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RP-A701 · 1 trial · 1 indication
Number of participants with Adverse Events following a single IV dose of RP-A701
Number of participants with Serious Adverse Events (SAE) following a single IV dose of RP-A701
Number of participants with Dose Limiting Toxicities (DLT) following a single IV dose of RP-A701
| Arm | Type | Description |
|---|---|---|
| Single ascending dose of RP-A701 in up to 2 consecutive cohorts | EXPERIMENTAL | Participants will receive a single intravenous dose of RP-A701 on Day 0 and will be followed for up to two years |
| Name | Type | Description |
|---|---|---|
| RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3) | GENETIC | One-time treatment with a single ascending dose |
Inclusion Criteria: Subjects are eligible for inclusion into the study only if all the following criteria apply: 1. Male or female between 18 and 65 years of age at the time of signing the informed consent 2. Capable of and willing to provide signed informed consent 3. Clinical diagnosis of DCM de...
RP-A701 is an investigational gene therapy being developed for Dilated Cardiomyopathy (DCM), specifically for patients with BAG3-associated dilated cardiomyopathy. It is designed to deliver the BAG3 transgene to patients with this genetic form of DCM. It is not yet approved for any indication and remains in clinical development.
RP-A701 targets the BCL2-associated Athanogene 3 (BAG3) gene. It is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the BAG3 transgene. By delivering this transgene, the therapy aims to address the underlying genetic cause of BAG3 dilated cardiomyopathy.
RP-A701 is being developed by Rocket Pharmaceuticals, Inc., which trades under the ticker RCKT. The company is sponsoring the clinical development of this gene therapy for BAG3 dilated cardiomyopathy.
RP-A701 is in Phase 1 clinical development. It is an investigational therapy and has not been approved by the FDA or any regulatory agency. The Phase 1 trial is currently recruiting participants.
RP-A701 is being evaluated in a Phase 1 clinical trial, NCT07137338, titled "A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy." The trial is recruiting in the United States and has an estimated enrollment of 8 participants.
RP-A701 is also known as a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene BCL2-associated Athanogene 3 (BAG3). These descriptions refer to the same investigational gene therapy developed by Rocket Pharmaceuticals.