Rgenta Therapeutics Inc. has synthesized new 2,3-dihydropyrollopyridine carboxamide compounds acting as PMS1 protein homolog 1 splicing modulators. They are reported to be useful for the treatment of amyotrophic lateral sclerosis, Friedreich ataxia, myotonic dystrophy, fragile X syndrome, frontotemporal dementia, Fuchs dystrophy, Huntington’s disease and spinal and bulbar muscular atrophy, among others.
N4-Acetylation of cytidine (ac4C) is an mRNA modification that enhances cellular mRNA stability and translation. Most eukaryotic organisms catalyze ac4C using a homologue of human N-acetyltransferase 10 (NAT10). Recent work has suggested the involvement of alterations in NAT10-mediated ac4C in several diseases, including autoimmune disorders, infections, inflammation and cancer.
Solve FSHD and Modalis Therapeutics Corp. have established a strategic collaboration to develop an innovative therapy for facioscapulohumeral muscular dystrophy (FSHD).
Merck Sharp & Dohme LLC has identified compounds acting as potassium/sodium hyperpolarization-activated cyclic nucleotide-gated channel 1 (HCN1) and/or 2 (HCN2) blockers reported to be useful for the treatment of neuropathic pain, tinnitus, psychiatric and neurological disorders.
Mindimmune Therapeutics Inc. has been awarded a grant by Rhode Island Life Science Hub to accelerate preclinical development work on MITI-101 for the treatment of mild to moderate Alzheimer’s disease. The award will accelerate development work needed to start first-in-human studies.
Scientists at Shanghai Tech University have used the scaffold-hopping artificial intelligence model Geminimol to identify N-methyl-D-aspartate (NMDA) receptor ligands that show selectivity and specificity. The researchers found that GM-10 could be a potent inhibitor of the GluN1/GluN3A subunits of the NMDA receptor, demonstrating the utility of this technique to develop new drugs.
Solvonis Therapeutics plc has completed synthesis of development candidates for its SVN-SDN-014 program, a novel series of serotonin, dopamine and noradrenaline modulators.
Casma Therapeutics Inc. has nominated its first development candidate, CSM-101, a first-in-class TRPML1 agonist. CSM-101 is being developed for the treatment of Gaucher’s disease patients with Parkinson’s disease, with the potential to expand into GBA-associated Parkinson’s disease and broader Parkinson’s disease populations.
Evotec International GmbH has divulged NLRP3 inflammasome inhibitors reported to be useful for the treatment of asthma, cryopyrin-associated periodic syndromes, hypertension, Alzheimer’s disease, rheumatoid arthritis, diabetes, multiple sclerosis and Parkinson’s disease.