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