Formycon AG has published preclinical in vivo results for the development of its COVID-19 drug FYB-207. In in vivo studies, data were collected in two different models on the pharmacokinetics and efficacy of various constructs of the ACE2-Fc fusion protein, in order to select the most appropriate candidate to enter the clinic.
Satellos Bioscience Inc. has created, prioritized and advanced novel small-molecule drug candidates into further preclinical studies. The company's compounds have been designed to be potent and selective inhibitors of a particular kinase protein in the Notch pathway.
Results from a study carried out by the COVID-19 Host Genetics Initiative show that rare deleterious variants in the immune-system gene TLR7 make carriers more than five times more likely to have a severe SARS-CoV-2 infection. The TLR7 gene encodes Toll-like receptor 7 protein, which plays a protective role in the immune system by identifying pathogens and activating innate immunity.
By pairing the expression of an inhibitory ion channel with an activity-dependent promoter, researchers have developed the first on-demand gene therapy that specifically silenced hyperactive cells and prevented epileptic seizures. The channels are expressed when the promoter is turned on by excessive neuronal activity, and so “we can’t stop the first seizures,” Dimitri Kullmann told BioWorld.
The FDA has awarded orphan drug designation to Omega Therapeutics Inc.'s OTX-2002, a first-in-class epigenomic controller engineered to downregulate c-Myc (MYC), for the treatment of hepatocellular carcinoma (HCC).
Inipharm Inc. has divulged thiazoles/isothiazoles acting as 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13) inhibitors reported to be useful for the treatment of nonalcoholic fatty liver disease, nonalcoholic steatohepatitis, cirrhosis, alcoholic liver disease, drug-induced liver injury and portal hypertension.
Stanford University and Acurex Therapeutics Corp. have discovered voltage-dependent T-type calcium channel subunit α-1H (Cav3.2) blockers reported to be useful for the treatment of neurodegeneration.
Standigm Inc. has disclosed leucine-rich repeat kinase 2 (LRRK2), particularly LRRK2 G2019S mutant, inhibitors reported to be useful for the treatment of cancer, neurodegeneration, rheumatoid arthritis, tuberculosis, axial spondyloarthritis, radiographic (ankylosing spondylitis), Crohn's disease and dyskinesia.
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