Tag: RP-A601

Rocket Pharmaceuticals Announces FDA Clearance of IND for Clinical Trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy (ACM)

Coronary/Structural Heart Regulatory by 0 comments

First clinical gene therapy program for PKP2-ACM, a devastating inherited heart disease affecting approximately 50,000 people in the U.S. and EU Robust preclinical proof of concept studies showed RP-A601 decreased arrhythmias and increased survival in the PKP2 knockout mouse model Initiating […]

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Rocket Pharmaceuticals Expands Cardiac Gene Therapy Portfolio with Addition of RP-A601 for PKP2-ACM and Announces Positive Updated Phase 1 Data for RP-A501 in Danon Disease

Coronary/Structural Heart by 0 comments

RP-A601 for Arrhythmogenic Cardiomyopathy due to PKP2 pathogenic variants (PKP2-ACM) represents meaningful commercial opportunity with estimated prevalence of 50,000 adults and children in the U.S. and EU; IND filing anticipated in Q2 2023 In support of Phase 2 pivotal study, […]

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