Capsida Biotherapeutics Inc. has obtained IND clearance from the FDA for CAP-003, an intravenously administered gene therapy, for Parkinson’s disease associated with GBA mutations (PD-GBA). A phase I/II trial will begin dosing in the third quarter of this year.
Epilepsy affects more than 70 million worldwide and while several drugs have proven effective for controlling seizures, around 30% of patients do not respond to them and their side effects can be intolerable.
4M Therapeutics Inc. has released promising results from completed GLP safety studies for its lead asset, 4MT-2001, supporting its potential to offer a safer treatment option to patients who are underserved by lithium and atypical antipsychotics.
Work at Sensorium Therapeutics Inc. has led to the identification of new serotonin transporter (SERT) inhibitors reported to be useful for the treatment of depression, anxiety and stress disorders.
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.