Rocket Completes Danon Cohort Under Modified Protocol
The recalibrated Danon disease treatment used a dose of 3.8 × 10¹³ GC/kg.
Rocket Pharmaceuticals (RCKT), the gene-therapy developer, completed treatment of the initial patient cohort in its Phase 2 RP-A501 study for Danon disease under a modified protocol.
The milestone marked a shift from May, when Rocket had restarted the study and scheduled treatment of the three-patient cohort. By August, all three patients had received RP-A501, advancing the program toward the remainder of its 12-patient pivotal trial.
Rocket also reported the first safety results from the modified protocol. The patients tolerated the recalibrated dose and revised immunomodulatory regimen.
No thrombotic microangiopathy, capillary leak syndrome, or other significant safety concerns were observed in the cohort. Those findings provided the initial clinical evidence from the protocol changes adopted before treatment resumed.
Rocket is engaging with the Food and Drug Administration on dosing additional patients and completing the pivotal trial. The company continues to expect an update on the regulatory pathway in the second half of 2026, unchanged from its May guidance.
A comprehensive update on the Danon program also remains scheduled for the second half, leaving the next phase of enrollment dependent on the company’s discussions with regulators.