Intracellular therapeutics pipeline

Using our proprietary EEV™ platform, we are creating a diverse and expanding pipeline of oligonucleotide, antibody and enzyme-based programs. Our lead programs are in neuromuscular diseases, including Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). Our modular approach supports efficient expansion of development into multiple therapeutic areas, including immunology, oncology and metabolic diseases.