Sarepta’s Leading RNA-Based Platform
Phosphorodiamidate Morpholino Oligomers – PMOs
The linking of sequenced monomers with nucleic acid bases to create oligonucleotides is common to all RNA-based therapeutics. However, researchers can change the chemical character of the therapeutic molecules in subtle but significant ways. For example, the fine structure and charge of the monomers determines the strength of the linkages that hold them to their target. The backbone of the oligonucleotide can be altered to achieve different properties, such as resistance to enzymic degradation or bioavailability. These and other differences in the characteristics of RNA-based therapeutics may alter the safety and efficacy profiles of different drug candidates, even if they are sequenced to target the same RNA.
Sarepta’s advanced RNA platform is based on our pioneering work with phosphorodiamidate morpholino oligomers, or PMOs. PMOs are synthetic structures modeled after the natural nucleic acid framework of RNA, but with critical structural modifications designed to allow for the functional manipulation of the molecules’ drug-like properties. The chemistry-based advantages built into our PMO technology address several pharmaceutical needs not consistently addressed by early generation antisense chemistries including stability, efficacy, specificity, delivery and safety. We describe our PMO compounds as “oligomers” rather than oligonucleotides due to their unique synthetic chemical structure.