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.
Obesity is a major public health concern, and effective treatments are still elusive. AMP-activated protein kinase (AMPK) is a crucial regulator of energy homeostasis and has been proposed as a target for metabolic diseases, including obesity. However, most available AMPK agonists target multiple tissues to activate AMPK, which can cause adverse effects.
Loss-of-function variants in the ELP1 gene are the most prevalent predisposing genetic factors in childhood medulloblastoma, accounting for about 30% of sonic hedgehog medulloblastoma (SHH-3 subtype).
Investigators from Anthem Biosciences Ltd. have published preclinical characterization data on their histone deacetylase (HDAC) inhibitor PAT-1102 for the potential treatment of cancer.
Pulmonary fibrosis is a potentially deadly lung disease characterized by progressive scarring and impaired lung function, with limited treatment options and a poor prognosis. Previous work found that targeting the activation, but not the production, of latent transforming growth factor 1 (TGF-β1) may offer therapeutic benefit in this condition.
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.
Monte Rosa Therapeutics Inc. has gained IND clearance from the FDA for MRT-8102, a NEK7-directed molecular glue degrader being developed to treat inflammatory conditions linked to NLRP3, IL-1β and IL-6 dysregulation.
Deepcure Inc. has nominated DC-15442, an oral selective STAT6 inhibitor, as its second development candidate. DC-15442 is designed to replicate the safety and efficacy of dupilumab, while offering an oral alternative to regular subcutaneous injections.
Pancreatic ductal adenocarcinoma (PDAC) is a type of cancer characterized by very poor prognosis and resistance to immunotherapy due to an immunosuppressive tumor microenvironment, which includes extensive desmoplasia and a dense stroma.
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