Contrary to current opinion, genomic instability is not the origin of cancer in patients with short telomere syndromes (STSs), researchers from the Johns Hopkins University School of Medicine reported April 2, 2023, in Cancer Cell. Instead, short telomeres appeared to cause memory T-cell dysfunction that increased the risk of a small number of tumor types in individuals with STS. Such syndromes can cause premature aging of different physiological systems. Read More
A team of researchers from Harvard University has developed a suite of Escherichia coli Nissle 1917 (EcN), named PRObiotic type 3 secretion E. coli therapeutic (PROT3ECT), engineered to secrete a TNF-α nanobody directly into the gut to reduce inflammation in a chemically induced inflammatory bowel disease (IBD) mouse model. Read More
Merck & Co. revealed the structure of an orally active and potent proprotein convertase subtilisin/kexin-type 9 (PCSK9) inhibitor macrocyclic peptide, MK-0616, which is being developed for the potential treatment of hypercholesterolemia and atherosclerosis. Read More
Scorpion Therapeutics Inc. has named STX-241 as its next development candidate. STX-241 is an orally bioavailable, highly selective, central nervous system (CNS)-penetrant, fourth-generation EGFR tyrosine kinase inhibitor (TKI) designed to inhibit C797S mutations with a co-occurring EGFR exon 19 deletion or exon 21 mutation (double mutant) in non-small-cell lung cancer (NSCLC). Read More
Frontotemporal dementia (FTG-GRN) is a fatal neurodegenerative disease caused by heterozygous mutations in the GRN gene, encoding progranulin (PRGN). Researchers at Arkuda Therapeutics Inc. reported on ARKD-104, a novel potent orally bioavailable PGRN-enhancing compound that increased PRGN secretion and lysosomal granulins in multiple relevant cell lines. Read More
Black Diamond Therapeutics Inc. has synthesized quinoline derivatives acting as Raf kinase B (B-Raf) mutant inhibitors reported to be useful for the treatment of cancer. Read More
Shanghai Institutes for Biological Sciences and Shanghaitech University have identified dopamine D2 receptor and/or 5-HT receptor agonists reported to be useful for the treatment of depression and schizophrenia. Read More
Primary biliary cholangitis (PBC) is the most common autoimmune liver disease; several genome-wide association studies have suggested chromosome locus 19p13.3 is associated with PBC pathology. Chinese researchers conducted a case-control study to unveil genetic variants in the 19p13.3 locus associated with PBC. The meta-analysis included 1,931 individuals with PBC and 7,852 controls. Read More
Alternative splicing aids in maintaining the complexity of the transcriptome in higher organisms, and small nuclear RNAs (snRNAs), which are major components of the spliceosome, are processed by the integrator complex. Dysfunctions of integrator complex lead to immature snRNA production and may result in secondary defects in mRNA splicing. Read More
Medshine Discovery Inc. has divulged halogen-substituted pyridazinone compounds acting as short transient receptor potential channel 5 (TRPC5) antagonists reported to be useful for the treatment of hypertensive nephropathy. Read More
Evergreen Discovery (Evergreen Theragnostics Inc.) and Orbit Discovery Ltd. have entered into a collaboration to identify specific cell-targeting peptides and advance the development of novel radiopharmaceuticals. Read More
Eli Lilly and Co. has described aryl hydrocarbon receptor (AhR) agonists reported to be useful for the treatment of psoriasis, ulcerative colitis, Crohn’s disease, graft-vs.-host disease and multiple sclerosis. Read More
