Rocket Pharmaceuticals, Inc. presented at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). Data updates demonstrate the continued safety and efficacy of the Phase 1/2 pivotal studies of KRESLADI? (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I) and RP-L102 for Fanconi Anemia (FA), in addition to the Phase 1 study of RP-L301 for Pyruvate Kinase Deficiency (PKD).

Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I Provides Sustained Efficacy with a Well-Tolerated Safety Profile: The oral presentation includes positive, updated data (cut-off July 24, 2023) from the global Phase 1/2 pivotal studies demonstrating sustained efficacy and safety of KRESLADITM from 18 to 45 months of follow-up for all nine patients with severe LAD-I. Observed 100% survival in the absence of allogeneic HSCT at least 18 months post-infusion in all nine patients; all patients enrolled at less than 12 months of age have surpassed 24 months without HSCT. All primary and secondary endpoints were met, including sustained genetic and phenotypic correction. When compared with pre-treatment history, data showed substantial decreases in the incidences of significant infections requiring hospitalization or intravenous antimicrobials, combined with evidence of resolution of LAD-I-related skin and periodontal lesions and restoration of wound repair capabilities.

KRESLADITM was well-tolerated in all patients with no drug-related serious adverse events reported to date. Adverse events related to other study procedures, including busulfan conditioning, have been previously disclosed and are consistent with the safety profiles of those agents and procedures. No cases of graft failure or autologous graft-versus-host-disease (GvHD) were reported.

Based on the positive efficacy and safety data from the global pivotal studies of KRESLADITM for severe LAD-I, the Biologics License Application (BLA) was accepted for review by the U.S. Food and Drug Administration (FDA) who has set the New Prescription Drug User Fee Act (PDUFA) target date of June 30, 2024. The oral presentation includes positive, updated data (cut-off February 5, 2024) from the Phase 1 study from two adult patients with PKD treated with RP-L301 followed up to 36 months and two pediatric patients followed up to 12 months.