Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy
Conclusions: Stable endogenous FIX concentrations >4% have now been observed out to 2.5 years post-treatment with AMT-060. In addition, we have observed continued reductions in annualized bleeds to near zero with the higher dose of AMT-060, and a 78-93% reduction in exogenous FIX use. The lack of additional safety concerns or treatment-related adverse events over the longer study period, including no recurring changes in ALT, support the continued development and transition of AMT-060 to AMT-061, which will use the same AAV5 vector to deliver the 2x1013 gc/kg dose with the enhanced Padua transgene, which is anticipated to increase FIX activity 6-8 fold.DisclosuresLeebeek: CSL Behring: Research Funding; Uniqure: Research Funding; Baxalta/Shire: Research Funding. Meijer: BMS: Honoraria; Aspen: Honoraria; Boehringer Ingelheim: Honoraria; Pfizer: Research Funding; Sanquin: Honoraria, Research Funding; Bayer: Honoraria, Other: Travel support, Research Funding; UniQure: Research Funding. Coppens: Bayer: Honoraria, Other: Non-financial support, Research Funding; CSL Behring: Honoraria, Other: non-financial support, Research Funding; Uniqure BV: Research Funding. Kampmann: Uniqure BV: Research Funding. Klamroth: Baxalta (Shire), Bayer, CSL Behring, Novo Nordisk, Octapharma, Pfizer, Shire, and SOBI: Consultancy; Baxalta (Shire), Bayer, CSL Behring, Novo Nordisk, Octapharma, Pfizer, Shire, and SOBI: Research Funding. Schutgens: Baxalta/Shire: Research Funding; Novo Nordisk: Research...
Source: Blood - Category: Hematology Authors: Leebeek, F., Meijer, K., Coppens, M., Kampmann, P., Klamroth, R., Schutgens, R., Castaman, G., Seifried, E., Schwaeble, J., Bonig, H., Sawyer, E. K., Miesbach, W. Tags: 801. Gene Therapy and Transfer: Poster II Source Type: research
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