Development of a novel lipid nanoparticle with widespread photoreceptor delivery of ceDNA & mRNA cargos
Vector Optimization for Non-Viral Antibody Gene Therapy and Expression of Human Monoclonal Antibodies in Mice
Non-Viral Gene Delivery of Human FVIII to Hemophilia A Mice and Non-Human Primates
When Size Matters: FVIII Construct Optimization Leveraging ceDNA, a Non-Viral Gene Therapy Platform