Novel RNA-Targeting Gene Therapy Shows Promise for Neuromuscular, Retinal Diseases
The CEO of Locanabio shares details of 2 presentations the company is making this week at the 24th Annual Meeting of the American Society of Gene & Cell Therapy.
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.
Urgency of Funding and Equity in Neuromuscular Research and Care
March 31st 2025Robert Califf, MD, MACC, a cardiologist and former FDA commissioner, discussed the necessity of policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities.
