Leveraging RNA Targeting CRISPR Therapeutics to Target Myotonic Dystrophy

Time: 11:55 am
day: Day Two


• Outlining the principles of Locana’s RNA Targeting approach and outlining its use in myotonic dystrophy (DM1) preclinical models and outlining behavioral and molecular biomarkers

• Global alternative splicing defects in DM1 to explore a battery of molecular endpoints, and how these correlate with regression of symptoms

• Evaluating data across different modalities including Cas9 and Cas13d

• Showing the breadth and opportunity as to how these systems are effective in the treatment of DM1 in animal studies, testing the safety and efficacy