In 2021, Ardem Patapoutian won the Nobel Prize in Physiology or Medicine for his discovery of the mechanosensitive receptors Piezo1 and Piezo2. The receptors, which are ion channels that respond to mechanical pressure, are important in touch sensation, as well as regulating processes including bladder control and blood pressure. Now, investigators at The Hospital for Sick Children in Toronto, Canada, and Central South University’s Xiangya Hospital have discovered a decidedly ignoble role for Piezo2. Read More
Self-organizing organoids resembling specific regions of the nervous system, including human cortical organoids (hCO), can be generated and used as disease models. However, the application of hCOs is precluded by several limitations, including the lack of integration into in vivo circuits that generate behavioral outputs. To overcome these issues, transplantation of hCOs into intact living brains has been proposed. Read More
HER2 is a receptor tyrosine kinase and well-established oncogenic driver with mutations present in various types of tumors such as bladder urothelial carcinoma, uterine corpus endometrial carcinoma, stomach adenocarcinoma and breast cancer. Read More
Regeneron Pharmaceuticals Inc. has elected to exercise its right to advance a therapeutic antibody candidate, discovered in partnership with Abcellera Biologics Inc. as part of a multitarget collaboration between the companies, into further preclinical development. Read More
CV6 Therapeutics (NI) Ltd. has secured US$9.2 million to progress its lead oncology asset CV6-168 into a first-in-human phase Ia trial and perform further scientific development work. CV6-168 is a novel, first-in-class DNA uracilation agent that selectively targets the enzyme dUTPase. Read More
Exelixis Inc. and Sairopa BV have entered into an exclusive clinical development and option agreement for ADU-1805, a potentially best-in-class monoclonal antibody that targets SIRPα. Read More
Sumitomo Pharma Co. Ltd. has divulged bicyclic pyridine derivatives acting as trace amine-associated receptor 1 (TAAR1) agonists reported to be useful for the treatment of neurological and psychiatric disorders. Read More
Eccogene (Shanghai) Co. Ltd. has received FDA clearance to begin a phase I study of its glucagon-like peptide 1 (GLP-1) receptor agonist ECC-5004 for type 2 diabetes. Read More
Immunic AG has disclosed deuterated dihydroorotate dehydrogenase (DHODH) inhibitors reported to be useful for the treatment of Crohn's disease, influenza, lupus erythematosus, multiple sclerosis, psoriasis, rheumatoid arthritis, SARS-CoV-2 infection (COVID-19) and cancer. Read More
Equillium Inc. has advanced EQ-102, a first-in-class multi-cytokine inhibitor of IL-15 and IL-21 for celiac disease, into a phase I study in healthy volunteers. Read More
Standigm Inc. has disclosed leucine-rich repeat kinase 2 (LRRK2), particularly LRRK2 G2019S mutant, inhibitors reported to be useful for the treatment of cancer, neurodegeneration, rheumatoid arthritis, tuberculosis, axial spondyloarthritis, radiographic (ankylosing spondylitis), Crohn's disease and dyskinesia. Read More
Stanford University and Acurex Therapeutics Corp. have discovered voltage-dependent T-type calcium channel subunit α-1H (Cav3.2) blockers reported to be useful for the treatment of neurodegeneration. Read More
The FDA has awarded orphan drug designation to Omega Therapeutics Inc.'s OTX-2002, a first-in-class epigenomic controller engineered to downregulate c-Myc (MYC), for the treatment of hepatocellular carcinoma (HCC). Read More
Inipharm Inc. has divulged thiazoles/isothiazoles acting as 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13) inhibitors reported to be useful for the treatment of nonalcoholic fatty liver disease, nonalcoholic steatohepatitis, cirrhosis, alcoholic liver disease, drug-induced liver injury and portal hypertension. Read More