More than half of all hearing loss cases are hereditary. Myelin protein zero-like 2, encoded by MPZL2, is widely expressed in cochlear cells in the inner ear. Mutations in MPZL2 have been identified as the second most prevalent cause of mild to moderate hereditary hearing loss.
Graft Polymer (UK) plc has announced a research agreement between its partner, Awakn Life Sciences Corp., and the University of Nottingham. The agreement covers in vivo research to evaluate the potential of Graft Polymer and Awakn’s aminoindane NCE co-lead series to enhance social cognition and pro-social behaviors.
At the United European Gastroenterology Week in Vienna, Redx Pharma plc presented data regarding their ROCK inhibitor RXC-008 for treating fibrostenotic Crohn’s disease. RXC-008 is restricted to the gastrointestinal tract, thus avoiding systemic exposure; ROCK1/2 kinases are involved in fibrosis and their blockade has been efficacious in several rodent models of fibrotic disease.
Westlake Therapeutics (Hangzhou) Co. Ltd. and affiliated organizations have reported the design and preclinical characterization of novel erythrocytes conjugated to major histocompatibility complex (MHC) class I (MHC-I) protein, being developed for the treatment of cancer.
Researchers from Harvard Medical School and the Icahn School of Medicine at Mount Sinai found two rare, recurrent patterns of mutation in the post-mortem brain tissue of individuals with schizophrenia that could reflect prenatal mutational processes.
The founding CEO of Alnylam Pharmaceuticals Inc. is now leading the charge with newly launched City Therapeutics Inc., which just completed a $135 million series A financing. City’s executive chair, John Maraganore, will be in familiar territory as the new company plans to develop RNAi-based medicines using next-generation siRNA engineering. He expects dozens of these therapies to reach the market in a relatively short period of time, not just from City Therapeutics but from other companies. It’s a period in the development timeline that he finds reminiscent of the rise and development of monoclonal antibodies.
Nanjing Zaiming Pharmaceutical Co. Ltd. has described Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer.
Shandong Luye Pharmaceutical Co. Ltd. has divulged tachykinin NK3 receptor antagonists reported to be useful for the treatment of attention deficit hyperactivity disorder (ADHD), depression, pain, obesity, cognitive disorders, inflammatory disorders, psychosis and urinary incontinence, among others.
Nanjing Mingde New Drug Research Co. Ltd. has designed proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to a GTPase KRAS (G12D mutant)-targeting moiety via a linker for the treatment of cancer.
Scientists at Oncopia Therapeutics Inc. (dba SK Life Science Labs) and The University of Michigan have synthesized proteolysis targeting chimera (PROTAC) compounds comprising a cereblon E3 ubiquitin ligase-binding moiety coupled to a probable global transcription activator SNF2L2 (SMARCA2; BAF190B; SNF2-α) and transcription activator BRG1 (SMARCA4; BAF190A; SNF2-β)-targeting agent through a linker.
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