programs now in sight of the FDA.
Capricor’s deramiocel, after a surprise summer 2025 rejection, is back under FDA review with an action date of August 22. Wave Life Sciences confirmed a new drug application for exon-53-skipper WVE-N531 is on track for this year. Novartis paid $12 billion for Avidity last October and is waiting on Phase 3 data for del-desiran in DM1, expected in the second half; one analyst put this readout at “80% of the acquisition.”
The gene-therapy line is thornier. REGENXBIO’s Phase 3 RGX-202 data arrived May 14 showing 93% of patients hitting at least 10% microdystrophin expression at week 12, but the company reported a case of myocarditis and a case of liver injury. The stock fell 37%. REGENXBIO had previously guided toward a mid-2026 submission and now says only that RGX-202 is expected to launch in 2027.
The two SAEs didn’t stop the trial; both resolved within weeks. But they matter as precedent. Three patient deaths linked to Sarepta’s gene-therapy platform last year already put FDA in a scrutiny posture. Solid Biosciences’ SGT-003 has now dosed 46 Phase 1/2 patients with no liver injury, myocarditis, or thrombotic microangiopathy. In a field where safety comparisons are becoming the first cut at every FDA meeting, that clean record converts from a footnote to a filing advantage.
Deramiocel’s action date of August 22 is the first hard answer.
— Sarah Chen