Gene-Editing Therapies for Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, which encodes dystrophin, a protein normally expressed in skeletal muscle.

Our scientists developed a method for treating DMD by using gene-editing proteins to edit the DMD gene to result in the production of a functional form of dystrophin protein.

Gene-Editing Therapies for Duchenne Muscular Dystrophy (DMD) is protected by U.S. Patent Number 10,752,918 (with additional patents pending in the U.S. and in other countries). Of note, certain claims of the granted patent are not limited by type of nucleic acid or formulation.

Example Applications

  • Alter dystrophin mRNA splicing, e.g., ablate the splice acceptor site upstream of a mutation-containing exon to generate functional dystrophin protein
  • Deliver the therapy directly to the patient’s skeletal muscle – avoid ex vivo cell manipulation
  • Combine with Factor’s Chromatin Context-Sensitive Gene-Editing Endonuclease for high-specificity in vivo gene editing
  • Combine with Factor’s ToRNAdo™ Nucleic-Acid Delivery System for high efficiency in vivo delivery – proven delivery to various cells and tissues ex vivo and in vivo