Rocket Pharmaceuticals (RCKT) said Monday that, while the Phase 2 trial for its experimental RP-L301 gene therapy is ready for patient enrollment, it will not begin until next year as the company is currently directing resources toward other programs.
RP-L301 is being studied for the treatment of Pyruvate Kinase Deficiency, a red blood cell disorder that causes mild to life-threatening anemia.
The company, meanwhile, said its RP-A501 gene therapy is generally well-tolerated in a phase 1 trial to treat male patients with Danon Disease, a genetic disorder characterized by the weakening of the heart muscle.
Rocket said the adverse events after patient dosing were mostly mild or moderate and not related to RP-A501 and non-serious.
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