Rocket Pharma doses first patient in Phase 1 study of Danon treatment

RP-A501 gene therapy can make a significant difference in patients who are suffering from Monogenic Heart Failure Syndrome

Rocket Pharmaceuticals Inc, a multi-platform clinical-stage gene therapy company based in the U.S., recently announced that they have commenced dosing patients in the open-label, Phase 1 clinical study of RP-A501, a gene therapy based on adeno-associated viral vector (AAV) that is developed to treat Danon disease.

Apparently, University of California San Diego (UCSD) Health is the lead and initial center for Phase 1 trial which is led by Barry Greenberg, Director of Advanced Heart Failure Treatment Program at UC San Diego Health and Professor of Medicine at UC San Diego School of Medicine and happens to be the trial’s principal investigator, along with Eric Adler, M.D., Director of Cardiac Transplant and Mechanical Circulatory Support at UCSD Health and Professor of Medicine at UCSD.

Chief Executive Officer and President of Rocket, Gaurav Shah, M.D., said that the Danon disease is a progressive cardiomyopathy wherein the company believes the gene therapy can make a significant difference in patient outcomes. The existing standards of care for Danon are not curative and are generally associated with certain morbidity and mortality.

Consequently, the age of 19 has been reported as the median survival age for male patients suffering for Danon disease, reportedly caused by heart failure, Shah said. This demands an urgent need for alternative options like RP-A501 gene therapy to address patients and families facing the fatal disease.

Dr. Adler said that the development of RP-A501 into clinic is an enormous step towards treating this fatal disease and rare cardiac disorders. UCSD Health is looking forward to being the first and lead center to hold RP-A501 Phase 1 clinical trial and it looks forward to rapidly advance it through the clinic for patients in need.

Apparently, the study is designed to specifically assess the tolerability and safety of RP-A501’s single infusion.

Source Credit- https://rocketpharmaceuticals.gcs-web.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-patient-dosing-has-commenced