Systemic sclerosis is an autoimmune disorder characterized by inflammation, vasculopathy and fibrosis, mainly affecting internal organs and the skin. There is still a lack of therapies for SSc-induced skin fibrosis.
BLR Bio LLC announced that the U.S. FDA has granted orphan drug designation to BLR-200, the company’s investigational therapy for the treatment of systemic sclerosis (SSc).
Researchers from Catholic University of Korea published data from a study that investigated the effect of miRNA-21a-5p on fibrosis development in systemic sclerosis (SSc). With the aim of assessing the pathological impact of miRNA-21a-5p on skin and lung fibrosis in vivo, a bleomycin-induced SSc murine model was developed, and the mice were hydrodynamically injected with plasmids containing pre-miRNA-21a-5p or anti-miRNA-21a-5p.
Previous research has shown that the three isoforms of transforming growth factor β (TGF-β) have distinct cellular expression patterns, with TGF-β2 and TGF-β3 expression being specifically elevated in human fibrotic lung and liver tissue. Since pan-TGF-β inhibition had proven to be toxic for chronic use, in recent work, researchers from Genentech Inc. aimed to assess whether specifically targeting one of the TGF-β isoforms could represent a novel therapeutic option for patients with chronic fibrotic disorders.
Japanese researchers have presented data from the biggest Asian genome-wide association study (GWAS) regarding susceptibility loci for systemic sclerosis (SSc), comprising a total of 1,428 cases and 112,599 controls and with an imputation reference panel containing more than 3,000 Japanese whole-genome sequencing data.
CD38 is the main NAD+-hydrolyzing enzyme, and it also catabolizes nicotinamide mononucleotide (NMN) and other extracellular NAD+ precursors prior to their intracellular transport for NAD+ biosynthesis.
Autotaxin (ATX) is an enzyme responsible for the production of lysophosphatidic acid (LPA), which plays a role in the pathogenesis of systemic sclerosis (SSc). Researchers from Mitsubishi Tanabe Pharma Corp. and colleagues have reported on the preclinical evaluation of MT-5562, a novel oral ATX inhibitor, as a therapeutic option for SSc.
Cantargia AB has completed a GLP toxicity study for its anti-inflammatory IL1RAP-binding antibody CAN-10, showing that CAN-10 was well tolerated when administered over 6 weeks.
A research team from Lille University Hospital in France has recently demonstrated that dual-energy computed tomography lung perfusion can provide valuable additional information for standard CT scans by visualizing perfusion changes in systemic sclerosis patients with normal or minimally infiltrated lung parenchyma. This clinical research team published their findings this summer in European Radiology.
