Drug Profile

RP A701

Alternative Names: REN-001 - Rocket Pharmaceuticals; RP-A701

Latest Information Update: 15 Oct 2025

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At a glance

Originator Renovacor
Developer Rocket Pharmaceuticals
Class Cardiovascular therapies; Gene therapies
Mechanism of Action BAG3 protein replacements; Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

Highest Development Phases

Preclinical Dilated cardiomyopathy

Most Recent Events

22 Aug 2025 Rocket Pharmaceuticals plans a phase I trial for Dilated cardiomyopathy in USA (IV) in June 2026 (NCT07137338)
07 Aug 2025 RP A701 receives Fast Track designation for Dilated cardiomyopathy [Intracoronary] in USA
03 Jul 2025 Rocket Pharmaceuticals plans phase I trial in Dilated cardiomyopathy in USA

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