Forx Therapeutics AG has identified Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer.
The University of California has synthesized α-synuclein (SNCA) and/or amyloid-β protein and/or microtubule-associated protein tau (PHF-tau; MAPT) propagation inhibitors reported to be useful for diagnosis and treatment of multiple system atrophy, Parkinson’s disease and Alzheimer’s disease.
Shandong Quanzhong Biomedical Technology Co. Ltd. has disclosed receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of atherosclerosis, obesity, cancer, osteoarthritis, inflammatory bowel disease, fibrosis, psoriasis and Alzheimer’s disease.
Ovarian cancer remains unresponsive to immune checkpoint inhibitors due to its ability to suppress cytotoxicity from immune cells that are infiltrating the tumor, where the O-glycosylation pathway may be disrupted. Precision Biologics Inc. is developing PB-vcMMAE-5, an antibody-drug conjugate composed of the monoclonal antibody PB-223, which targets tumor-specific truncated core-2 O-glycans, conjugated to microtubule inhibitor MMAE and with a drug-to-antibody ratio of 3.92.
Many patients who survive myocardial infarction go on to experience heart failure. Researchers at Qilu Hospital of Shandong University found that bone morphogenetic protein 1 (BMP1) was upregulated in patients after myocardial infarction, as well as in a mouse model of the same condition.
The FDA has granted orphan drug designation to FRF-001, the FOXG1 Research Foundation’s lead gene therapy candidate for the treatment of FOXG1 syndrome. This follows the FDA’s earlier award of rare pediatric disease designation to the investigational therapy.
CSPC Pharmaceutical Group Ltd. has obtained clearance from China’s National Medical Products Administration (NMPA) to conduct clinical trials in China with SYH-2070 injection, a double-stranded small interfering RNA (siRNA) drug for the treatment of hypertriglyceridemia or mixed hyperlipidemia.
Convelo Therapeutics Inc. has presented data on their 3-β-hydroxysteroid-Δ8,Δ7-isomerase (EBP) inhibitor CVL-1001 as a remyelinating compound for treating multiple sclerosis.
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