Data from a preclinical study assessing Mission Therapeutics Ltd.’s clinical-stage USP30 inhibitor MTX-652, for the prevention of transverse aortic constriction (TAC)-induced cardiac hypertrophy and remodeling, were recently presented.

Ferroptosis is a programmed cell death mechanism driven specifically by lipid peroxidation. Previous research found that ferroptosis is linked to the onset and progression of cardiovascular diseases, particularly those closely associated with vascular endothelial cell (VEC) injury.

Recent findings have unveiled that 15-HETE is the endogenous agonist for G protein-coupled receptor 39 (GPR39) in vascular smooth cells, so researchers hypothesized that GPR39 could work as a therapeutic target in pulmonary arterial hypertension and its deletion might prevent the development of the disease.

Potent siRNAs against B4GALT1 were designed in silico and screened in vitro (Huh7 cells, primary mouse, human hepatocytes) as well as in vivo (C57BL/6 mice) for the selection of a lead Galomic siRNA.

Investigators from Duke University hypothesized that hirudin-like protease inhibitors could be generated by linking exosite-binding aptamers with small-molecule active site inhibitors, thus generating more potent “EXACT” inhibitors.

At the Breakthroughs in Muscular Dystrophy special meeting held in Chicago Nov. 19-20, 2024, and organized by the American Society of Gene & Cell Therapy (ASGCT), multiple interventions at the RNA level were among the approaches that were presented to fight muscular dystrophies.

Pulmonary arterial hypertension (PAH) is a condition characterized by high blood pressure in the pulmonary arteries, potentially leading to heart failure. Previous research had found that knockout of Egln1 specific to endothelial cells, which encodes prolyl 4-hydroxylase-2, led to spontaneous PAH development.

Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA.