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Rare Disease Day 2026 NF2-SWN

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Accelerating a Cure for NF2

Rare Disease Day is a global call to action for conditions that are too often overlooked. We are steadily moving toward clinical trials for NF2, and this year, we need your help to begin manufacturing the clinical-grade AAV9 (Adeno-Associated Virus 9) NF2 gene therapy product.

We are committed to full transparency about how your donations are used: The first step in the AAV manufacturing process is creating the DNA blueprint, called the plasmid. A plasmid is a small circular piece of DNA that acts as the instruction sheet to tell the gene therapy AAV9 what DNA to deliver into cells. In our case, we will deliver a healthy copy of the NF2 into the tumor.

Our Goal

We aim to raise $300,000 by February 28, 2026, to launch this first manufacturing stage. We currently need an additional $30,000. 
Thanks to the incredible generosity of our community, our Giving Tuesday campaign raised $65,000.
Our board members and ambassadors have pledged $110,000, providing strong early momentum.
An additional $5,000 has come from dedicated NF2 families.
Most recently, a $90,000 gift from the Kantor family has brought our total to $270,000.
We need your help to raise the remaining $30,000 to reach our goal.

How You Can Help
Your gift directly supports the completion of plasmid production and allows us to secure a contract and production slot for the clinical-grade viral vector. Once AAV9.NF2 manufacturing is complete, we will advance to a toxicology (safety) study and then to clinical trials.
Every contribution moves this work forward. Whether you give $25, $100, or more, you are helping accelerate progress toward a cure.
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Supporting

NF2 BioSolutions UK

We are a registered UK charity that supports research to bring an end to the pain and suffering of children and adults with Neurofibromatosis Type 2 (NF2). NF2 is very rare with under 2000 cases in the UK, it's a condition that causes tumours to grow in the brain and spine as well as elsewhere in the nervous system. As the tumours grow, they cause permenent and life changing damage. Those affected have the condition from birth, NF2 is degenerative and a ticking time bomb. We are a not for profit, patient led charity with all donations going directly to end NF2. Our board of trustees includes the lead specialist for NF2 in the UK Professor Gareth Evans. We can't do it without you Thank you

£358.75 raised of £30,000.00 target by 8 supporters

£315.00 donated plus £43.75 in GiftAid

When

From the 19th February 2026 until the 28th March 2026

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