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)
Latest Information Update: 20 Aug 2026
At a glance
- Drugs RP-A601 (Primary)
- Indications Cardiomyopathies
- Focus Adverse reactions
- Sponsors Rocket Pharmaceuticals
Most Recent Events
- 10 Aug 2026 According to a Rocket Pharmaceuticals media release, company is actively engaging with the FDA to seek alignment on the path to dosing additional patients and completing the pivotal Phase 2 trial and expects to provide an update on the regulatory pathway in the second half of 2026.
- 16 Apr 2026 Planned End Date changed from 1 Sep 2026 to 1 Sep 2029.
- 16 Apr 2026 Planned primary completion date changed from 1 Sep 2025 to 1 Sep 2028.