Rocket points to Danon gene therapy’s safety after Phase II protocol change
Rocket Pharmaceuticals said its RP-A501 Danon disease gene therapy has safely treated three Phase II patients after an FDA-triggered protocol change. The FDA allowed relaunch of NCT06092034 in Aug 2025. Rocket reports no capillary leak syndrome, blood vessel damage, or other serious adverse events since dosing with a recalibrated dose plus rituximab, sirolimus, and corticosteroids.


