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Rocket Pharmaceuticals Presents Positive Clinical Data from Fanconi Anemia, Pyruvate Kinase Deficiency and Severe Leukocyte Adhesion Deficiency-I Programs at the 64th American Society of Hematology (ASH) Annual Meeting
Sustained genetic and phenotypic correction and concomitant hematologic stabilization observed in at least six of 10 evaluable FA patients between 12 and 36
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\nSustained genetic and phenotypic correction and concomitant hematologic stabilization observed in at least six of 10 evaluable FA patients between 12 and 36 months after RP-L102; a seventh patient potentially demonstrates engraftment and stabilization after 36 months\n\nRobust and sustained efficacy in both adult PKD patients at 24 months post-RP-L301 demonstrated by normalized hemoglobin and improved hemolysis parameters\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces positive clinical data from its lentiviral (LV)-based gene therapy programs at the 64th American Society of Hematology (ASH) Annual Meeting, taking place in New Orleans, Louisiana, from December 10-13.\n\nLentiviral-mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Updated Results from Global RP-L102 Clinical Trials\n\nThe poster presentation includes positive updated data (cut-off October 26, 2022) from the ongoing Phase 2 pivotal trial of RP-L102, Rocket’s ex vivo lentiviral gene therapy candidate for Fanconi Anemia (FA).\n\n\nRP-L102 conferred phenotypic correction in at least six of 10 evaluable patients with ≥12 months of follow-up as demonstrated by increased resistance to mitomycin-C (MMC) in bone marrow (BM)-derived colony forming cells, concomitant genetic correction and hematologic stabilization.\n\n\nA seventh patient has displayed evidence of progressively increasing genetic correction as demonstrated by peripheral blood and BM vector copy numbers (VCNs), with recent development of MMC resistance and indicators of hematologic stability after 36 months of follow-up.\n\n\nThe primary endpoint has been achieved, based on a trial protocol in which statistical and clinical significance requires a minimum of five patients to attain increased MMC resistance at least 10% above baseline at two or more timepoints and concomitant evidence of genetic correction and clinical stabilization.\n\n\n\n\nThe safety profile of RP-L102 has been highly favorable, and the treatment, administered without any cytotoxic conditioning, has been well tolerated. No signs of bone marrow dysplasia, clonal dominance or insertional mutagenesis related to R...