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June June 28 Product: Rocket Pharmaceuticals Kresladi Indication: Severe leukocyte adhesion deficiency-I (LAD-I) Rocket Pharmaceuticals announced Friday that the FDA rejected its rare disease gene therapy Kresladi (marnetegragene autotemcel) in a Complete Response Letter requesting limited additional chemistry, manufacturing and controls information to complete the regulators review

Its integration with zebrafish models facilitates large-scale drug screening with minimal sample consumption and enables systematic analysis of dose-response relationships and compound synergies through controlled concentration gradients and combinatorial strategies