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KPV Safety Profile KPV demonstrates a favorable safety profile in all preclinical studies reviewed, with no acute toxicity at therapeutic doses

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BLA for Rockets LAD-I Gene Therapy Under Review by FDA Again October 14, 2025 The FDA has accepted Rocket Pharmaceuticals resubmission of a BLA for marnetegragene autotemcel (RP-L201, to be marketed as Kresladi), a genetically modified autologous hematopoietic stem cell therapy intended to treat LAD-I