Drug the undruggable.
RNA gene silencing delivery and selectivity unlock value
Access to Intracellular Biology
Challenge: Many high-value disease drivers are inside cells and can’t be addressed with antibodies or small molecules.
Solution: RNA therapeutics silence gene expression at the source, enabling first-in-class mechanisms.
Targeted Delivery
Challenge: Systemic RNA exposure can limit efficacy and safety.
Solution: GalNAc delivers to hepatocytes for metabolic disease; AOCs (Antibody-Oligo-Conjugates) deliver into defined cells for oncology.
Durable, Measurable, Scalable
Challenge: Pharmaceutical products need clear translation: durable effect, biomarkers, and manufacturability.
Solution: Long-acting knockdown with clean PK/PD readouts and conjugation formats compatible with scale-up and repeat dosing.