An experimental gene therapy based on the prime editing technique could become an effective treatment for alternating hemiplegia of childhood, a severe and currently incurable rare disease. David Liu’s lab at the Broad Institute, the inventor of this gene edition methodology, together with scientists from The Jackson Laboratory, successfully reversed the effects of five mutations associated with this disorder in a mouse model. Read More
People with the subtype of B-cell precursor acute lymphoblastic leukemia (BCP-ALL) associated with fusion of MEF2D with other genes show quite poor prognosis. Most patients fail to respond to hematopoietic stem cell transplantation or other conventional treatments. Researchers at Shanghai Jiao Tong University School of Medicine have reported a lead compound that could treat BCP-ALL. Read More
Radiopharm Theranostics Ltd. announced that the U.S. FDA has cleared the IND application for betabart (RV-01), its Lu177-B7H3 monoclonal antibody designed with strong affinity for the 4Ig isoform of B7-H3 that is highly expressed in tumors and not in healthy tissues. Read More
Centauri Therapeutics Ltd. and Kinvard Bio Inc. have separately announced new funding from CARB-X (Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator). The funds will support Centauri's ABX-01 program for multidrug-resistant bacterial strains and Kinvard Bio's oxepanoprolinamides program targeting lower respiratory tract infections as well as skin and soft tissue infections. Read More
Muna Therapeutics ApS has synthesized triggering receptor expressed on myeloid cells 2 (TREM2) agonists reported to be useful for the treatment of Alzheimer’s disease, inflammatory bowel disease, metabolic syndrome, multiple sclerosis, obesity, Parkinson’s disease, rheumatoid arthritis and stroke, among others. Read More
Collaborators in Australia, South Africa, Sweden and the U.K. have linked reduced levels of neuronal cell adhesion molecule (NrCAM) in maternal or placental blood to greater risk of fetal growth restriction
in newborns and of preeclampsia in the mothers, based on analysis of various cohorts of patients from several countries.
Chitinase-3-like protein 1 (CHI3L1) is a secretory glycoprotein from the glycoside involved in the regulation of immune and inflammatory responses. In the brain, CHI3L1 is primarily produced by activated astrocytes, where it is involved in inflammatory neurotoxicity, emerging as a potential biomarker of neuroinflammatory disorders, such as Alzheimer’s disease. Read More
Researchers from Iama Therapeutics Srl and the Italian Institute of Technology have prepared and tested solute carrier family 12 member 2 (SLC12A2; NKCC1) inhibitors reported to be useful for the treatment of complex regional pain syndromes, Alzheimer’s disease, Down syndrome, glioma, Rett syndrome, temporal lobe epilepsy, traumatic brain injury and obsessive-compulsive disorder. Read More
Intracerebral hemorrhage accounts for 10%-15% of all cases of stroke, and it is associated with particularly poor prognosis due to primary and secondary brain injury driven by neuroinflammation. This inflammation involves the activation and subsequent pyroptosis, or lytic cell death, of microglia. Read More
Yichang Humanwell Pharmaceutical Co. Ltd. has discovered nociceptin receptor (OPRL1; KOR3; ORL1) agonists reported to be useful for the treatment of pain. Read More
Humanwell Healthcare (Group) Co. Ltd. has described fused ring compounds acting as angiotensin AT2 receptor (AGTR2) agonists reported to be useful for the treatment of disorders of the ovary and female reproductive tract, metabolic diseases, cardiovascular, eye, gastrointestinal, neurological, renal and respiratory disorders. Read More
Osteoporosis involves degradation of bone throughout the body, and it already affects nearly a quarter of a billion people in the aging global population. Read More
