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.

At the American Heart Association’s Scientific Sessions, E-Therapeutics plc presented preclinical data for ETX-258, a Galomic siRNA being developed for the treatment of heart failure.

An international consortium of thousands of scientists is creating the Human Cell Atlas, a three-dimensional map of all the cells in the body. The goal is to understand all the cells that make up human tissues, organs and systems, which will enable multiple medical applications. This collection of cell maps is openly available for navigation at single-cell resolution, identified through omics analyses that reveal the tridimensional distribution of each cell.

Hypertrophic cardiomyopathy (HCM) is the most common genetic cardiovascular disorder with around 85% of people with HCM remaining undiagnosed. There are no treatments approved for nonobstructive HCM (nHCM) to date.

Avidity Biosciences Inc. has announced two new precision cardiology development candidates targeting rare genetic cardiomyopathies. AOC-1086 targets phospholamban (PLN) cardiomyopathy and AOC-1072 targets protein kinase AMP-activated noncatalytic subunit γ2 (PRKAG2) syndrome.

While the size of the market is enormous, drug development and treatments for women’s health care still lag behind what is offered for men. There has been a renaissance in the past few years, however, led by investors and companies that have wrestled with determining exactly what encompasses women’s health and how to meet its challenges.