Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RP-A501 · 1 trial · 1 indication
Increase of myocardial tissue expression of LAMP2 protein and decrease in left ventricular mass index (LVMI).
| Arm | Type | Description |
|---|---|---|
| RP-A501 | EXPERIMENTAL | One planned dose of RP-A501 in cohorts of subjects with a confirmed diagnosis of Danon Disease. |
| Name | Type | Description |
|---|---|---|
| RP-A501 | GENETIC | RP-A501 is a gene therapy product consisting of a rAAV9 capsid containing the human LAMP2B transgene which will be administered as a single IV infusion. |
Key Inclusion Criteria: 1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene. 2. Male. 3. Age ≥8 years. 4. Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following: 1. Abnormal thickening of Left ventricul...
RP-A501 is an investigational gene therapy being developed for the treatment of Danon Disease, a rare genetic condition. It is currently in Phase 2 clinical development and has not been approved by the FDA. The therapy is being studied in male patients with the disease.
RP-A501 is a gene therapy designed to address the underlying genetic cause of Danon Disease. It is intended to deliver a functional gene to cells to compensate for the defective gene responsible for the condition. The specific molecular target is not disclosed in the available information.
RP-A501 is being developed by Rocket Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol RCKT. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy for Danon Disease.
RP-A501 is currently in Phase 2 clinical development. It is an investigational drug, meaning it has not yet received FDA approval. The ongoing Phase 2 trial is recruiting participants to further assess the therapy's effects in patients with Danon Disease.
RP-A501 is being studied in a Phase 2 clinical trial with the identifier NCT06092034. This trial is titled 'A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease' and is currently recruiting participants in the United States, Germany, Italy, and the United Kingdom. The trial aims to enroll 14 male patients aged 8 years and older.
RP-A501 has received several FDA designations, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug designation, and Rare Pediatric Disease designation. These designations are intended to support the development of therapies for serious or life-threatening conditions with unmet medical needs.