Scientists at the Center for Genomic Regulation (CRG) have developed an AI-based tool to design thousands of sequences that regulate DNA. They have also synthesized these molecules, called enhancers, to control gene activation in mouse hematopoietic stem cells, which they have tested in vitro.
Anticoagulant drugs, whether classical ones such as warfarin and heparin or newer ones such as dabigatran and apixaban, can be effective for treating and preventing deep vein thrombosis, myocardial infarction, ischemic stroke and pulmonary embolisms. Targeting factor XIa, a serine protease in the coagulation pathway, may inhibit thrombosis without increasing bleeding risk. Numerous inhibitors of factor XIa have been developed and several have entered clinical trials, but most have shown problems of poor efficacy, inadequate selectivity or drug-drug interactions.
A recent study by researchers from Texas A&M University presented a new vaccine designed to target the ligand-binding domain of the serotonin 2A receptor (5-HT2AR), which resides in the second extracellular loop (EL2) and was previously identified as the key region for receptor activation. The new candidate, called EL2-5HTVac, was shown to provide a long-lasting and selective therapeutic approach to avoid increased bleeding risk complications.
The University of Michigan has divulged lysine-specific histone demethylase 1A (KDM1A; LSD1) inhibitors reported to be useful for the treatment of cancer, autism, myocardial fibrosis and more.
Sepsis-induced coagulopathy (SIC) is a complication of sepsis tied to high mortality in patients. Anticoagulation using a coagulation factor IIa and Xa dual inhibitor might have the potential to improve the treatment of this severe condition.
Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors.
Sanofi SA gained U.S. FDA approval for fitusiran as a first-in-class siRNA therapy for hemophilia. Branded Qfitlia, the antithrombin-lowering therapy is indicated for use as a prophylactic treatment to prevent or reduce bleeding episodes in people with hemophilia A or B, with or without inhibitors, and joins the small but growing group of non-factor therapy options, with the advantage of a broad label and a convenient dosing regimen.
Ono Pharmaceutical Co. Ltd. struck a licensing deal with Ionis Pharmaceuticals Inc. for sapablursen, which is in phase II trials for polycythemia vera. Under terms, Osaka, Japan-based Ono gains an exclusive license to develop and commercialize sapablursen worldwide. Carlsbad, Calif.-based Ionis will be responsible for completing the ongoing phase II Imprssion study, while Ono will be responsible for subsequent development, regulatory filings and commercialization.
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