This study evaluates the efficacy of fitusiran, an investigational RNA interference (RNAi) therapeutic, in reducing bleeding episodes in a mouse model deficient in coagulation Factor X. The research aims to determine whether fitusiran can restore hemostasis in the context of Factor X deficiency by targeting antithrombin to rebalance coagulation pathways.
Administration of fitusiran led to a significant reduction in bleeding events in Factor X-deficient mice, indicating that lowering antithrombin levels can compensate for the lack of Factor X and restore hemostatic balance.
The study utilized a Factor X-deficient mouse model developed in collaboration with genOway. This model carries a targeted deletion of the F10 gene, resulting in a severe bleeding phenotype that mimics human Factor X deficiency.
Hemophilia, Coagulation disorders, RNA interference therapy, Antithrombin modulation, Bleeding management
Gene Knockout, Hemostatic assessment, RNAi therapeutic evaluation, Coagulation factor deficiency model, Antithrombin targeting
From model design to experimental results
Tailor-made solutions adapted to scientific questions
Comprehensive dataset package
Generated with biopharma partners and in-house
Scientific follow-up and advice along the project
Collaborative approach for problem solving and development of innovative models
Breeding facilities in US and Europe
Certified health status from professional breeders
