Oxford Nanopore Technologies Ltd. has established a new collaboration with UK Biobank to create the world’s first comprehensive, large-scale epigenetic dataset. The project will utilize Oxford Nanopore’s DNA/RNA sequencing technology to map the epigenome of 50,000 blood samples from UK Biobank to unlock insights into disease mechanisms, with the aim of improving patient outcomes.

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.

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.

Rome Therapeutics Inc. has presented data demonstrating the potential of its LINE-1 reverse transcriptase (RT) inhibitors.

Voyager Therapeutics Inc. has selected a lead development candidate, VY-1706, for its tau silencing gene therapy program in Alzheimer’s disease. The company anticipates filing an IND application with the FDA and a clinical trial application (CTA) with Health Canada for VY-1706 in 2026.

Ractigen Therapeutics Co. Ltd.’s RAG-21, a novel siRNA therapy targeting the FUS gene, has been awarded orphan drug designation by the FDA for the treatment of amyotrophic lateral sclerosis (ALS).