Our Pipeline

  • Duchenne Muscular Dystrophy Exon Skipping (Clinical Stage 3) 100% 100%
  • Duchenne Muscular Dystrophy                                   Gene Therapy (Preclinical Stage 2) 50% 50%
  • Spinal Muscular Atrophy                                        Antisense (Preclinical Stage3) 90% 90%
  • Ataxia telangiectasia                                            Antisense (Preclinical Stage1) 25% 25%