Investigators from Biomarin Pharmaceutical Inc. have presented the first preclinical data for BMN-293, a novel adeno-associated virus (AAV) gene transfer vector carrying the MYBPC3 gene. MYBPC3 mutations can cause hypertrophic cardiomyopathy (HCM), a heart medical condition characterized by an abnormally thick myocardium, which makes it more difficult for the heart to pump blood.
Researchers from Hokkaido University (Japan) and colleagues have identified 2-thiouridine (s2U) as a broad-spectrum antiviral ribonucleoside analogue, by phenotypic screening of a library of 753 nucleoside analogues for antiviral effects. s2U showed antiviral activity against several ssRNA+ viruses, including DENV, SARS-CoV-2 and its variants of concern.
Heterozygous familial hypercholesterolemia (HeFH) is caused by mutations in the LDL receptor gene, resulting in unusually high levels of low-density lipoprotein (LDL-C) in serum. Researchers from Epigenic Therapeutics Co. Ltd. presented the discovery of an epigenetic modulation therapeutic, EPI-001, for HeFH.
Vyne Therapeutics Inc. has released promising new preclinical data on its oral small-molecule BD2-selective BET inhibitor, VYN-202, in preclinical models of psoriasis and rheumatoid arthritis. In a well-established model, a psoriasis phenotype was induced in BALB-C mice, and treatment was administered intraperitoneally with either VYN-202, the allosteric TYK2 inhibitor Sotykto (deucravacitinib, Bristol Myers Squibb Co.) or placebo.
During the IDWeek conference held in Boston earlier this month, presentations on Climate Change were spread throughout the program. Some talks were on the direct effects of weather on infectious agents. Others discussed what healthcare workers could do to mitigate the effects of climate change, from antibiotic stewardship to decarbonization of day to day operations.
A Hangzhou Highlightll Pharmaceutical Co. Ltd. patent describes new NLRP3 inflammasome inhibitors reported to be useful for the treatment of cancer, inflammation, liver and metabolic disease, neuroinflammation, immunological, cardiovascular and renal disorders.
Yiteng Pharmaceutical Industry (Taizhou) Co. Ltd. has disclosed new inhibitors of cyclin-dependent kinase (CDK), particularly CDK7, reported to be useful for the treatment of cancer.
Indolinone derivatives acting as mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1) inhibitors are detailed in a recent Chengdu Baiyu Pharmaceutical Co. Ltd. patent and described as potentially useful for the treatment of cancer.
FL2022-001 Inc. has patented new 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) inhibitors reported to be useful for the treatment of liver fibrosis and nonalcoholic steatohepatitis.
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