Novel RNA-Targeting Gene Therapy Shows Promise for Neuromuscular, Retinal Diseases

RNA-targeting gene therapy company Locanabio is debuting new data this week from 2 of their pipeline initiatives in neuromuscular and retinal diseases. The preclinical research highlights the company's novel approach to gene therapy, which involves delivering RNA-binding protein-based systems that correct dysfunctional RNA.

In an interview with GeneTherapyLive, Locanabio chief executive officer James Burns, PhD, shared details of the 2 presentations being shared at the 24th Annual Meeting of the American Society of Gene & Cell Therapy, the first which includes preclinical in vivo data demonstrating the safety and efficacy of their novel adeno-associated virus serotype 9-delivered PUF RNA-binding protein system for the elimination of toxic CUG repeats in a mouse model of myotonic dystrophy type 1. The second presentation centers around the company's CRISPR/Cas13d gene therapy system for the treatment of Usher syndrome type 2.