# Oligonucleotide therapeutics

Source: https://onco.cc/technologies/antisense-sirna/  
OnCo record `antisense-sirna` (Technology). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Oligonucleotide therapeutics are short synthetic strands of genetic code that silence a specific cancer gene.

## Summary

Oligonucleotide therapeutics are short synthetic nucleic acid strands that base-pair to a target mRNA and induce RNase H cleavage (antisense) or RISC-mediated degradation (siRNA), silencing a gene at the transcript level. In principle any gene is targetable, including undruggable transcription factors. Antisense and siRNA drugs are established outside oncology; in cancer, delivery to tumours is the barrier, since existing chemistries concentrate in the liver. Antibody-oligonucleotide conjugates and lipid nanoparticle delivery are in early trials, with targets including KRAS, STAT3, and MYC. Whether enough drug can reach solid tumours to silence a driver is the open question. The simple version is a synthetic strand of genetic code that switches off one cancer gene, if it can be delivered.

## Fields

- Kind: Technology
- Status: phase-2
- Last checked: 2026-09-04
- Tags: frontier
- Principle: Base-pairing to mRNA induces RNase H cleavage or RISC-mediated degradation.
- Strengths: Any gene is in principle targetable
- Limitations: Delivery beyond liver

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Antisense_therapy
- Wikipedia: https://en.wikipedia.org/wiki/Antisense_therapy

## Connected records

- fronts: [Targeted Therapy](https://onco.cc/fronts/targeted-therapy/)
- companies: [Achieve Life Sciences (formerly OncoGenex)](https://onco.cc/companies/achieve-life-sciences/), [Ionis Pharmaceuticals](https://onco.cc/companies/ionis/), [Isarna Therapeutics](https://onco.cc/companies/isarna-therapeutics/), [Stitchpoint Bio](https://onco.cc/companies/stitchpoint-bio/)
- terms: [Prostate cancer programmes that failed, and what each failure taught](https://onco.cc/terms/prostate-failed-programmes/), [RNA](https://onco.cc/terms/rna/)
- drugs: [Custirsen](https://onco.cc/drugs/custirsen/), [Imetelstat](https://onco.cc/drugs/imetelstat/)
- trials: [AFFINITY](https://onco.cc/trials/affinity-custirsen/), [IMerge](https://onco.cc/trials/imerge/), [IMpactMF](https://onco.cc/trials/impactmf/), [Study to Evaluate Imetelstat in Patients With High-Risk MDS or AML Failing HMA-based Therapy](https://onco.cc/trials/nct05583552/), [SYNERGY](https://onco.cc/trials/synergy-custirsen/)
- technologies: [Antibody-oligonucleotide conjugates](https://onco.cc/technologies/antibody-oligonucleotide-conjugates/), [DNA origami nanorobots](https://onco.cc/technologies/dna-origami-nanorobots/), [Engineered exosomes as drug carriers](https://onco.cc/technologies/exosome-therapeutics/), [Programmable DNA-targeting therapeutics](https://onco.cc/technologies/programmable-dna-targeting-therapeutics/)
- people: [Adrian R. Krainer](https://onco.cc/people/adrian-krainer/)
- bottlenecks: [The undruggable drivers](https://onco.cc/bottlenecks/b-undruggable-targets/)
- ideas: [A standing rulebook for one-patient treatments](https://onco.cc/ideas/idea-bio2-n-of-1-framework/), [Small molecules that cut the RNA message of an undruggable oncogene](https://onco.cc/ideas/idea-bio1-rna-targeting-small-molecules/)
- pathways: [RNA splicing](https://onco.cc/pathways/rna-splicing/)
- roadmaps: [Drug discovery roadmap: screening in mice → maps of dependency → designing in silico](https://onco.cc/roadmaps/drug-discovery-roadmap/), [Targeted therapy roadmap: imatinib → designed for resistance → the undruggable drivers fall](https://onco.cc/roadmaps/targeted-therapy-roadmap/)

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