Accel-ASO brings together the expertise, infrastructure and pathways needed to develop antisense oligonucleotide therapies for rare and genetic diseases.
Established in 2026 through the Medical Research Future Fund’s National Critical Research Infrastructure Initiative, we work with researchers, clinicians and industry partners to move promising therapies from genetic diagnosis and target identification towards preclinical evaluation and clinical translation.
Our services
We provide specialist expertise across the antisense oligonucleotide development pathway, from identifying and assessing a therapeutic target to planning preclinical studies. Our team supports personalised “n-of-1” therapies as well as treatments that could benefit broader patient populations.
Variant and target assessment
Determine whether a genetic variant may be suitable for antisense therapeutic development and identify potential therapeutic targets.
ASO design and optimisation
Select an appropriate ASO mechanism, then design and optimise candidate oligonucleotides.
Cellular evaluation
Evaluate candidate ASOs using patient-derived fibroblasts, induced pluripotent stem cell models and other disease-relevant cellular systems.
Efficacy assessment
Assess therapeutic efficacy using molecular, transcriptomic and proteomic methods.
Safety assessment
Investigate potential off-target effects and toxicity in disease-relevant cellular systems.
Translational planning
Design preclinical studies and plan the pathway towards clinical translation.
Who we work with
Accel-ASO works with organisations and specialists developing potential therapies for rare and genetic diseases:
- Researchers
- Clinicians
- Industry partners
- Patient advocacy organisations
- Australian and international collaborators.
Discuss a potential project
Contact the Accel-ASO team to discuss a genetic variant, therapeutic target or potential collaboration. We can help determine whether the project aligns with our expertise and identify appropriate next steps.
Email: accel-aso@murdoch.edu.au
Meet the team
Supported by
Our partners
Contact us
If you have any questions, please contact us at accel-aso@murdoch.edu.au.