RNA-Targeting Lends Renewed Focus to Gene Therapy Development Efforts

No one can deny the lightning speed at which the gene and cell therapy space is advancing on a daily basis, but the ongoing evolution is particularly sweet for long-time industry players like James Burns, PhD, chief executive officer of Locanabio, Inc., a clinical-stage company focused on RNA-targeting gene therapies.

Locanabio recently presented new data at the 24th Annual Meeting of the American Society of Gene & Cell Therapy, including an RNA-targeting CRISPR/Cas13d gene therapy approach for skipping USH2A exon 13 for the treatment of retinal disease, as well as preclinical in vivo data demonstrating safety and efficacy of their novel PUF RNA-binding protein system delivered via an adeno-associated virus serotype 9 (AAV9) vector in eliminating toxic CUG repeats in a mouse model of myotonic dystrophy type 1.

In an interview with GeneTherapyLive, Burns commented on the changing landscape of gene therapy research, adding a poignant reminder of why the field continues to push forward.