A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Conclusions: Current treatments have reduced life-threatening bleeds, chronic joint disease and disability; however, people with haemophilia B continue to have significantly decreased quality of life. This study has shown a single infusion of low dose FLT180a leads to FIX levels of >40%, greatly reducing the risk of spontaneous or traumatic bleeds and raises the prospect of achieving a functional cure by normalising FIX levels with a higher dose. This will reduce the risk of life-threatening bleeds following trauma and surgery, transform quality of life and thus represents a major therapeutic advance for people with haemophilia B.DisclosuresChowdary: Bayer, CSL Behring, Novo Nordisk, Pfizer, and SOBI (publ): Research Funding; Baxalta (Shire), Baxter, Biogen Idec, CSL Behring, Freeline, Novo Nordisk, Pfizer, Roche, Shire, and SOBI: Consultancy. Shapiro: Freeline Therapeutics Ltd: Consultancy. Alade: Freeline: Employment. Brooks: Freeline Therapeutics Ltd: Employment. Dane: Freeline: Employment. McIntosh: Freeline: Consultancy. Short: Freeline Therapeutics Ltd: Employment. Tuddenham: BioMarin: Consultancy, Patents & Royalties; Freeline: Consultancy. Nathwani: Freeline: Consultancy, Equity Ownership, Membership on an entity's Board of Directors or advisory committees.

Source: Blood - November 21, 2018 Category: Hematology Authors: Chowdary, P., Shapiro, S., Davidoff, A. M., Reiss, U., Alade, R., Brooks, G., Dane, A., McIntosh, J., Short, G., Tuddenham, E., Nathwani, A. C. Tags: 322. Disorders of Coagulation or Fibrinolysis: Hemophilia: Emerging Therapies Source Type: research