China’s National Medical Products Administration has approved Sino Biopharmaceutical Ltd.’s rovadicitinib, branded as Anxu, for first-line treatment of adults with intermediate- or high-risk primary myelofibrosis, as well as post polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.
On the average, humans – and pigs, and deer, and birds – who live at high altitudes have better blood glucose control than their counterparts near sea level. In work published in the Feb. 19, 2026, issue of Cell Metabolism, investigators have linked this phenomenon to red blood cells that directly take up and metabolize glucose from the blood under low oxygen conditions.
Agios Pharmaceuticals Inc. is preparing to present a mixed bag of phase III Rise Up data to the U.S. FDA in hopes of “full approval” for mitapivat in sickle cell disease (SCD), which would make it its third indication in rare hematology.
The pressure to replace animal testing with human-relevant assays that are more predictive of human-drug responses has now reached a tipping point, and there is a movement toward greater acceptance of these potentially more translatable tests.
Sunshine Lake Pharma Co. Ltd. has divulged heterocyclic fused ring compounds acting as pyruvate kinase PKLR activators potentially useful for the treatment of anemia, abetalipoproteinemia, hemolysis, hereditary spherocytosis, sickle cell disease and thalassemia.
A University of Sydney patent details new cyclic peptides acting as coagulation factor XIa inhibitors reported to be useful for the treatment of thrombosis.
Linkcure Therapeutics has synthesized molecular glue degraders acting as zinc finger protein 803 (ZNF803; WIZ) degradation inducers reported to be useful for the treatment of sickle cell anemia and β-thalassemia.
The acquired von Willebrand syndrome (AVWS) is a rare bleeding disorder with laboratory findings similar to those of inherited von Willebrand disease. Researchers from the Nara Medical University and collaborating institutions presented a potential therapeutic approach for AVWS.
Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether.
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-cells-in-blood-flow.webp?height=488&t=1772563584&width=650 "Primary myelofibrosis (PMF) cells in blood flow")
