Joseph Rossano, MD, on the Potential of Gene Therapy in Danon Disease


Rossano discussed unmet needs for patients with Danon disease and the potential of investigational gene therapy RP-A501.

“Potentially, a gene therapy for this disorder can be a real game changer. In the current state, there really is no effective medical therapy. We have medicines that can hopefully help treat symptoms of heart failure and can treat arrhythmias. But we have no therapies that alter the natural history of the disease. And so what's really exciting about this therapy is that it offers the prospect and the hope of dramatically changing the natural history.”

Patients with Danon disease have limited options. While the current standard of care involves treatment of symptoms, many patients eventually require heart transplant, and there are currently no disease-modifying therapies available for the disorder.

Joseph Rossano, MD, MS, FAAP, FACC, chief, Division of Cardiology, Children's Hospital of Philadelphia, recently presented data from a phase 1 clinical trial of RP-A501 (NCT03882437), an investigational adeno-associated serotype 9 (AAV9) vector-based gene therapy intended to treat Danon disease, at the Heart Failure Society of America (HFSA) Annual Scientific Meeting, held in Washington DC, September 30 – October 3, 2022.

In an interview with CGTLive, Rossano discussed the unmet needs for patients with Danon disease and the limitations of currently available treatment options. He then gave an overview of the safety and efficacy data that has come out of the clinical trial for RP-A501 so far and spoke about what he sees as the potential of the therapy for patients. He additionally noted that more follow-up data is still needed and concluded by outlining his hopes for the future of gene therapy in treating rare cardiovascular disorders in general.

Rocket Pharmaceuticals announces positive updates from phase 1 clinical trial for RP-A501 in Danon disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022. News release. Rocket Pharmaceuticals. September 30, 2022. 
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