Rocket Pharmaceuticals Receives FDA IND Clearance for BAG3-DCM Gene Therapy RP-A701
BAG3-DCM is a rare and inherited heart condition caused by mutations in the BAG3 gene (Bcl2-associated athanogene 3). This leads to early-onset, rapidly progressing heart failure characterized by progressive ventricular enlargement and impaired systolic function, resulting in high morbidity and premature mortality.
Scientific equipment in a lab setting, revealing the cutting edge of biotechnology research.
A loss of BAG3 leads to the accumulation of misfolded and damaged proteins, impairing the heart's ability to contract. Rocket Pharmaceuticals estimates that ~30,000 individuals in the US are affected by BAG3-DCM. Current therapies don't prevent disease progression and are associated with complications.
Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) is a late-stage biotechnology company that develops gene therapies for rare diseases in the US.
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Disclosure: None. This article is originally published at Insider Monkey.
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