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Modifications of ASO compounds to improve drug stability and delivery have advanced through several generations, and include chemical alteration of the backbone or sugar, conjugation with targeting agents, and others.
Phosphorodiamidate Morpholino Oligomers, or PMOs, are the most recent modified ASOs. Four PMO drugs have been approved by the FDA for treatment of Duchenne muscular dystrophy (DMD). Numerous additional PMO agents are in development throughout the biopharma industry pipeline, for DMD and many other diseases as well.
CELURCA is developing a core technology of novel and unique chemistry to design and optimize a 4th generation of PMOs with modifications that improve upon the current and pipeline drugs. These next-gen PMOs hold great promise to improve drug delivery, enhance specificity and targeting, reduce side effects, and advance production efficiency.
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