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RTW Biotech Opportunities Ltd - New York-based investment firm focused on the life sciences sector - Notes announcement by its largest portfolio company position, Rocket Pharmaceuticals, Inc. Says Rocket has reached alignment with the US Food and Drug Administration on the trial design for its phase two trial of RP-A501 for Danon disease, a fatal inherited cardiomyopathy for which there is currently no cure. The global, single-arm, multi-centre trial will evaluate the efficacy and safety of RP-A501 in 12 patients with Danon. To support accelerated approval, the study will assess the efficacy of RP-A501 as measured by the biomarker-based primary endpoints. RTW Biotech has a 10% stake in Rocket Pharma. Current stock price: $1.26 12-month change: up 7.7% Copyright 2023 Alliance News Ltd. All Rights Reserved.
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