Rocket Pharma ((RCKT)) announced an update on their ongoing clinical study.
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Rocket Pharmaceuticals (RCKT) is initiating a Phase 1 clinical trial titled A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human BCL2-associated Athanogene 3 (BAG3) Gene Coding Sequence (RP-A701) in Subjects With Dilated Cardiomyopathy Arising From Pathogenic BAG3 Variants (BAG3-DCM). The study aims to assess the safety, tolerability, and preliminary efficacy of RP-A701 in patients with BAG3 Dilated Cardiomyopathy, a condition with significant unmet medical needs.
The intervention being tested is RP-A701, a genetic therapy delivered through a recombinant viral vector. This therapy involves a single intravenous dose intended to address the genetic basis of BAG3 Dilated Cardiomyopathy by introducing a functional BAG3 gene.
The study is designed as an open-label, dose-escalation trial with a sequential intervention model. Participants will receive a single ascending dose of RP-A701, with no masking involved, focusing on treatment as the primary purpose.
The trial is set to begin on July 25, 2025, with the last update submitted on August 20, 2025. These dates mark the initiation of recruitment and the latest information available, respectively, indicating the trial’s progression towards commencement.
This update could influence Rocket Pharmaceuticals’ stock performance positively by showcasing innovation in genetic therapies, potentially boosting investor confidence. However, the impact will also depend on competitive developments within the gene therapy sector.
The study is ongoing, with further details available on the ClinicalTrials portal.