UltRare

Research Focus

Ultra-rare genetic conditions often affect only a handful of patients worldwide, leaving them without viable treatment options under traditional drug development models. Conventional approaches can take years and require large-scale trials, which are impractical for patients with ultra-rare conditions. The rapid development of individualised antisense oligonucleotide (ASO) therapies offers a transformative solution, allowing the delivery of precision treatments tailored to a patient’s unique mutation in a fraction of the time.

This approach is critical as many ultra-rare disorders are progressive and life-limiting, meaning every day without intervention can lead to irreversible decline. By accelerating therapy development, we not only provide hope but also set a new standard for equitable access to cutting-edge medicine, ensuring that rarity is no longer a barrier to care.

Led by Dr. May Aung-Htut and Professor Steve Wilton, global leaders in ASO research and therapeutic development, our group combines scientific innovation with compassion to bring hope to families who previously had no options. We aim to create a rapid pathway for individualised ASO treatments, ensuring that no child is left behind. Through collaboration with clinicians, researchers, and families, we are building a sustainable model for delivering life-changing therapies.

Key Objectives

• Advance Precision Medicine
• Accelerate Therapy Development
• Ensure Scientific Rigor and Safety
• Empower Families and Communities
• Develop strategies for long-term sustainability

Key Projects

• Rapid ASO Development for Rare and Ultra-Rare Diseases
• Comprehensive Toxicity Evaluation platform for Antisense Oligonucleotides

Community Conversations and Nationwide Collaborations

We value the perspectives of consumers and individuals with lived experience in shaping our research direction. To strengthen this commitment, we partnered with the WAHTN Consumer and Community Involvement Program (CCIProgram) and recruited members of the West Australian community with lived experience of disease and family health history for community conversations in 2022 and 2025. Community Conversation Summary reports can be found here.

Beyond community engagement, we have established collaborations with leading research institutes, universities, and hospitals across Australia to provide access to our Rapid ASO development programs for patients with rare and ultra-rare conditions.

Opportunities

If you are interested in pursuing an Honours or PhD, please explore our list of available projects below.

View Projects

If you are a clinician in seeking molecular diagnosis and development of suitable antisense oligomer drugs for amenable conditions, complete the form below. We’ll review your submission and be in contact within a week.

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