Recently, researchers at Cincinnati Children’s Hospital, in collaboration with colleagues in Japan, have developed a human vascular organoid model that accurately mimics the vascular damage caused by SARS-CoV-2.
Baudax Bio Inc.’s lead clinical candidate, TI-168, has been awarded U.S. orphan drug designation by the FDA for the treatment of hemophilia A with inhibitors. TI-168 is a next-generation, factor VIII (FVIII)-specific regulatory T-cell (Treg) therapy designed to address hemophilia A in patients with FVIII inhibitors.
Precision Biosciences Inc. uses its proprietary Arcus platform to develop in vivo gene editing therapies and has outlined new data from its wholly owned and partnered pipeline.
Current anticoagulant strategies include small-molecule inhibitors and biological entities targeting factor XI (FXI) which, although effective, still have bleeding as a major risk.
Researchers have demonstrated that inhibiting mitophagy in ‘old’ hematopoietic stem cells (HSCs) completely restored their blood reconstitution capabilities, raising the prospect of addressing the age-related weakening of the immune system that stems from HSCs deteriorating over time.
Researchers from University of Michigan presented preclinical data for the novel oxylipin analogue CS-585 being developed as an antithrombotic agent. CS-585 was synthesized as an orally available analogue of 12(S)-hydroxy-eicosatrienoic (12-HETrE), with potent and selective prostacyclin (IP) receptor agonist activity and ability.
Under pathologic conditions such as vascular injury or atherosclerosis, the hyperactivation of platelets may lead to occlusive thrombus formation, myocardial infarction or stroke. Although there are several targets for clot prevention validated clinically, these strategies may present bleeding risk as a limitation. Researchers from the University of Michigan have reported on CS-014, a histone deacetylase (HDAC) inhibitor aimed to reduce clot formation without risk of bleeding.
Scientists have discovered that a small chemokine protein released by activated platelets, platelet factor 4 (PF 4), reduced neuroinflammation, and improved cognition in aged mice. The study was published on Aug. 16 in the online edition of Nature.
Agios Pharmaceuticals Inc. and Alnylam Pharmaceuticals Inc. have entered into an exclusive worldwide license agreement under which Agios will acquire the rights to develop and commercialize Alnylam’s novel preclinical siRNA targeting TMPRSS6, as a potential disease-modifying treatment for patients with polycythemia vera (PV).
Scribe Therapeutics Inc. has announced an expanded collaboration with Sanofi SA, under which Sanofi receives an exclusive license to use Scribe’s CRISPR X-Editing (XE) genome editing technologies for the development of in vivo therapies, including for sickle cell disease.