Oryzon Genomics SA has been awarded a $498,690 grant by the ALS Association to support the regulatory preclinical development of ORY-4001, a highly selective histone deacetylase 6 (HDAC6) inhibitor, for amyotrophic lateral sclerosis (ALS).
Preveceutical Medical Inc. is working to accelerate the completion of its preclinical diabetes and obesity dual gene therapy program. The company is pursuing a dual-gene therapy strategy using in vitro validated Smart-siRNA sequences, which when paired with its proprietary bioresponsive lipid-nanoparticulate delivery systems, effectively target the PTPN1 gene.
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).
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