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).
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.
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.
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.
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.
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.
Eterna Therapeutics Inc. has entered into a sponsored research agreement with a collaborator at The University of Texas MD Anderson Cancer Center (MD Anderson).
Hainan Sparkle Therapeutics Co. Ltd. has described ubiquitin carboxyl-terminal hydrolase 1 (USP1) inhibitors reported to be useful for the treatment of cancer.
Lifearc has identified pyrimidine compounds acting as serine/threonine-protein kinase ULK inhibitors reported to be useful for the treatment of chronic myeloid leukemia.