Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RP-A601 · 1 trial · 1 indication
Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)
| Arm | Type | Description |
|---|---|---|
| RP-A601 | EXPERIMENTAL | Single ascending dose of RP-A601 in 2 consecutive cohorts |
| Name | Type | Description |
|---|---|---|
| RP-A601 | GENETIC | RP-A601 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, human plakophilin 2 (PKP2), transcript variant 2a (PKP2a) |
Key Inclusion Criteria: 1. Male or female ≥18 years at the time of signing the informed consent 2. Capable and willing to provide signed informed consent 3. Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC) 4. Documentation of a pathogenic or likely pathogenic trunc...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Lexeo Therapeutics, Inc. | LXEO | 3 | PHASE1 | LX2020 |
RP-A601 is an investigational gene therapy being developed for PKP2 variant-mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM), a cardiovascular condition. It is designed to address the genetic cause of the disease by delivering a functional PKP2 gene. RP-A601 is currently in Phase 1 clinical development and has not been approved by the FDA.
RP-A601 targets the PKP2 gene, which encodes plakophilin-2, a protein essential for heart muscle cell adhesion and electrical signaling. By delivering a functional copy of the PKP2 gene, RP-A601 aims to correct the underlying genetic defect in patients with PKP2 variant-mediated Arrhythmogenic Cardiomyopathy. This gene therapy approach is being evaluated in a Phase 1 clinical trial.
RP-A601 is being developed by Rocket Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker RCKT. The company is conducting a Phase 1 clinical trial of RP-A601 in patients with PKP2 variant-mediated Arrhythmogenic Cardiomyopathy. The trial is currently recruiting participants in the United States.
RP-A601 is in Phase 1 clinical development. It is an investigational gene therapy and has not received FDA approval. The drug has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA, which is intended to expedite the development and review of regenerative medicine therapies for serious conditions.
RP-A601 is being studied in a single Phase 1 clinical trial with the identifier NCT05885412. This is a dose escalation trial enrolling approximately 9 adult patients with PKP2 variant-mediated Arrhythmogenic Cardiomyopathy. The trial is actively recruiting in the United States and is an open-label, uncontrolled study.
RP-A601 is a gene therapy that targets the PKP2 gene, but it is not referred to by an alternative name in the available information. It is specifically designed to treat PKP2 variant-mediated Arrhythmogenic Cardiomyopathy by delivering a functional PKP2 gene. The drug is being developed by Rocket Pharmaceuticals under the name RP-A601.