The INAD Foundation Australia and Cure INAD UK are proud to be working together to fundraise for the pioneering gene therapy program at University College London (UCL). Following our recent discussions with Profs. Kurian & Rahim, we are pleased to share the following updates:

Prof Rahim’s mouse model work is advancing and is being prepared for publication.
Dr Soo has prepared a paper on natural history, radiological biomarkers, and disease progression. This is about to be submitted for publication.
Early findings show gene replacements are being entered into machine learning tools to help identify the most reliable markers and form a clinical scale.
Over 300 patients have contributed to the natural history study — providing a strong foundation for future clinical planning.

Meetings are underway with viral vector producers.
Engagement is underway with UK regulators too.
Work is ongoing to refine outcome measures, validate mouse model data,
and approach national grant providers.
The programme is moving towards what we hope will be a first-in-human clinical trial.

Both Cure INAD UK and the INAD Foundation Australia now share a clear goal:
to raise £2 million towards the production of the viral vector required for
the gene therapy treatment.

It is envisaged that any future trial would be sponsored by Great Ormond Street
Hospital (GOSH), a renowned international centre for delivering gene therapy.

This update marks an encouraging step for the INAD community. With continued collaboration, research progress, and support, we hope to move closer to a treatment option for children with INAD worldwide.

We want to raise the £2 million needed to fund the production of the viral vector, and we need to do this quickly. Every donation, fundraiser, or share helps to bring this vision closer to reality.

The INAD Foundation Australia and Cure INAD UK are accepting donations that will go towards this gene therapy programme.

At OHSU, a dedicated team of scientists and clinicians is contributing to vital research into Infantile Neuroaxonal Dystrophy (INAD) as part of their broader focus on NBIA disorders.

OHSU is home to the NBIAcure Research Team, led by Dr Susan Hayflick, a globally recognised expert in NBIA conditions, including INAD. Their work includes:

• Genetic studies to better understand the PLA2G6 mutations responsible for INAD.
• Natural history studies to track progression of INAD to inform clinical trial readiness.
• Biomarker development to identify measurable indicators of progression & treatment response.
• Collaborations with other research institutions to support future gene therapy trials.

Through their deep scientific knowledge and patient-centered approach, OHSU is playing a key role in moving INAD research forward and bringing hope to affected families worldwide.