The archetypal UbiB protein COQ8 has human homologues COQ8A and COQ8B, both with well-established connection to human disease, with inactivating mutations in COQ8A resulting in autosomal recessive cerebellar ataxia. Researchers from the University of Wisconsin-Madison and affiliated organizations have now recently reported the discovery of small-molecule inhibitors of COQ8A.
Researchers from Nalo Therapeutics Inc. presented the discovery and preclinical evaluation of a novel orally bioavailable and brain-penetrant epidermal growth factor receptor (EGFR) inhibitor, NX-019, being developed as a potential therapeutic agent to treat EGFR-mutant solid tumors.
Researchers from Suven Life Sciences Ltd. presented the discovery and preclinical characterization of a novel muscarinic acetylcholine M4 receptor positive allosteric modulator (PAM), SUVN-L1305022.
Researchers from Cerevel Therapeutics LLC presented data from a study that aimed to evaluate preclinical antipsychotic properties of M4 agonism while minimizing off-target side effects using novel muscarinic acetylcholine M4 receptor full and partial agonists, CV-0000042 (CVL-042) and CV-0000071, respectively.
Maze Therapeutics Inc. recently presented data from preclinical studies of a small-molecule APOL1 pore function inhibitor, MZ-301, describing the compound’s in vitro and in vivo activity. APOL1 G1 and G2 genetic variants are associated with an increased risk of progressive kidney diseases in African ancestry people. There are no APOL1-targeted therapies addressing the underlying driver of these diseases.
Rhabdomyosarcoma (RMS) occurs in 3% of all pediatric cancers. Aberrant Hedgehog (Hh) activation can be ligand-dependent or -independent, but current clinical Hh inhibitors targeting SMO are effective only for ligand-independent tumors. Researchers at Vall d’Hebron Institut de Recerca, Barcelona, Spain, developed a targeted therapeutic for Hh ligand-dependent tumors.
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