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PMLA-LOEt-AON
- Development stage
- Preclinical
- Lead developer
- Cedars-Sinai
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Biodegradable Polymers → Polymer-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
PMLA-LOEt-AON is a research-stage nanobioconjugate drug delivery system based on a poly(β-L-malic acid) (PMLA) scaffold. It is designed to deliver antisense oligonucleotides (AONs) into tumor cells. The construct incorporates leucine ethyl ester (LOEt) as a pH-independent membrane-destabilizing unit to facilitate endosomal escape and direct transmembrane delivery. The active therapeutic component is typically a Morpholino AON targeting oncogenic proteins such as EGFR, HER2, or tumor vascular markers like laminin-411. Developed primarily at Cedars-Sinai Medical Center, this platform has been investigated for the treatment of glioblastoma and breast cancer. Research indicates that while LOEt enables membrane penetration, its pH-independent nature can lead to non-specific binding compared to pH-dependent alternatives like trileucine (LLL).
- Other names
- PMLA-LOEt-AON nanobioconjugatepoly(beta-L-malic acid)-leucine ethyl ester-antisense oligonucleotidePMLA-LOEt-Morpholino
02
Targets
EGFR T790M (Epidermal growth factor receptor T790M mutant)LAMA4 (Laminin subunit alpha-4)ERBB2 (Erb-b2 receptor tyrosine kinase 2)
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