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.
Sparingvision SAS has obtained FDA clearance for its IND application for SPVN-06, its lead gene-independent therapy for the treatment of retinitis pigmentosa (RP). SparingVision has also submitted a clinical trial authorization (CTA) application to the French regulator (ANSM), which is currently under review.
Opus Genetics Inc. has received FDA clearance for its IND application for a first-in-human phase I/II trial of OPGX-001 in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5). The trial is due to start in the U.S. early next year (ClinicalTrials.gov Identifier NCT05616793).
University College Cardiff has divulged heteroaryl compounds acting as GABA(A) receptor subunit α5 (GABRA5) negative allosteric modulators reported to be useful for the treatment of cognitive disorders.
Researchers from Shouyao Holdings (Beijing) Co. Ltd. presented the discovery of novel Wee1-like protein kinase inhibitors being investigated as agents for the treatment of cancer.
Researchers from Anhui Medical University published data from a study that aimed to assess the antifibrotic effects of the transforming growth factor-β (TGF-β) receptor type I (TGFBR1/ALK5) inhibitor Cpd-0225.
RSS
