Our platform provides uniquely powerful ways to repair mutated RNA molecules, reconstitute mutated genes with dual AAVs, or apply engineered sequences to an RNA of choice.
Many diseases are caused by mutations in genes that resist gene replacement or benefit from the safety features of our platform.
Many genes are too large to fit in a single AAV vector. Our platform supports the most efficient means to reconstitute full-length genes from dual AAV vectors
Apply engineered sequences to a chosen RNA, creating exquisitely cell type-specific expression of therapeutic proteins with applications in immuno-oncology.