Netherton syndrome (NS) is caused by mutations in the serine protease inhibitor Kazal type 5 gene (SPINK5), which encodes lympho-epithelial Kazal-type-related inhibitor (LEKTI).
It is known that transient receptor potential cation channel subfamily V member 3 (TRPV3) is crucial for the modulation of skin homeostasis by regulation of Ca2+.
Mast/stem cell growth factor receptor KIT is a receptor tyrosine kinase that plays a key role in mast cell differentiation, proliferation, survival and activation.
Since utrophin compensates for lack of dystrophin in mdx mice, which results in a milder phenotype of muscular dystrophy compared to humans, the mdx/utrn-/- mouse has been developed to mimic early onset of muscle dystrophy, severe muscle weakness and premature death.
Researchers from the University of Colorado presented preclinical data for the myeloid leukemia cell differentiation protein Mcl-1 inhibitor S-64315, currently in phase I clinical development for the treatment of hematologic cancers.
Inhibition of OX40 is known to induce and maintain responses in moderate to severe atopic dermatitis (AD). Astria Therapeutics Inc. and Ichnos Sciences Inc. are developing STAR-0310, a YTE-modified (M252Y/S254T/T256E) monoclonal antibody targeting OX40.
The cannabinoid CB1 receptor inverse agonist INV-202 (monlunabant) is in clinical studies at Inversago Pharma Inc. as a potentially first-in-class drug for metabolic disorders, including diabetic kidney disease and obesity.
Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of renal failure characterized by development of multiple fluid-filled cysts linked to increased cAMP levels and fibrosis in the kidneys leading to progressive renal failure and need for dialysis or renal transplant.
Based on findings from recent studies that have identified MUC5AC in mice infected with the nematode Trichuris trichiura and during colitis, researchers from Medical University of South Carolina aimed to assess the effects of Clostridioides difficile infection on MUC5AC in the intestinal mucosa.
A proprietary Src homology 2 (SH2) platform of integrated technologies was applied for the discovery of REX-7117 and REX-5376, two highly potent, selective and orally available SH2 domain-targeting STAT3 inhibitors.
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