CAN204

**CAN204** is a next-generation dual AAV vector gene therapy in preclinical development for Duchenne muscular dystrophy (DMD) and other dystrophinopathies. It employs the proprietary StitchR™ RNA Assembly Technology, exclusively licensed from Scriptr Global Inc., to overcome AAV payload limitations by delivering left and right halves of large gene sequences via two independent vectors; these halves are seamlessly stitched together at the mRNA level by ribozymes to produce functional full-length proteins. In mdx mouse models, CAN204 expresses high levels of midi-dystrophin—approximately twice the size of approved micro-dystrophins and over half the size of native dystrophin—resulting in significant improvements in muscle and heart health.[1][3]