A study from Weill Cornell Medicine and The Jackson Laboratory has described the epigenetic mark SARS-CoV-2 left on immune system stem cells in the most severe cases of COVID-19 early in the pandemic, before the development of vaccines. In their work published in Cell on Aug. 18, 2023, the researchers presented a new methodology to analyze the epigenetic changes in monocytes and circulating hematopoietic stem and progenitor cells (HSPCs) that give rise to monocytes. That allowed corresponding author Steven Josefowicz and his colleagues to see if there were already changes induced by COVID-19 before HSPCs differentiated into monocytes. Read More
Phenylketonuria (PKU) is an inborn error of metabolism caused by heritable phenylalanine hydroxylase gene mutations that result in decreased metabolism of phenylalanine (Phe) causing brain damage. The most severe phenotype termed PKU occurs when untreated individuals achieve plasma Phe concentrations of >1200 microM/L, which are neurotoxic. Read More
Mysthera Therapeutics AG has launched to develop first-in-class, oral therapeutics to treat complex autoimmune diseases. Seed capital will be used to advance its portfolio of preclinical stage, pan-PIM kinase inhibitors to modulate multi-lineage immune cell functions. Read More
Protease form factor Xia (FXIa) is a therapeutic target for thrombosis prevention due to its well-known contribution to VTE and ischemic stroke in humans. Researchers from Janssen Pharmaceutica NV presented an antithrombotic target FXIa inhibitor obtained through a non-classical interactions strategy to improve the pharmacokinetic and pharmacological activity for the treatment of thrombosis. In the series, the potency was increased by using water-mediated hydrogen bonds to reduce polar hydrogen bond donors and using methyl to displace high-energy waters. Read More
Researchers at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have synthesized benzimidazole derivatives acting as serine/threonine-protein kinase SIK2 (QIK) and/or SIK1 (SNF1LK) and/or SIK3 (QSK) inhibitors reported to be useful for the treatment of rheumatoid arthritis, juvenile idiopathic arthritis, nonalcoholic steatohepatitis, primary sclerosing cholangitis, inflammatory bowel disease, atherosclerosis, type 2 diabetes and glomerulonephritis, among others. Read More
Ideaya Biosciences Inc. has announced FDA clearance of an IND for a GSK-sponsored phase I/II trial of GSK-101 (IDE-705), a small-molecule inhibitor of Polϴ helicase, in combination with GSK's PARP inhibitor niraparib tosylate monohydrate for the treatment of tumors with BRCA or other homologous recombination mutations or homologous recombination deficiency. Read More
Sichuan Haisco Pharmaceutical Co. Ltd. has identified phosphodiesterase PDE3 and phosphodiesterase PDE4 inhibitors reported to be useful for the treatment of chronic obstructive pulmonary disease (COPD) and asthma. Read More
Influenza A virus (IAV) is an RNA virus that can infect humans and also animals such as birds and pigs with high infectivity. Although there are several groups of anti-IAV drugs in the market, there is a need for new strategies due to the emergence of resistance and the high variability of the virus. One of the potential targets to watch in this disease is hemagglutinin (HA). Read More
Etern Biopharma (Shanghai) Co. Ltd. has divulged transcriptional coactivator YAP1 and TAZ/transcriptional enhancer factor (TEAD) interaction inhibitors reported to be useful for the treatment of cancer. Read More
Jiangsu Tasly Diyi Pharmaceutical Co. Ltd. has described Wee1-like protein kinase (Wee1) inhibitors reported to be useful for the treatment of inflammation, infection, cancer, and autoimmune disease. Read More
Canariabio Inc.'s MAb-AR20.5 has been awarded orphan drug designation by the FDA for pancreatic cancer. MAb-AR20.5 is an IgG1k type murine monoclonal antibody that binds specifically to the circulating and tumor-associated antigen (MUC1) expressed on pancreatic cancer cells. Read More
