Novartis AG has synthesized new sodium channel protein type 5 subunit alpha (SCN5A; Nav1.5) blockers reported to be useful for the treatment of heart failure, long QT syndrome, hypertrophic cardiomyopathy, angina pectoris, myotonia, ventricular tachycardia, atrial and ventricular fibrillation, among others.
Tyligand Bioscience Ltd. has prepared and tested tyrosine-protein phosphatase non-receptor type 11 (PTPN11; PTP-2C; SHP-2) inhibitors reported to be useful for the treatment of cancer.
KRAS G12C mutations, present in approximately 13% of lung adenocarcinoma cases, are associated with limited responses to current KRAS G12C inhibitor treatments such as adagrasib and sotorasib, with overall response rates ranging between 34% and 43%.
Intercellular communication between tumor cells and cancer-associated fibroblasts (CAFs) plays a key role in tumorigenesis and progression, and CAFs generate an extracellular matrix that provides a repository for factors that promote cancer progression. Alpha Beta Therapeutics Inc. researchers aimed to investigate the mechanisms by which CAFs facilitate tumor initiation and tumorigenesis of pancreatic cancer.
Myocardial fibrosis is a key process for heart failure development that may lead to severe phenotypes, including cardiac arrest or sudden death. Vasorin (VASN) is a protein with high expression in the cardiovascular system and its upregulation had been found to protect smooth muscle cells from calcification.
It was hypothesized that the crosstalk between Toll-like receptors, especially Toll-like receptor 4 (TLR4), and serotonergic machinery during inflammation may be an important factor for understanding the neuroimmune mechanisms participating in inflammatory bowel disease pathogenesis.
Stargardt disease, the most common form of juvenile macular degeneration, results from biallelic mutations in the ABCA4 gene. In a recently published study, researchers at the Institute of Molecular and Clinical Ophthalmology Basel and collaborators proposed a novel dual AAV vector system to deliver a split-intein adenine base editor for precise correction of the most common Stargardt mutation, c.5882G>A (p.Gly1961Glu).
Researchers from Columbia University Irving Medical Center published data from a study that assessed GiGA1, a selective G protein-gated inwardly rectifying potassium channel (GIRK) activator, in physiologically and clinically relevant ex vivo mouse models of tumor-associated and provoked seizures.
Dewpoint Therapeutics Inc. has nominated a second development candidate, DPTX-3496. The oral, small-molecule condensate modulator targets β-catenin to address Wnt-driven colorectal cancer, triple-negative breast cancer and non-small-cell lung cancer. IND-enabling studies have commenced, with an IND filing planned in the second half of this year.
Multiple organ dysfunction syndrome is a life-threatening condition derived from heat stress or sepsis, among other conditions, that can lead to irreversible organ damage and death if left untreated. Gasdermin (GSDMD) is an executor protein that causes cell lysis, facilitates the release of inflammatory mediators and is involved in pyroptosis, apoptosis and necroptosis processes leading to inflammatory cell death or PANoptosis.