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 the treatment of PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), a cardiovascular condition. It is currently in Phase 1 clinical development and has not been approved by the FDA.
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 to evaluate the safety and efficacy of RP-A601 in patients with PKP2-ACM.
RP-A601 is currently in Phase 1 clinical development. It is an investigational gene therapy and has not received FDA approval. The ongoing Phase 1 trial is a dose escalation study designed to assess the safety and tolerability of RP-A601 in patients with PKP2-ACM.
RP-A601 is being studied in a Phase 1 clinical trial with the identifier NCT05885412. This is a dose escalation trial enrolling approximately 9 participants with PKP2 variant-mediated Arrhythmogenic Cardiomyopathy. The trial is currently recruiting participants in the United States and is not randomized or blinded.
RP-A601 is a gene therapy specifically designed to treat PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), a condition caused by variants in the PKP2 gene. It is not the same as the disease itself; rather, it is an investigational therapeutic approach targeting the underlying genetic cause of the condition.