At the recent annual congress of the European Association for Haemophilia and Allied Disorders, researchers from Novalgen Ltd. presented the discovery and preclinical characterization NVG-444, a potentially first-in-class next-generation FVIII mimetic antibody equipped with autoregulation to reduce prothrombotic risks that is being developed for the treatment of hemophilia A.
Researchers at E-Therapeutics plc recently presented efficacy and safety data on ETX-148, a pan-hemophilia agent in murine models of hemophilia A and B.
Bioray Pharmaceutical Co. Ltd. has announced clinical trial clearance in China by the National Medical Products Administration (NMPA) for BR-111 for injection for the treatment of ROR1-positive hematological malignancies and solid tumors.
Fabry disease is an X-linked lysosomal storage disorder caused by a deficiency of the enzyme α-galactosidase A (GLA), which leads to complex multisystemic involvement, with the majority of patients developing hypertrophic cardiomyopathy.
Xaicure Pharmaceuticals (Suzhou) Co. Ltd. has described histone acetyltransferase KAT6A (monocytic leukemia zinc finger protein; MOZ; MYST-3) and/or histone acetyltransferase KAT6B (MOZ2; MYST-4) inhibitors reported to be useful for the treatment of cancer.
Maze Therapeutics Inc. has divulged apolipoprotein L1 (APOL1) inhibitors reported to be useful for the treatment of chronic kidney disease, focal segmental glomerulosclerosis, diabetic retinopathy, diabetic HIV-associated nephropathy, lupus nephritis, pre-eclampsia and sepsis.
Mindrank AI Co. Ltd. and Mindrank Therapeutics (Suzhou) New Drug Research & Development Co. Ltd. have identified molecular glue degraders comprising cereblon (CRBN) ligands acting as eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1) degradation inducers reported to be useful for the treatment of cancer.
Pharmaengine Inc. and Sentinel Oncology Ltd. have synthesized Wee1-like protein kinase (Wee1) inhibitors and proteolysis targeting chimera (PROTAC) compounds reported to be useful for the treatment of cancer.
Hanyang University and Korea Institute of Science and Technology have disclosed HER2 (erbB2) inhibitors reported to be useful for the treatment of cancer.
Early dysfunction of the endothelial/blood-brain barrier (BBB) is involved in the pathogenesis of cerebral small vessel disease (SVD), which is a contributor to about 50% of dementias. Sphingosine-1-phosphate (S1P) is a sphingolipid that regulates the BBB integrity when bound to its receptor S1P1 on endothelial cells.
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