RP-A501 (Potential Future Indication)
PKP2-Associated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Pre-clinicalResearch/Discovery
Key Facts
Indication
PKP2-Associated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Phase
Pre-clinical
Status
Research/Discovery
Company
About Rocket Pharmaceuticals
Rocket Pharmaceuticals is a publicly traded gene therapy company dedicated to developing curative treatments for rare genetic disorders. Its most advanced program targets Danon Disease, a life-threatening cardiac condition, with a Phase 2 pivotal trial underway. The company leverages its expertise in gene therapy platforms to build a pipeline addressing significant unmet medical needs, guided by a patient-centric philosophy and core values of trust, generosity, curiosity, and elevation.
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