dystrophin-encoding oRNA

dystrophin-encoding oRNA is an investigational circular RNA (oRNA) therapeutic developed by Orna Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). The therapy utilizes a proprietary engineered circular RNA molecule that encodes the dystrophin protein, encapsulated in lipid nanoparticles (LNPs) for systemic delivery. Unlike linear mRNA, oRNA's closed-loop structure enhances stability and protein expression while potentially minimizing the innate immune response. This platform is particularly significant for DMD because it can accommodate the full 12-kilobase dystrophin coding sequence, overcoming the cargo size limitations of adeno-associated virus (AAV) vectors. By delivering the genetic instructions for full-length or Becker-variant dystrophin, the treatment seeks to restore the essential structural protein in muscle fibers, offering a potentially superior alternative to truncated micro-dystrophin gene therapies.

Other names

oRNA-dystrophincircular RNA encoding dystrophin