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RP-A501 · 2 trials · 1 indication
Myocardial tissue expression of LAMP2 protein improved from baseline to ≥ Grade 1 (by IHC), AND, ≥ 10% decrease in LVMI from baseline.
Evaluation of safety associated with RP-A501
Assessment of safety at both doses (single IV administration)
Assessment of cardiomyocyte histologic correction following administration of RP-A501 via endomyocardial biopsy
Assessment of clinical stabilization of cardiomyopathy following infusion of RP-A501 via cardiopulmonary testing
| Arm | Type | Description |
|---|---|---|
| RP-A501 | EXPERIMENTAL | A single intravenous infusion 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 an rAAV9 capsid containing the human LAMP2B transgene which will be administered as a single IV infusion. Dose of RP-A501: Cohorts 1, 2 \& 3 = 6.7 x10\^13 genome copies (GC)/kg; Cohort 4 = 3.8 x10\^13 GC/kg. An immunomodulation regimen is initiated before RP-A501 administration to reduce the risk of gene therapy-related complications. The regimen includes rituximab or a biosimilar, administered in two doses for Cohorts 1-3 and three doses for Cohort 4, along with sirolimus and corticosteroids. Pegcetacoplan was incorporated into the immunomodulation regimen for Cohort 3 but excluded in Cohort 4. |
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 Danon Disease, a rare inherited heart condition. It is designed to deliver the LAMP2B gene to patients and is currently in Phase 2 clinical development. Rocket Pharmaceuticals, Inc. (ticker RCKT) is the developer. RP-A501 has received RMAT, Orphan Drug, Rare Pediatric Disease, and Fast Track designations from the FDA.
RP-A501 is being developed to treat Danon Disease, a rare genetic disorder that primarily affects the heart and is caused by mutations in the LAMP2 gene. The therapy is intended for male patients aged 8 years and older. It is investigational and has not been approved for commercial use.
RP-A501 targets LAMP2B, the protein encoded by the LAMP2 gene. In Danon Disease, mutations in LAMP2 lead to deficient or absent LAMP2B protein, which causes cellular dysfunction, particularly in heart muscle. RP-A501 is designed to deliver a functional copy of the LAMP2B gene to restore production of this protein.
RP-A501 is being developed by Rocket Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker symbol RCKT. Rocket Pharmaceuticals is a clinical-stage biotech company focused on gene therapies for rare diseases. RP-A501 is its investigational gene therapy candidate for Danon Disease.
RP-A501 is in Phase 2 clinical development. A Phase 1 trial has been completed, and a Phase 2 trial is currently recruiting. RP-A501 is investigational and has not been approved by the FDA. It holds RMAT, Orphan Drug, Rare Pediatric Disease, and Fast Track designations.
RP-A501 has been studied in two clinical trials. The completed Phase 1 trial, NCT03882437, enrolled 7 male patients with Danon Disease in the United States. The ongoing Phase 2 trial, NCT06092034, is recruiting 18 male patients aged 8 years and older across the United States, Germany, Italy, and the United Kingdom.