Rocket Pharmaceuticals Inc. (NASDAQ: RCKT) receives FDA clearance for RP-A701 gene therapy to treat rare BAG3-associated Dilated Cardiomyopathy, a condition with high morbidity and premature mortality. The therapy aims to address the impaired heart function caused by BAG3 mutations, affecting an estimated 30,000 individuals in the US.

The company, known for developing gene therapies for rare diseases, highlights the urgent need for effective treatments for BAG3-DCM, as current therapies do not prevent disease progression and come with complications. Rocket Pharmaceuticals aims to provide a novel solution with its RP-A701 gene therapy candidate, designed to address the underlying genetic cause of the condition.

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Read more at Yahoo Finance: Rocket Pharmaceuticals Receives FDA IND Clearance for BAG3-DCM Gene Therapy RP-A701