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ASH 2020 | First-in-human gene therapy study of peboctocogene camaparvovec (BAY 2599023) in severe hemophilia A
Steven Pipe, MD, University of Michigan, Ann Arbor, MO, discusses the results of a Phase I/II study (NCT03588299) aiming to evaluate peboctocogene camaparvovec (BAY 2599023) infusion therapy in previously treated patients with hemophilia A (HA). In this first-in-human gene therapy study of AAVhu37 capsid vector technology in severe HA, the main aims were to measure post-treatment factor VIII (FVIII) expression and evaluate the overall safety of the treatment. The findings of this study were encouraging FVIII expression and sustained FVIII levels, with no evidence of expression loss over time being found. peboctocogene camaparvovec also has a favorable safety profile, with no serious treatment-emergent adverse events reported in the 18-month follow-up period. The study continues, aiming to find an optimal dose to be carried into a Phase III study. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.
Disclosures
Consulting: Apcintex, Bayer, Biomarin, Catalyst Biosciences, CSL Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, Roche/Genentech, Sangamo Therapeutics, Sanofi, Takeda, Spark Therapeutics, uniQure
