NEW YORK – The US Food and Drug Administration has cleared Rocket Pharmaceuticals' investigational new drug application, granting permission for the start of a Phase I clinical trial for RP-A601, the gene therapy developer said Tuesday.
RP-A601 is an adeno-associated virus (AAV)-based gene therapy designed to treat arrhythmogenic cardiomyopathy, a rare inherited heart disease that is caused by mutations in the PKP2 gene (PKP2-ACM) and can result in arrhythmias, cardiac structural abnormalities, and sudden cardiac death.
In the Phase I dose escalation trial, investigators will study the drug's effect on plakophilin 2 pathogenic (PKP2) myocardial protein expression, cardiac biomarkers, and clinical predictors of life-threatening ventricular arrhythmias and sudden cardiac death. The Cranbury, New Jersey-based firm plans to enroll at least six adult PKP2-ACM patients in the multicenter study.
Rocket Pharmaceuticals said it is studying RP-A601 as a one-time, potentially curative therapy for PKP2-mutated arrhythmogenic cardiomyopathy.
The company conducted preclinical studies of the therapy in partnership with researchers at New York University's Grossman School of Medicine. In those studies in PKP2 knockout mice, RP-A601 decreased arrhythmias, improved right ventricular morphology and function, and increased survival.