Rocket Pharmaceuticals has started dosing patients in a Phase I clinical trial of its adeno-associated viral vector (AAV)-based gene therapy, RP-A501, to treat Danon disease.

University of California San Diego (UCSD) Health is serving as the initial and lead centre for the non-randomised, open-label Phase I trial, which is expected to enrol 12-24 pediatric and young adult male patients.

The trial has been designed to evaluate the safety and tolerability of a single infusion of RP-A501.

Rocket Pharmaceuticals CEO and president Gaurav Shah said: “Danon disease is a rapidly progressive cardiomyopathy where we believe gene therapy can make a meaningful difference in patient outcomes. Current standards of care for Danon disease, including heart transplant, are not curative and are associated with considerable morbidity and mortality.

“As a result, median survival for male Danon disease patients has been reported at age 19, caused by progressive heart failure.

“This underscores the urgent need for new treatment options like RP-A501 gene therapy for the patients and families contending with this debilitating, fatal disease.”

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Four patient cohorts will be separated by pediatric and adult age groups and two dose levels will be investigated.

A low dose level of 6.7×1013 genome copies/kg will be given to the first cohort, and the cohort will receive a higher dose upon completion.

Estimated to affect between 15,000 and 30,000 people in the US and EU, Danon disease is caused by mutations in the gene encoding lysosome-associated membrane protein 2.

Cardiac transplantation is the only therapy currently available for Danon disease.