Meta Pharmaceuticals Inc. announced that the FDA has granted rare pediatric disease designation to its investigational new drug META-001-PH for the treatment of primary hyperoxaluria (PH), an autosomal recessive metabolic disorder in which oxalate is overproduced and deposited in the body.
Researchers from Wayshine Biopharm Holding Ltd. published new data regarding the preclinical systemic pharmacokinetics (PK), dose proportionality, and central nervous system (CNS) distribution of the novel ataxia telangiectasia mutated (ATM) inhibitor WSD-0628, being developed for the treatment of brain tumors.
Researchers from Changzhi Medical College and affiliated organizations presented the discovery of DYC-1, a novel dual-target inhibitor of dual-specificity tyrosine phosphorylation-regulated kinase 2 (DYRK2) and histone deacetylase 8 (HDAC8), being developed for the treatment of hepatocellular carcinoma (HCC).
Investigators at Washington University in St. Louis and Umea University have reported that the small molecule PS-757 was effective in culture and animal models against Streptococcus pyogenes, a gram-positive pathogen responsible for more than 500,000 deaths per year globally.
Scientists at the Novartis Institutes for Biomedical Research (NIBR) in Cambridge have discovered a small molecule that could be used as a therapy for sickle cell disease (SCD). The molecular glue oral degraders of the WIZ transcription factor called dWIZ-1 and dWIZ-2, bind to cereblon (CRBN) and WIZ, marking it for degradation and inducing the expression of fetal hemoglobin (HbF).
Researchers from the University of Minnesota have presented the discovery and preclinical characterization of a novel potent inhibitor of multidrug resistance-associated protein 1 (MRP1), ZW-1226, that is being developed as a therapeutic candidate for the treatment of multidrug resistant (MDR) cancers.
Researchers from East China Normal University and Fudan University presented the discovery of a new oral compound, Z-526, being developed as a treatment to alleviate chemotherapy-induced cachectic muscle loss.
Researchers from Xiamen University and affiliated organizations have presented the discovery of NB-1, a novel orally active Nur77 ligand being developed for the treatment of cancer.
Ensho Therapeutics Inc. launched in July after licensing a pipeline of four oral α4β7 inhibitors for inflammatory and gastrointestinal disorders, including inflammatory bowel disease (IBD), from EA Pharma Co. Ltd. “Millions of people worldwide are living with IBD,” Ensho founder, president and executive chair Neena Bitritto-Garg recently told BioWorld, “and while there are a number of approved medications to address the symptoms of IBD, it remains a difficult-to-treat disease with high relapse rates for a considerable proportion of patients.”
Researchers from University of Washington presented the discovery of novel bumped kinase inhibitor (BKI) derived compound (BKIDC), BKIDC-1553, being developed for the treatment of prostate cancer.
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