Steric hindrance and electrostatic interactions often prevent the subsequent development of clinically relevant nanoparticles to the in vivo stage. Researchers at the Shanghai Institute of Materia Medica, Chinese Academy of Sciences, have now demonstrated the development of nanoparticles exhibiting pH-sensitive properties triggering the stretching of peptides to reveal accessible liver-targeted ligands that can deliver biologically active peptides in vivo.
Axon loss is an initiating event common to several neurodegenerative disorders. In healthy axons, SARM1 (sterile α and Toll/IL-1 receptor motif-containing 1) activity, crucial for programmed axon degeneration, is restrained by the NAD+ biosynthetic enzyme NMNAT2.
Chengdu Baiyu Pharmaceutical Co. Ltd. has synthesized NLRP3 inflammasome inhibitors acting as pyroptosis inhibitors reported to be useful for the treatment of cancer, autoimmune, cardiovascular, endocrine, respiratory, renal, neurological and gastrointestinal disorders, among others.
Medshine Discovery Inc. has disclosed alkyl carboxylic acid compounds acting as soluble guanylate cyclase (sGC) activators reported to be useful for the treatment of chronic kidney disease.
Charcot-Marie-Tooth disease 2A (CMT2A) is a common hereditary motor and sensory neuropathy of the peripheral nervous system caused by mutations in the mitofusin 2 gene (MFN2). CMT2A is characterized by progressive axonal degeneration without myelin involvement, predominantly affecting the distal limbs, but the mechanisms underlying the axonal pathology remain unclear.
The high and specific expression of Kita-Kyushu lung cancer antigen 1 (KK-LC-1) in multiple types of tumors makes it an ideal target for drug delivery.
Researchers from BioCryst Pharmaceuticals Inc. have developed a series of plasma kallikrein inhibitors with potential for the treatment of hereditary angioedema (HAE).
Scientists from the Icahn School of Medicine at Mount Sinai have found a sexual dimorphism of depression based on the different expression of a molecule that could be developed as a therapeutic strategy. “There is a big sex difference in depression. Women are much more likely to have depression than men. They tend to have different subsets of symptoms. They tend to respond better to different antidepressants, and the depression tends to be more severe,” Orna Issler, the first author of the study and a postdoctoral researcher at the Nash Family Department of Neuroscience, Friedman Brain Institute, Icahn School of Medicine at Mount Sinai, told BioWorld. Their project, directed by Eric Nestler, a professor of neuroscience and director of the Friedman Brain Institute at the Icahn School of Medicine at Mount Sinai, had the aim to understand the biology of these sex differences of depression and to find therapeutic targets for it.
George Washington University has described N-ACYL fosmidomycin prodrug analogues acting as 1-deoxy-D-xylulose-5-phosphate reductoisomerase (Dxr; IspC) (Mycobacterium tuberculosis) and Dxr (Plasmodium falciparum) inhibitors reported to be useful for the treatment of malaria and tuberculosis.