NRG Therapeutics Ltd., has nominated NRG-5051 as its first development candidate, and secured a $5 million grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support its preclinical development of as a disease-modifying treatment for Parkinson’s disease.
Hepatoblastoma is the most common pediatric liver cancer and has limited therapeutic options, with platin-based therapy showing severe side effects. WNTinib is a kinase inhibitor with specificity for β-catenin (CTNNB1)-mutated hepatocellular carcinoma (HCC) and is a therapeutic approach that shows promise for treating hepatoblastoma.
Collagen-producing activated myofibroblasts, which are key effector cells in fibrogenesis in different organs, express high levels of platelet-derived growth factor receptor β (PDGFRβ).
The FDA has granted orphan drug designation to Capsida Biotherapeutics Inc.’s CAP-002, an investigational gene therapy for the treatment of developmental and epileptic encephalopathy due to syntaxin-binding protein 1 (STXBP1) mutations.
Methionine adenosyltransferase 2A (MAT2A), the rate-limiting enzyme in the methionine cycle, catalyzes the formation of S-adenosylmethionine (SAM) from methionine and adenosine triphosphate (ATP).
Malaria is caused by Plasmodium species that infect hundreds of millions of people annually. Among the plasmodia, Plasmodium falciparum is considered the most dangerous due to frequent severe clinical complications and high mortality rates. Researchers from the University of California at Riverside described the discovery and mechanism of action of MED-6189, a kalihinol analog effective against drug-sensitive and drug-resistant P. falciparum strains in vitro and in vivo.
Backed by AI technology, Aigen Sciences Inc. raised ₩12 billion (US$8.8 million) in a series A financing round to further advance its cancer and rare disease drug pipelines. Aigen said Oct. 16 that the series A round was joined by existing investors Partners Investment, Quad Investment Management and Medytox Venture Investment, as well new investors Premier Partners, K2 Investment Partners and Scale Up Partners.
Jiangsu Alicorn Pharmaceutical Co. Ltd. has described AP2-associated protein kinase 1 (AAK1) inhibitors reported to be useful for the treatment of Alzheimer’s disease, bipolar disorder, Parkinson’s disease, schizophrenia, diabetic peripheral neuropathy and postherpetic neuralgia.
Shanghai Yidi Biotechnology Co. Ltd. has identified dihydrothienopyrimidine compounds acting as phosphodiesterase PDE4B2 and/or phosphodiesterase PDE4D2 inhibitors reported to be useful for the treatment of autoimmune disorders, metabolic diseases, neurological diseases and inflammatory disorders.
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