New partnership to advance gene therapy for retinal disease

Viral Vector Manufacturing Facility (VVMF) and Melbourne-based biotechnology company Mirugen have signed a non-exclusive Letter of Intent to collaborate on a pilot study evaluating the potential development and manufacturing pathway for Mirugen’s adeno-associated virus (AAV)-based gene therapy candidate.

The investigational therapy is being developed to target retinitis pigmentosa, a genetic disorder marked by the progressive degeneration of photoreceptors and the associated loss of vision. Mirugen’s approach seeks to harness the regenerative capacity of the retina’s own stem cells to restore vision, offering a potential new treatment avenue for millions of people living with currently untreatable forms of blindness.

Founded by stem cell biologist A/Professor Raymond Wong, Mirugen is a spinout from the Centre for Eye Research Australia (CERA) and the University of Melbourne. The company’s technology uses cellular reprogramming to regenerate light-sensing photoreceptors lost through disease. Built on Nobel Prize–winning advances in cellular biology, the platform uses gene therapy delivered directly into the eye, with the aim of restoring retinal function without the need for cell transplantation.

Stephen Thompson, CEO of VVMF, said “Collaborating with Mirugen provides an opportunity to support the evaluation and development of promising gene therapy candidates. This Letter of Intent reflects our focus on enabling high-quality process development and manufacturing pathways for emerging therapies, while strengthening Australia’s capability in viral vector manufacturing for the global CGT market.”

To date, Mirugen has secured approximately $7 million in funding, including a $4.5 million seed round backed by Brandon Capital, Tin Alley Ventures and the University of Melbourne Genesis Pre-Seed Fund. This investment builds on earlier non-dilutive funding received through the MRFF CUREator biotech incubator, supporting the company’s progression towards a first-in-human clinical trial.

Charlotte Casebourne Stock, Executive Chair of Mirugen, stated “This study will allow us to assess an emerging Australian manufacturing capability against technical criteria, while supporting the continued growth of the local gene therapy ecosystem. We are taking a staged and evidence-led approach to manufacturing and look forward to evaluating the results of this initial work.”

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