Genetic deletion of protease-activated receptor 4 (PAR4) in experimental models has been linked to improvement in neurological dysfunction and reduction of infarct volume in the context of stroke. Moreover, pretreatment with PAR4 antagonists in models of traumatic brain injury has shown protection against secondary tissue injury and thrombo-inflammatory activation.
A group of scientists from Basel University Hospital have designed an antibody-drug conjugate (ADC) that eliminated blood cancer cells without attacking healthy hematopoietic stem cells (HSCs), which they modified by base editing and transplanted to renew an altered blood system. They achieved this by focusing on the panhematopoietic marker CD45.
Mast/stem cell growth factor receptor KIT is a receptor tyrosine kinase that plays a key role in mast cell differentiation, proliferation, survival and activation.
Transthera Biosciences Co. Ltd. has described NLRP3 inflammasome inhibitors reported to be useful for the treatment of autoimmune disease, inflammatory disorders and immunological disorders.
Novo Nordisk A/S has identified C-type natriuretic peptide (CNP) compounds reported to be useful for the treatment of achondroplasia and heart failure.
Scientists at Shanghai Synergy Pharmaceutical Sciences Co. Ltd. and Zhejiang Huahai Pharmaceutical Group Co. Ltd. have synthesized non-receptor tyrosine-protein kinase TYK2 inhibitors reported to be useful for the treatment of psoriasis, inflammatory bowel disease (IBD) and lupus erythematosus.
Scientists at Hansoh Bio LLC, Hansoh Pharmaceutical Group Co. Ltd. and Shanghai Hansoh Biomedical Co. Ltd. have disclosed antibody-drug conjugates (ADCs) comprising HER3 (erbB3)-targeting antibodies linked to a cytotoxic drug through a linker.
Since utrophin compensates for lack of dystrophin in mdx mice, which results in a milder phenotype of muscular dystrophy compared to humans, the mdx/utrn-/- mouse has been developed to mimic early onset of muscle dystrophy, severe muscle weakness and premature death.
Researchers from the University of Colorado presented preclinical data for the myeloid leukemia cell differentiation protein Mcl-1 inhibitor S-64315, currently in phase I clinical development for the treatment of hematologic cancers.