Hematology

Mouse studies suggest nanoparticles loaded with Factor IX mRNA could help treat hemophilia. The nanoparticles consisted of an ionizable lipid, cholesterol and PEG ( Arcturus Therapeutics Inc.'s LUNAR nanoparticle system) loaded with human Factor IX mRNA. In a mouse model of hemophilia, the Factor IX mRNA-loaded nanoparticles increased clotting activity for up to 48 hours compared with no treatment. Also in the model, the nanoparticles loaded with an engineered hyperfunctional variant of Factor IX mRNA increased clotting activity to normal levels, whereas vehicle had no effect. Next steps in collaboration with Arcturus could include testing the nanoparticle-based therapy in a canine model of hemophilia.

Dimension Therapeutics Inc. and RegenxBio Inc. have DTX101, a recombinant adeno-associated virus (AAV) serotype 10 (AAVrh10) vector delivering the wild-type Factor IX gene, in Phase I/II for hemophilia B...