At the United European Gastroenterology Week in Vienna, Redx Pharma plc presented data regarding their ROCK inhibitor RXC-008 for treating fibrostenotic Crohn’s disease. RXC-008 is restricted to the gastrointestinal tract, thus avoiding systemic exposure; ROCK1/2 kinases are involved in fibrosis and their blockade has been efficacious in several rodent models of fibrotic disease.

Researchers from Proqr Therapeutics NV recently presented preclinical data on AX-0810, a single-stranded RNA editing oligonucleotide (EON) targeting RNA coding for NTCP cotransporter

Pancreatic cancer is among the most lethal cancers and the fourth leading cause of cancer deaths worldwide, where 90% of cases fall into the pancreatic ductal adenocarcinoma (PDAC) type.

Satellos Bioscience Inc. has developed and presented data for a compound that targeted the process of muscle regeneration based on modulation of satellite stem cell polarity.

Facioscapulohumeral muscular dystrophy (FSHD) is a severe muscle disorder caused by aberrant DUX4 mRNA expression in skeletal muscle. DUX4 activates downstream target transcriptome, known as D4T, leading to myofiber loss and muscle weakness.

Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the sarcolemma and protects muscle cells during contraction.

It was hypothesized that the MEK1/2 inhibitor ATR-002 could reduce inflammation and clear Staphylococcus aureus infection during cystic fibrosis, thus potentially showing a dual effect.

Khalifa University and The University of Queensland presented preclinical data for a novel acid-sensing ion channel 1 (ASIC1) inhibitor, Hi1a, evaluated in models of multiple sclerosis (MS).