Fitusiran's mechanism of reducing antithrombin levels could apply to all hemophilia patients, as well as those with other rare bleeding disorders. Reducing antithrombin increases thrombin levels and helps to increase normal clotting.
Alnylam Pharmaceuticals Inc. is gearing up to move its hemophilia candidate fitusiran into pivotal trials next year, and is hoping that its novel mechanism of action will differentiate the antithrombin-targeting RNA-interference drug from the increasingly crowded field.
The hemophilia market has heated up in recent years with a range of new products and player. Shire PLC...
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US FDA approval of Niktimvo for third-line chronic GVHD, a competitor to Sanofi’s Rezurock, came less than two months ahead of anticipated approval of Syndax’s leukemia drug revumenib.
The company’s giroctocogene fitelparvovec, partnered with Sangamo, could compete with BioMarin’s hemophilia A gene therapy Roctavian, but longer-term data may be needed.
2seventy bio sold full rights to a hemophilia A program and in vivo gene editing technology to partner Novo Nordisk.
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Pipelines in bioconjugates continue to develop, from ADCs to XDCs, with radionuclide drug conjugates (RDCs) and antibody-oligonucleotide conjugates (AOCs) leading the expansion.
Interim results from the Phase III REMARK study presented at WCLC showed the Chinese company’s second-generation ALK/ROS1 inhibitor met its primary endpoint as a first-line therapy in ALK-positive advanced NSCLC.
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