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.
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.
Researchers have developed NCP-112 (Novacell Technology Inc.), a novel FPR2-selective synthetic heptameric peptide ligand, and tested it in preclinical models of atopic dermatitis.
Bluerock Therapeutics LP has reported the first in vitro and in vivo results on the company’s new cell therapy DA-02, consisting of human induced pluripotent stem cell (iPSC)-derived dopaminergic neurons.
Researchers from Innate Pharma SA recently presented preclinical data for IPH-6501, a novel CD20-targeting tetraspecific antibody-based natural killer (NK)-cell engager therapeutic being developed for the treatment of patients with B-cell non-Hodgkin lymphoma (B-NHL).
At the recent International Society for Stem Cell Research meeting, Vita Therapeutics Inc. presented the first preclinical results on VTA-100, a cell therapy consisting of induced pluripotent stem cell (iPSC)-derived satellite cells, for the treatment of limb-girdle muscular dystrophy 2A/R1.
Granulosa cell tumors (GCT) are a rare subtype of ovarian cancers, characterized by a slow clinical progression and high rates of late recurrence. With limited treatment options available apart from invasive surgery, targeted therapies would be key. In previous research, scientists at Hudson Institute of Medical Research in Australia demonstrated that targeting X-linked inhibitor of apoptosis protein (XIAP) using small-molecule inhibitors (called Smac-mimetics [SM]) in combination with other compounds could work as a therapeutic approach for GCT.
It has been previously demonstrated that in zebrafish, the activity of UXS1, a gene that encodes UDP-glucuronate decarboxylase 1, is essential for production and organization of skeletal extracellular matrix. Now, researchers from the University of Oslo and affiliated organizations have identified a novel pathogenic variant in UXS1.
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