Mirugen: gene therapy to 'switch on sight’
Imagine waking up one day to find your world slowly fading to darkness. For millions of people worldwide suffering from retinal degeneration, this nightmare is a reality. Conditions like retinitis pigmentosa, age-related macular degeneration, and Stargardt disease affect over 190 million people globally, robbing them of their sight and independence. But what if there was a way to not just halt this progression, but to reverse it? Enter Mirugen, a biotech startup on a mission to turn this hope into reality.
The challenge of retinal degeneration
Retinal degeneration is when the light sensing cells at the back of our eyes, called photoreceptors, begin to die off and cause vision loss. Until now there have been no effective treatments or cures for most patients facing this condition.
Mirugen’s approach
A/Prof Raymond Wong, founder of Mirugen, explains their solution: “Our gene therapy is designed to treat blindness by harnessing the regenerative power of the retina’s own stem cells. We inject a virus carrying reprogramming genes into the eye. These genes stimulate the stem cells to develop into new photoreceptors, replacing the ones that have been lost.”
This approach is unique to stimulate the eye’s regenerative potential. The Mirugen team are not just slowing down these diseases; they’re working to restore sight, offering hope to patients who currently have none.
At the helm of Mirugen is A/Prof Raymond Wong, a stem cell biologist with over 20 years of experience. After earning his PhD in Melbourne and completing a postdoctoral fellowship in the United States, Raymond returned to Melbourne to focus on developing therapies for retinal degeneration at the Centre for Eye Research Australia (CERA).
Raymond’s journey from academia to entrepreneurship has been driven by the goal of translating his scientific discoveries into real-world treatments that can change lives.
Milestones and achievements
Mirugen has achieved significant milestones in both scientific and business development. Their gene therapy has shown promising results in animal models, a crucial step towards human clinical trials.
On the funding front, the discovery phase of the research program has received support from prestigious organisations, including the National Health and Medical Research Council (NHMRC), Medical Research Future Fund (MRFF), National Stem Cell Centre of Australia, and Retina Australia. “We’ve gained a lot of traction in recent years,” Raymond notes. “We cannot wait to advance the program to the next stage for the product development.”
Next steps for Mirugen
Mirugen is now focused on advancing its gene therapy through preclinical testing and preparing for human trials. The team is also looking to secure the funding needed to accelerate development and bring their therapy to market.
“We’re finalising investment proposals that will allow us to take our next steps,” Raymond shares. “Our goal is to make this treatment available to patients as soon as possible.”
The TRAM experience and advice for aspiring entrepreneurs
“TRAM Air has been a fantastic program,” Raymond explains. “It’s well-resourced and a great community of startup colleagues and we have been coached by very experienced mentors.”
For academics considering the leap into entrepreneurship, Raymond has some valuable advice: “If your goal is to help patients, you need to think beyond the scientific discoveries at the lab bench. Translating your research into practical applications through commercialisation is essential. It’s an accelerated pathway to making a real impact.”
Reflecting on his own journey, Raymond emphasised the importance of being open to the challenges and rewards of entrepreneurship.
As Mirugen continues its work in gene therapy for retinal degeneration, the future looks brighter for millions affected by blindness worldwide.
Read more about our 2024 TRAM Air cohort HERE.
If you’re interested in applying for TRAM Air in 2025, please fill out our expression of interest form or email Masha Pelipas, TRAM Air Program Manager: maria.pelipas@unimelb.edu.au.
Connect with Mirugen
Founder: Raymond Wong