VVMF and Mirugen Partner to Advance AAV-Based Gene Therapy Manufacturing for Retinitis Pigmentosa in Australia
Key Insights
Viral Vector Manufacturing Facility (search) (VVMF) and Mirugen (search) have signed a non-exclusive Letter of Intent to evaluate an AAV-based gene therapy candidate targeting Retinitis Pigmentosa (search).
VVMF will conduct an initial pilot study assessing productivity, recovery, and quality attributes of Mirugen (search)'s lead candidate as it moves toward a first-in-human clinical trial.
The collaboration aims to strengthen Australia's onshore advanced therapy manufacturing capabilities and support growth of the cell and gene therapy sector across the Asia-Pacific region.
Sydney-based Viral Vector Manufacturing Facility (search) Pty Ltd (VVMF) and Melbourne-headquartered Mirugen (search) Pty Ltd have signed a non-exclusive Letter of Intent (LoI) to collaborate on evaluating and potentially developing an adeno-associated virus (AAV)-based gene therapy candidate for Retinitis Pigmentosa (search), a move the partners say will bolster Australia's advanced therapy manufacturing capabilities.
Under the agreement, VVMF — described as Australia's only contract development and manufacturing organisation dedicated to viral vector research, design, development and manufacture for advanced therapeutic applications — will conduct an initial pilot study to assess the productivity, recovery and quality attributes of Mirugen (search)'s lead gene therapy candidate. Mirugen, a preclinical biotechnology company developing regenerative therapies for retinal diseases, is advancing the program toward a first-in-human clinical trial.
Retinitis Pigmentosa (search) is an inherited condition that causes progressive degeneration of photoreceptors and consequent vision loss. The condition represents an area of significant unmet medical need, and AAV-based gene therapy approaches aim to deliver functional genetic material directly into affected retinal cells to slow or halt disease progression.
Strengthening Australia's Biomanufacturing Ecosystem
The collaboration reflects a shared commitment to advancing gene therapy innovation while reinforcing Australia's onshore capabilities in the development and manufacture of advanced therapy medicinal products (ATMPs). ATMPs — therapies based on genes, cells or tissue engineering — require specialised manufacturing expertise and infrastructure due to their inherent complexity. Viral vectors serve as a key component in delivering genetic material into cells and are employed in the development of treatments for cancers, rare diseases and other advanced therapeutic applications.
VVMF chief executive officer Stephen Thompson emphasised the strategic alignment of the partnership. "Collaborating with Mirugen (search) provides an opportunity to support the evaluation and development of promising gene therapy candidates," Thompson said. "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."
The partners noted that the collaboration supports the continued growth of the cell and gene therapy sector across the Asia-Pacific region and reinforces Australia's position as an emerging advanced biomanufacturing hub. Mirugen (search)'s lead program could also benefit from Australia's clinical development environment, including research and development tax incentive frameworks.
A Staged, Evidence-Led Approach
Mirugen (search) executive chair Charlotte Casebourne Stock highlighted the pragmatic nature of the manufacturing evaluation. "We are pleased to work with VVMF on this pilot evaluation, as Mirugen advances its lead program towards clinical development," Casebourne Stock said. "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."
The non-exclusive nature of the agreement provides Mirugen (search) with flexibility as it progresses its lead candidate through preclinical development and toward first-in-human studies. The pilot study outcomes will inform subsequent manufacturing strategy decisions for the Retinitis Pigmentosa (search) gene therapy program.