Rocket Pharmaceuticals’ investigational new drug (IND) application for a Phase I clinical trial of RP-A601, an AAV.rh74-based gene therapy candidate to treat arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM), has received clearance from the US Food and Drug Administration (FDA).

The dose escalation, multi-centre trial will enrol at least six adult subjects with PKP2-ACM.

Patients with implantable cardioverter defibrillators (ICDs) and who are at high risk for arrhythmias will receive 8 x 10¹³ GC/kg single dose of RP-A601 having rh74 serotype.

The study intends to assess the safety and preliminary efficacy of RP-A601 along with its impact on cardiac biomarkers and clinical predictors of ventricular arrhythmias.

Furthermore, the impact of RP-A601 on PKP2 myocardial protein expression and sudden cardiac death will be studied in the trial.

Rocket Pharma president and chief operating officer Kinnari Patel said: “RP-A601 offers the potential of a one-time, curative alternative to medical therapy, ICDs, and ablations which are associated with adverse effects, complications, and recurrence of arrhythmias and do not halt the progression of the disease.

“Robust preclinical proof of concept has demonstrated decreased arrhythmias and increased survival.

“With IND clearance in hand, we are rapidly advancing the first investigational gene therapy for PKP2-ACM into the clinic.”

An estimated 50,000 people in the US and EU are affected with PKP2-ACM, a devastating inherited heart disease.

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