The misassembly of transthyretin (TTR) into toxic amyloid aggregates leads to a variety of degenerative disorders known as TTR amyloidosis (ATTR). Researchers from the Universitat Autònoma de Barcelona and collaborators recently reported on the design and preclinical characterization of PITB, a TTR-selective kinetic stabilizer with potential for the treatment of ATTR.
A multi-institutional research team has suggested that aberrant TDP-43 processing of the pre-mRNA of a microtubule-associated protein, stathmin-2 (STMN2), may be the primary contributor to amyotrophic lateral sclerosis (ALS).
Idorsia Pharmaceuticals Ltd. researchers have prepared and tested new thiazoloaryl-methyl substituted cyclic hydrazine-N-carboxamide derivatives acting as orexin OX1 and OX2 receptor antagonists. They are reported to be useful for the treatment of substance abuse and dependence, anxiety, cognitive, sleep, eating and mood disorders.
An F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. patent describes indole derivatives acting as EGFR L858R mutant, T790M/L858R double mutant, T790M/L858R/C797S triple mutant and/or L858R/C797S double mutant allosteric inhibitors reported to be useful for the treatment of non-small-cell lung cancer (NSCLC).
Work at Wake Forest University has led to the development of an H2O2-responsive doxorubicin hybrid codrug able to release H2S (acting as cardioprotective agent). They are reported to be useful for the treatment of cancer.
Ventus Therapeutics US Inc. has disclosed oxoindolinyl amide derivatives acting as NLRP3 inflammasome inhibitors. As such, they are reported to be useful for the treatment of cancer, metabolic, autoimmune diseases, liver, renal, respiratory, cardiovascular and inflammatory disorders, among others.
A Character Biosciences Inc. patent describes polypeptides acting as low-density lipoprotein receptor (LDLR) ligands and reported to be useful for the treatment of age-related macular degeneration (AMD).
Synthesis and optimization of substituted 2-amino[1,2,4]-triazolopyrimidines and related heterocycles by Wuyi University investigators has led to the identification of compound [I], and its water-soluble phosphate sodium salt prodrug, compound [II].
Trypto Therapeutics GmbH scientists have discovered novel tryptophan hydroxylase (TPH) inhibitors, which are being developed for the treatment of colorectal cancer.