Biotech News

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 Pharmaceuticals2026-08-03 11:00 ESTClinical 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 Pharmaceuticals2026-08-03 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

RCKTRocket Pharmaceuticals2026-05-28 11:00 EST

Rocket Pharmaceuticals to Participate in Upcoming Investor Conferences

RCKTRocket Pharmaceuticals2026-05-07 20:00 EST

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

RCKTRocket Pharmaceuticals2026-04-28 11:00 EST

Rocket Pharmaceuticals Announces $180 Million Sale of Priority Review Voucher

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Close: 3.54 USD
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