Researchers have gained new insights into physiological mechanisms that protect against blood clotting in immobilized individuals by studying animals that stay immobile for a good chunk of the year at a time: hibernating bears. “As a clinician, if you think about immobility, you always think about thrombosis,” Tobias Petzold told BioWorld. But his team’s work, which was published in the April 13, 2023, issue of Science, demonstrated that “immobility can trigger antithrombotic mechanisms.”
Base editing (BE), a technique that modifies a single nucleotide in living cells, has been successfully tested to resolve the CD3δ mutation in severe combined immunodeficiencies (SCIDs) and produce functional T cells. For now, scientists at the University of California, Los Angeles (UCLA), completed the study on patient stem cells and artificial thymic organoids, shortening the way for future clinical trials.
Incyte Corp.’s bid for a once-daily vs. twice-daily version of the Janus kinase inhibitor Jakafi (ruxolitinib) was foiled, at least temporarily, by a complete response letter (CRL) from the U.S. FDA.
Janssen Pharmaceutica NV has disclosed 5-oxo-1,2,3,5,8,8a-hexahydroindolizine-3-carboxamide derivatives acting as coagulation factor XIa and plasma kallikrein (KLKB1) inhibitors reported to be useful for the treatment of thromboembolism, diabetes, diabetic retinopathy, septic shock, hereditary angioedema, arthritis, nephropathy and inflammatory disorders, among others.
Two molecules that affected the cell cycle only of acute myeloid leukemia (AML) cells could be used as a clinical strategy against this pathology. Scientists at Memorial Sloan Kettering Cancer Center and Harvard University have discovered that DEG-35 and DEG-77 arrested the cell cycle and promoted cell differentiation and apoptosis in these cells.
Current antithrombotic therapies for the prevention and management of cardiovascular disorders such as thrombosis, myocardial infarction (MI) or stroke present an associated risk of bleeding. The essential events leading to the formation of hemostatic clots are platelet activation and fibrin formation. When activated, the prostacyclin (IP) receptor prevents platelet aggregation in arteries and veins after injury.
The U.S. FDA has approved the priority BLA for Sanofi SA’s hemophilia A treatment nearly a week before its Feb. 28 PDUFA date. The approval is for efanesoctocog alfa, a recombinant factor VIII (rFVIII) therapy – the company has managed to partially incorporate rFVIII into the drug’s brand name, Altuviiio. The price per dose was not released by the company.
The positive opinion Jan. 27 from the EMA’s Committee for Medicinal Products for Human Use regarding Reblozyl (luspatercept) from Bristol Myers Squibb Co. to treat adults with non‑transfusion-dependent beta-thalassemia marked an advance in the space, where several developers are jockeying for position. Reblozyl, a first-in-class erythroid maturation agent, was first approved in November 2019 in the hands of Celgene Corp., acquired by Princeton, N.J.-based BMS the same year.
The success of the treosulfan-based conditioning regimen in patients with β-thalassemia undergoing hematopoietic cell transplantation (HCT) is limited due to several complications, such as mixed chimerism and graft rejection. Researchers previously found that polymorphisms in the NQO1 or glutathione S-transferase A1 (GSTA1) genes had an impact on treosulfan pharmacokinetics, which then impacted related toxicities after HCT.
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