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

Rocket Pharmaceuticals (RCKT)Rocket PharmaceuticalsClinical readout

Initial three patients were treated safely; RP-A501 was well-tolerated with no TMA or capillary leak syndrome observed Rocket is actively engaging with the FDA to align on the path to pivotal trial completion; regulatory pathway update expected in the second half of 2026 Comprehensive Danon disease

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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 Pharmaceuticals2026-10-07 13:36 EST

Rocket Pharmaceuticals Highlights FDA-Aligned Pivotal Phase 2 Trial and Updated Clinical Findings for RP-A501 Following Danon Disease Investor Webinar

RCKTRocket Pharmaceuticals2026-10-06 20:12 EST

Rocket Pharmaceuticals Secures Strategic Credit Facility for Up to $150 Million from Hercules Capital

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 Pharmaceuticals2026-09-15 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

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2.48 USD
Market cap
315.57M USD
Exchange
NASDAQ
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Biopharma
Location
United States