Investigators at Cold Spring Harbor Laboratory have discovered a method of switching cancerous rhabdomyosarcoma cells back into normal muscle cells that could lead to the development of a treatment for this rare aggressive childhood cancer. Read More
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. Read More
Researchers from Ashvattha Therapeutics LLC recently reported on ASH-41020, a new dendranib compound acting as a CSF-1R inhibitor with anti-inflammatory and immunomodulatory effects. Read More
Beactica Therapeutics AB has selected BEA-17 as a preclinical candidate from its lysine demethylase 1 (LSD1) program targeting aggressive brain tumors and other cancers, and IND-enabling toxicology studies will now be initiated. Read More
Vertex Pharmaceuticals Inc. has disclosed apolipoprotein L1 (APOL1) inhibitors reported to be useful for the treatment of focal segmental glomerulosclerosis and pancreatic cancer. Read More
Uniqure NV has received FDA clearance of its IND application for AMT-260, the company’s gene therapy candidate that represents a potential one-time administered approach to treating refractory mesial temporal lobe epilepsy (MTLE). Read More
Enliven Therapeutics Inc. has synthesized HER2 (erbB2) inhibitors, including Exon 20 insertion (Ex20Ins) mutant inhibitors, reported to be useful for the treatment of cancer. Read More
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. Read More
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. Read More
Molecular glue degraders are compounds intended to prompt the degradation of E3 ubiquitin ligases by engaging and modifying their surface. Molecular glue-induced protein degradation is considered an emerging strategy in drug discovery. Read More
Researchers from Bristol Myers Squibb Co. recently discussed the discovery and preclinical characterization of a series of Toll-like receptor 7 (TLR7) agonists with potential as anticancer drug candidates. Read More
Biogen Inc. has described 3-β-hydroxysteroid-δ(8),δ(7)-isomerase (EBP) inhibitors reported to be useful for the treatment of multiple sclerosis. Read More
Researchers from Genentech Inc. have reported the discovery of new cyclin-dependent kinase 2 (CDK2) inhibitors as potential anticancer agents. Read More