Intracellular biologics pipeline

Using our proprietary EEV™ technology, we are creating a pipeline of oligonucleotide therapies with the potential to address a wide range of diseases and conditions. Our lead programs are in neuromuscular diseases, including Duchenne muscular dystrophy (DMD). Our other intracellular programs span multiple therapeutic areas and focus on biologics including antibodies, peptides, enzymes and other proteins.

Oligonucleotides
Neuromuscular
Duchenne Muscular Dystrophy
Preclinical
Duchenne Muscular Dystrophy
Discovery
Other Neuromuscular Diseases
Discovery
Other Therapeutic Areas
Immunology
Discovery
Central Nervous System
Discovery
Antibodies and Peptides
Oncology
Protein Degradation
Discovery
PPI Inhibition
Discovery
Enzymes and Proteins
Inborn Errors of Metabolism
MNGIE
Preclinical