The University of Sydney has patented P2X7 receptor antagonists reported to be useful for the treatment of stroke, amyotrophic lateral sclerosis, multiple sclerosis, Alzheimer’s disease, Huntington’s disease, atherosclerosis, diabetic retinopathy and myocardial infarction, among others.
Chronic kidney disease (CKD) is the 10th leading cause of death in the United States because of the increased risk for cardiovascular mortality. The major functional cell type of the kidney, tubular epithelial cells (TECs), possess a limited ability to regenerate tissues. Infections can cause proximal G2/M cell cycle arrest, senescence and paracrine secretion of the profibrotic cytokines transforming growth factor-β1 (TGF-β1) and connective tissue growth factor (CTGF), which stimulate extracellular matrix production ultimately leading to loss of the epithelial phenotype in TECs. Identification of targets controlling the G2/M cell cycle arrest in TECs may enable the development of therapeutics that can prevent CKD progression.
ΔF508 is the most prevalent mutation detected in patients with cystic fibrosis (CF), and it causes a loss of F508 within CFTR’s first nucleotide binding domain (NBD1). Researchers from Sionna Therapeutics Inc. recently reported the discovery and preclinical evaluation of novel small-molecule CFTR NBD1 stabilizers and CFTR assembly correctors as potential new agents for the treatment of CF.
Researchers from Children’s Hospital of Philadelphia presented data from a study that linked variants in DNA methyltransferase 1-associated protein 1 (DMAP1) to a novel neurodevelopmental disorder.
Anoat Therapeutics has raised €2 million (US$2.17 million) in seed funding to develop novel therapies for cystic fibrosis. The financing comes from a seed funding partnership between Adbio Partners SAS and Inserm Transfert SA.
Incretin mimetics have revolutionized the treatment of obesity and type 2 diabetes. These therapies have shown remarkable efficacy in promoting weight loss and improving glucose metabolism and cardiometabolic health. However, a significant drawback of these therapies is the concurrent loss of lean body mass (LBM), which can account for a substantial overall weight reduction (15% to 40%). The reduction in LBM affects resting metabolic rate, often leading to a weight loss plateau and other negative outcomes.
To address the absence of clinical trials evaluating immunotherapeutics for Acinetobacter baumannii infections, a team from the University of Texas at San Antonio conducted a study using immunoinformatics (EigenBio’s proprietary epitope prediction software) to identify peptides that contain both putative B- and T-cell epitopes from proteins associated with the pathogenesis of A. baumannii.
The NLRP3 inflammasome is a multiprotein complex that plays a pivotal role in regulating the innate immune system and inflammatory signaling. Upon activation by pathogen-associated molecular patterns (PAMPs), NLRP3 oligomerizes and activates caspase-1 which initiates the processing and release of pro-inflammatory cytokines IL-1β and IL-1 that are overproduced in many inflammatory disease conditions.
New isoindolines acting as mismatch repair endonuclease PMS2 inhibitors have been reported in a Neophore Ltd. patent as useful for the treatment of cancer.
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