Help Us Bring Hope to Families Affected by NF2-Related Schwannomatosis

Giving Tuesday Goal: $50,000 to Kickstart the Next Step Toward a Cure

NF2-related Schwannomatosis (NF2-SWN) is a rare disease affecting 1 in 28,000 people. Individuals with NF2-SWN develop multiple tumors along nerves in the brain, spinal cord, and other parts of the body. These tumors, known as schwannomas, meningiomas, and ependymomas, cause serious complications such as hearing loss, balance difficulties, paralysis, vision impairment, and mental health struggles. NF2-SWN has a substantial negative impact on quality of life and dramatically reduces life expectancy.

There is no cure. Current treatments aim to temporarily shrink or remove tumors, but they carry significant risks and fail to provide lasting solutions. Families affected by NF2-SWN urgently need safer, more effective therapies that address the underlying cause of the disease. Gene therapy is the only approach that directly targets the underlying genetic cause of NF2-SWN.

A Promising Path Forward: AAV9-NF2 Gene Addition Therapy

Since 2019, NF2 BioSolutions has partnered with researchers at Nationwide Children’s Hospital in Ohio to develop an innovative gene therapy known as AAV9-NF2 Gene Addition Therapy.

This therapy uses a harmless, engineered viral vector (AAV9) to deliver a healthy copy of the NF2 gene, enabling production of the essential protein Merlin. In NF2-SWN, mutations in the NF2 gene prevent the body from producing enough Merlin, a protein that regulates cell growth, leading to tumor development.

NF2 BioSolutions' approach is unique because it aims to restore Merlin production to healthy levels in all NF2-affected cell types. By doing so, it could help prevent or slow tumor growth throughout the nervous system.

Where We Are Now and What Comes Next?

NF2 BioSolutions has successfully designed and validated the therapy in the laboratory at Nationwide Children’s Hospital. The next critical step is producing the viral vectors required for toxicology and safety studies. This is essential work that must be completed before human clinical trials can begin.

Manufacturing the AAV9 vectors is estimated at $2.2 million. Our immediate goal is to raise $50,000 by January 2026 to launch production. Once this first phase begins, we’ll continue fundraising throughout next year to secure the remaining funds needed to bring this therapy to the clinic.

Why Your Support Matters

This Giving Tuesday, your donation will directly fund the creation of the first batch of AAV9-NF2 vectors. This will bring us one crucial step closer to human trials.

Every donation, big or small, helps turn the hope of a cure into a reality for families living with NF2-related Schwannomatosis.

Your support can transform scientific promise into life-changing progress.

Please consider joining us this Giving Tuesday.