Pfizer Inc. has divulged AMP-activated protein kinase (AMPK) activators reported to be useful for the treatment of autoimmune diseases as well as inflammatory and gastrointestinal disorders.
Biosplice Therapeutics Inc. has discovered dual specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A) inhibitors reported to be useful for the treatment of cancer, diabetes and Alzheimer’s disease.
Researchers from Mariana Oncology Inc. reported preclinical data on MC-339, a DLL3-targeting macrocyclic peptide in several tumor models. Delta-like ligand 3 (DLL3) is overexpressed on the cell surface of small-cell lung cancer (SCLC), neuroendocrine prostate cancer (NEPC) and metastatic melanoma.
Bruton tyrosine kinase (BTK) inhibitors are commonly used in the treatment of hematological cancers. However, approximately 67% of patients with relapsed chronic lymphocytic leukemia (CLL) develop resistance to acalabrutinib and ibrutinib due to mutations in BTK, initially most often C481S.
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.
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