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A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)

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Trial Profile

A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 06 Aug 2025

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At a glance

  • Drugs RP-A601 (Primary)
  • Indications Cardiomyopathies
  • Focus Adverse reactions
  • Sponsors Rocket Pharmaceuticals

Most Recent Events

  • 17 Jul 2025 According to a Rocket Pharmaceuticals media release, announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A601, RMAT designation was granted based on positive safety and efficacy data from the Phase 1 RP-A601 clinical trial.
  • 15 May 2025 According to a Rocket Pharmaceuticals media release, results of this study were presented at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) and will be discussed on a company webinar today at 4:30 p.m. ET.
  • 15 May 2025 Preliminary results presented in a Rocket Pharmaceuticals Media Release.

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