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Rocket Pharmaceuticals Announces FDA Alignment on Path to Complete Pivotal Phase 2 Trial of RP-A501 in Danon Disease

Rocket Pharmaceuticals (RCKT)Rocket PharmaceuticalsFDA / regulatory

FDA confirms pivotal efficacy population of 12 patients, including the first three treated under the modified protocol FDA review of initial safety data supports continued enrollment and dosing under modified protocol FDA confirms established 12-month co-primary endpoints intended to support

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Rocket Pharmaceuticals

Gene Therapies for Rare Disease Rocket Pharmaceuticals develops gene therapies for rare hematologic and cardiovascular disorders using lentiviral and adeno-associated-virus vectors. Commercial Hematology Franchise KRESLADI is an ex vivo lentiviral gene therapy approved for severe leukocyte adhesion deficiency type I. It represents...

Recent company news

RCKTRocket Pharmaceuticals2026-09-15 11:00 EST

Rocket Pharmaceuticals Announces FDA Alignment on Path to Complete Pivotal Phase 2 Trial of RP-A501 in Danon Disease

RCKTRocket Pharmaceuticals2026-09-03 11:00 EST

Rocket Pharmaceuticals to Participate in Upcoming Investor Conferences

RCKTRocket Pharmaceuticals2026-08-10 20:00 EST

Rocket Pharmaceuticals Reports Second Quarter 2026 Financial Results and Highlights Recent Progress

RCKTRocket Pharmaceuticals2026-08-03 11:00 EST

Rocket Pharmaceuticals Announces Positive Clinical Safety Update from Initial Three Patients Treated with RP-A501 Under Modified Phase 2 Protocol for Danon Disease

RCKTRocket Pharmaceuticals2026-06-12 11:00 EST

Rocket Pharmaceuticals Announces Closing of Sale of Rare Pediatric Disease Priority Review Voucher for $180 Million

Stock data

Close
3.50 USD
Market cap
315.57M USD
Exchange
NASDAQ
Sector
Biopharma
Location
United States