RNA targeting gene therapeutic / Locana - LARVOL DELTA

Locanabio’s RNA-Targeting Gene Therapy May Offer Durable Response for DM1 Patients (BioSpace) - "Locanabio...showed promising results with an RNA-targeting therapy against the toxic RNA that is at the heart of myotonic dystrophy type 1 (DM1)...'This study is the first of its kind to show in vivo ability to use RNA targeting proteins to manipulate RNA to correct its message to treat disease in an animal model'....Locanabio plans to begin IND-enabling studies in 2021."

IND • Preclinical • Myotonic Dystrophy

Treatment with RNA-Targeting Gene Therapy Reverses Molecular and Functional Features of Myotonic Dystrophy Type 1 in Mice (PRNewswire) - "Locanabio...today announced that results from a preclinical study of the company's therapeutic systems for the potential treatment of myotonic dystrophy type 1 (DM1) were published in Nature Biomedical Engineering. For the full article, titled 'The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy'....These results are consistent with earlier findings from several in vitro studies in muscle cells derived from DM1 patients...represent a promising advance and a novel scientific approach for a group of patients who represent a major unmet medical need..."

Preclinical • Myotonic Dystrophy