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Rocket Pharma advances lead AAV gene therapy in cardiomyopathy study

September 2, 2020

Rocket Pharmaceuticals (NASDAQ:RCKT) has treated the first patient in the higher-dose cohort in its Phase 1 clinical trial of RP-A501, an adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon disease, a rare inherited disorder characterized by weakening of heart and skeletal muscles.

The second cohort evaluates RP-A501 at a higher-dose level of 1.1×1014 genome copies/kilogram in male patients 15 years of age and older. The Phase 1 study will assess the safety, tolerability and preliminary efficacy of RP-A501.

Treatment of the higher-dose cohort comes after successful completion of the low-dose cohort and FDA clearance.

Following the review of safety data from the first cohort, all subsequent cohorts will include 2-4 patients per cohort, adjusted down from 3-6 patients in the original protocol.

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