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BioWorld - Saturday, April 25, 2026
Home » Topics » Small molecule, BioWorld Science

Small molecule, BioWorld Science
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Cross section illustration of ion channel in cell membrane
Neurology/psychiatric

Nav1.8 inhibitor MSD-199 has activity in inflammatory, neuropathic pain models

Aug. 7, 2024
Researchers from Merck & Co Inc. have presented preclinical data for the novel Nav1.8 inhibitor MSD-199, being developed for the treatment of inflammatory and neuropathic pain.
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Multiple myeloma illustration
Cancer

Fibroblast growth factor traps have anti-myeloma activity

Aug. 6, 2024
Researchers from the University of Brescia have presented the discovery and preclinical characterization of novel fibroblast growth factor (FGF) trap small molecules, being developed for the treatment of multiple myeloma (MM).
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Kidney illustration
Nephrology

META-001-PH gets rare pediatric disease designation

Aug. 6, 2024
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.
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Test tubes, dropper
Cancer

Preclinical analysis to optimize dosing regimens for ATM inhibitor WSD-0628

Aug. 5, 2024
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.
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Illustration of tumor in the liver
Cancer

Dual-target inhibitor shows activity and safety in models of liver cancer

Aug. 5, 2024
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).
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Streptococcus pyogenes before and after treatment
Infection

PS-757 fights flesh-eating bacteria, biofilm

Aug. 5, 2024
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.
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Hematologic

WIZ glue degrader restores fetal hemoglobin

Aug. 5, 2024
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).
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Drug discovery illustration
Cancer

Deazaflavin analog reverses MRP1-mediated drug resistance

Aug. 2, 2024
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.
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Musculoskeletal

Z-526 alleviates chemotherapy-induced cachexia muscle atrophy in vivo

Aug. 2, 2024
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.
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3D illustration of cancer in crosshairs
Cancer

Nur77 ligandNB-1 has anticancer activity in preclinical models

Aug. 1, 2024
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.
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