CureCell Award winner Matteo Pitteri – developing precision genetic therapies and a ‘Trojan horse’ delivery mechanism to treat motor neuron disease
In July, 65 PhD students submitted single-page synopses on their research into cell or biological therapies. In this series of posts we talk with the seven award winners about their ground-breaking science, their lives as a researcher and how they’ll spend their $10,000 prize…
Award winner: Matteo Pitteri
Institution: Florey Institute
Award: AusHealth CureCell Award, 2025
Project: Unlocking Brain-Penetrating Antisense Therapies for Neurological Diseases
Congratulations on winning the AusHealth CureCell Award! How did it feel when you found out?
It felt surreal! I’m grateful for the recognition of my work and to my supervisors. And the cash prize is definitely appreciated. Throughout all of my academic studies I have, as they say, lived off the smell of an oily rag!
What was the application process like?
It was relatively straightforward and the interview was surprisingly quite nice. I was nervous at first, but it felt like a celebration of the science – an opportunity to discuss the impact of my work, rather than just focusing on technical details.
How would you explain your research to a lay audience?
My research is focused on developing precision genetic therapies to target a gene that is one of the most common causes of motor neuron disease (MND). It’s also about how we can best deliver this therapy. The blood-brain barrier makes it difficult for therapies to get across into the brain. So we’re developing a peptide delivery vector, in which we attach a small protein to our gene therapy that is designed to hijack the blood-brain barrier and trick it into thinking it’s absorbing something regular. This is a ‘Trojan horse’ mechanism to smuggle the therapy into the brain, instead of patients needing invasive spinal injections.
Did you always want to pursue this kind of research?
Not really. I’ve never had such a strong conviction about any particular career path, and ended up here mostly by following my curiosity in science. I was on the fence about chasing a career in finance like my dad, but he’s always encouraged me to focus on something that helps people. That led me to studying biomedicine, and along the way, I fell in love with research. I didn’t plan on getting into precision genetic therapies, but I’m glad I ended up here.
It sounds like you’ll continue in this field after your PhD?
Yes, we’ve made great progress in the last four years and I feel like we’re just getting started. My supervisors are happy with my work, and I’m keen to see where this can go.
Has commercialisation been part of your research from the beginning?
Yes, it’s always been a goal. We knew what the target was: working on improving the delivery of precision genetic therapies. There’s already an FDA-approved therapy for the gene we’re targeting, but it requires invasive spinal injections. Our goal is to improve delivery using this peptide shuttle technology, enabling a less invasive, systemic delivery to the brain, which could lead to better health outcomes for MND patients. The challenge is to find the best solution, make it as safe and effective as possible, then get it into the clinic.
Is there a timeline for clinical trials?
We’ve shown that our therapy works in mice: it can be delivered systemically and gets into the brain. Now, we need to tweak it a bit more. Ideally, we’d like to see this in clinical trials in the next five years or so, possibly starting with primates.
What have you learned doing a PhD in science?
Resilience is key. PhD research is filled with ups and downs – plenty of failures, a lot of one step forward, two steps back – but also periods of unbelievable highs. I’ve learned to manage expectations, try to remain grounded, and not let the highs or lows overwhelm me. It’s about consistency and staying focused on the bigger picture.
Applications for the next AusHealth CureCell Awards will begin in June 2026.
For more information, visit CureCell.org