Generation Bio Co. has announced a breakthrough in its nonviral genetic medicine platform with the development of a proprietary, novel DNA called immune-quiet DNA (iqDNA).
Research at Purdue Research Foundation has led to the identification of SYK kinase inhibitors reported to be useful for the treatment of glucose-6-phosphate dehydrogenase deficiency, sickle cell disease, α- and β-thalassemia.
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.