Tetra Pharm Technologies Aps has completed preclinical testing and announced promising in vivo study results with its candidate compound, TPT-0301, which targets the endocannabinoid system.
Microtubule-targeting agents impact microtubule dynamics to induce mitotic arrest and tumor cell death. Tubulin is therefore a relevant target for the discovery of new agents acting as tumor neovasculature disruptors. Researchers from Nanjing Keygen Biotech Co. Ltd. have reported on the design and optimization of novel tubulin polymerization inhibitors with antiangiogenesis activity that led to the identification of [I] as the compound.
A large-scale genetic study found 26 risk loci for epilepsy, a chronic brain disease with multiple forms, not all of them heritable. The work, by more than 300 authors from the International League Against Epilepsy (ILAE), investigated seven different subtypes of this neurological condition. “There are over 100 genes that we know can harbor mutations that cause epilepsy,” the co-corresponding author Gianpiero Cavalleri told BioWorld. These genes have rare forms that cause that epilepsy. However, “this particular GWAS is focused more on common forms of epilepsy,” he said.
Messenger RNA, or mRNA, represents a relatively new class of therapeutics with the potential to prevent and treat a wide range of diseases. A well-known success story is of the mRNA vaccines that controlled the COVID-19 pandemic, which has fueled enthusiasm for the field. But biotechs are also developing mRNA candidates for several other infectious diseases, including malaria, tuberculosis and HIV, as well for cancer, autoimmune and allergic diseases. However, delivering nucleic acid therapeutics can be challenging, since mRNA cannot get into cells on its own.
Madrigal Pharmaceuticals Inc. has divulged thyroid hormone receptor β (THR-β) agonists reported to be useful for the treatment of familial hypercholesterolemia, hyperlipidemia, hypertriglyceridemia and nonalcoholic steatohepatitis.
Gasherbrum Bio Inc. has identified glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes, dyslipidemia, hyperglycemia, hypertension, obesity, stroke, myocardial infarction and gout, among others.
Enliven Therapeutics Inc. has synthesized HER2 (erbB2) inhibitors, including Exon 20 insertion (Ex20Ins) mutant inhibitors, reported to be useful for the treatment of cancer.
Vertex Pharmaceuticals Inc. has disclosed apolipoprotein L1 (APOL1) inhibitors reported to be useful for the treatment of focal segmental glomerulosclerosis and pancreatic cancer.