RCKT
Rocket aligns with FDA on Danon disease pivotal trial
The FDA confirmed the pivotal study design, protocols for continued dosing, and co-primary endpoints for RP-A501, including a 12-patient efficacy population.
RCKT news
Latest 4 stories · newest Sep 15, 2026
RCKT
Rocket Q2 Increases Cash and Clears Key Safety Observations
Rocket reported $283.7M in cash and cleared safety observations for its Danon disease candidate RP-A501, with management stating the achievements strengthen the company's position.
RCKT
Rocket Reports Positive Clinical Safety Data from Three RP-A501 Patients
Rocket Pharmaceuticals reported that its RP-A501 gene therapy was well-tolerated with no significant safety concerns observed in three treated patients, leading the company to engage actively with the FDA on the next steps for the Phase 2 pivotal trial.
RCKT
Rocket Pharmaceuticals closes $180M sale of Rare Pediatric Disease Priority Review Voucher
Rocket Pharmaceuticals completed the sale of its Rare Pediatric Disease Priority Review Voucher for $180 million in gross proceeds, increasing pro forma cash to approximately $322.6 million. The non-dilutive funding is expected to support operations into Q2 2028 and strengthens the company's financi…
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