In a regulatory filing, Rocket Pharmaceuticals (RCKT) stated: “As previously disclosed, the Company has reached agreement with the U.S. Food and Drug Administration on the study design of the Phase 2 pivotal trial for RP-L301, the Company’s ex vivo LV-based program targeting Pyruvate Kinase Deficiency, a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. While the Phase 2 RP-L301 Study is ready for patient enrollment, the Company is currently focusing its resources on its other programs and has not initiated enrollment in the Phase 2 RP-L301 Study. The Company anticipates resuming patient enrollment in 2025.”
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