Rocket Pharmaceuticals Inc.
Clinical trials sponsored by Rocket Pharmaceuticals Inc., explained in plain language.
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First-in-Human gene therapy trial aims to fix faulty heart genes
Disease control Not yet recruitingThis is a very early study testing the safety of a new gene therapy for adults with a specific inherited form of heart muscle weakness (dilated cardiomyopathy). The therapy, called RP-A701, is given as a single intravenous dose and aims to deliver a working copy of a faulty gene …
Phase: PHASE1 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
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Scientists track rare genetic heart condition in 30 patients worldwide
Knowledge-focused Not yet recruitingThis study aims to learn how a specific inherited form of heart muscle disease (dilated cardiomyopathy) progresses over time in adults who have a harmful change in their BAG3 gene. Researchers will follow about 30 patients to document their symptoms, heart function, and quality o…
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 UTC