Late-stage biotechnology company Rocket Pharmaceuticals Inc (NASDAQ:RCKT) announced on Monday that the US Food and Drug Administration (FDA) has cleared its Investigational New Drug application for RP-A701, a gene therapy targeting BAG3-associated dilated cardiomyopathy (BAG3-DCM).

RP-A701 is a first-in-class, AAVrh.74-based gene therapy designed to treat BAG3-DCM, a rare and inherited heart condition marked by progressive heart failure, significant morbidity and early mortality.

Rocket Pharmaceuticals plans to initiate a Phase 1 clinical trial in the United States, with preparations already underway to begin dosing the first patient.

The Phase 1 trial will be a multi-centre, dose-escalation study evaluating the safety, biological activity and early efficacy of RP-A701 in adult patients with advanced BAG3-DCM and implantable cardioverter defibrillators. Endpoints will include BAG3 protein expression, cardiac biomarker levels and other clinical indicators of disease progression.

This programme adds to Rocket's existing clinical portfolio, which spans hypertrophic, dilated and arrhythmogenic genetic cardiomyopathies.