Adding another name to an impressive roster of partners assembled over the past few years, Skyhawk Therapeutics Inc. inked a neurology-focused deal with Merck KGaA aimed at discovering small-molecule RNA-targeted drugs that could be worth more than $2 billion. Read More
One of the major obstacles to treating neurological disorders such as Alzheimer’s disease is the blood-brain barrier: drugs that are injected into the circulation usually do not enter the brain effectively. Researchers at Denali Therapeutics Inc., Biogen Inc. and the University of Minnesota have devised a vehicle for transporting antibodies against amyloid-β that can bypass the blood-brain barrier by binding to the transferrin receptor, which is expressed much more abundantly in capillaries than in arteries. Read More
Hangzhou Qihan Biotech Co. Ltd. has gained IND approval by the FDA for QT-019B, a universal, dual-target CAR T-cell therapy for refractory systemic lupus erythematosus (SLE). A phase I/II trial will be conducted in the U.S. Read More
Researchers from China Pharmaceutical University and collaborators have identified various naphthalene and indane derivatives as selective USP7 inhibitors. Read More
Rpxds Co. Ltd. has described α-ketoglutarate-dependent dioxygenase FTO inhibitors reported to be useful for the treatment of obesity, diabetes, atherosclerosis and more. Read More
Opus Genetics Inc. has obtained IND clearance from the U.S. FDA application for OPGx-BEST1, a gene therapy for the treatment of bestrophin-1 (BEST1)-related inherited retinal disease (IRD). Read More
Mutations in the GTPase KRAS drive proliferation of many types of tumors, and daraxonrasib (RMC-6236), which inhibits various KRAS mutants, can slow growth of pancreatic and lung cancers. Researchers at City University of New York and Weill Cornell Medical College have demonstrated that it also showed effects in osteosarcoma models expressing mutated KRAS. Read More
Researchers at Wenzhou Medical University reported the synthesis and preclinical evaluation of a novel series of betulin-succinate derivatives developed as potential anticancer agents. Betulin is a naturally occurring triterpene that shows significant potential as a lead compound for the development of novel antitumor agents, owing to its selective cytotoxicity against cancer cells and low toxicity toward normal cells. Read More
Rezolute Inc. has disclosed plasma kallikrein (KLKB1) inhibitors reported to be useful for the treatment of diabetic macular edema and uveitis. Read More
Sorafenib is a widely used treatment for hepatocellular carcinoma that has advanced beyond surgical resection, but only 30% of patients respond and many develop resistance within half a year. The resistance has been linked to showing greater ‘stemness’ characteristics of tumor cells, such as activation of signaling pathways mediated by Wnt/b-catenin, Hedgehog and PI3K-Akt. Read More
Viking Therapeutics Inc. has identified amylin analogues acting as calcitonin receptor (CALCR; CT-R) and amylin receptor agonists reported to be useful for the treatment of diabetes and obesity. Read More
Esophageal carcinoma (EC) is a tumor type of the digestive tract that accounts for about 300,000 deaths worldwide annually, with high aggressiveness and poor prognosis. Chinese researchers have investigated the implication of the enzyme aldehyde dehydrogenase 18 family member A1 (ALDH18A1) in EC. Read More
