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S77-PMO
- Development stage
- Preclinical
- Lead developer
- The Chinese University of Hong Kong
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
S77-PMO is a muscle-targeted therapeutic chimera consisting of the muscle-specific aptamer S77 hybridized with a complementary strand extended with a phosphorodiamidate morpholino oligomer (PMO) [596]. Developed by researchers at the Chinese University of Hong Kong, S77-PMO is designed to improve the delivery of PMO to skeletal and cardiac muscles for the treatment of Duchenne muscular dystrophy (DMD) [596]. The S77 aptamer, identified via cell-SELEX against C2C12 myoblasts, acts as a muscle-targeting ligand that facilitates internalization and accumulation in muscle tissues [596]. In preclinical mdx mouse models of DMD, S77-PMO demonstrated significantly higher accumulation in heart and skeletal muscles compared to naked PMO, offering a promising strategy to enhance dystrophin restoration and therapeutic efficacy while reducing off-target side effects [596].
- Other names
- S77-PMO chimeraS-77-PMO chimeraS 77-PMO chimera
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