Giving Tuesday 2023 - Gene Therapy for NF2

Unlocking Hope: Gene Therapy for NF2 – Giving Tuesday Fundraiser

We're rallying together to make a difference and need your support. Every single dollar counts in our mission to find a cure for NF2. 🙌

🌱 The Mission:

We are moving forward with our gene therapy project, and now we're raising funds to move to the next step, which is the toxicology study, a pivotal part of the IND (Investigational New Drug) process preceding Phase 1 of a clinical trial. We believe in progress, and your contributions will make this a reality! 

🔍 The Research:

The groundbreaking Gene Therapy led by Dr. Meyer at Nationwide Children's Hospital focuses on restoring the Merlin protein in the NF2 gene by using an AAV9 viral construct as a delivery mechanism. The efficacy of this construct is currently undergoing testing. Testing using intratumoral injections is also ongoing in the KO (Knockout) mouse and xenograft mouse models. We successfully showed the expression of merlin in tumor cells, accompanied by reduced tumor growth in the xenograft model.

Pre-IND documents explaining our clinical trial plan are being prepared to be submitted to the FDA. We have also applied for a National Institute of Health (NIH) grant to fund the preparation of a future trial.

🌎 Why it Matters:

The toxicology study is the vital piece of the puzzle that helps ensure the safety and efficacy of the therapy before it reaches patients. It's a critical checkpoint on our path toward finding a cure for NF2. With your help, we can take this vital step and change lives.

More information here: https://nf2biosolutions.org/gene-therapy-and-nf2/research-initiatives/understanding-the-crucial-role-of-toxicology-studies-in-nf2-gene-therapy/ 

💡 Why Donate?

Regardless of size, your contribution brings us closer to realizing this vision. Together, we can impact countless lives, bringing hope and healing to those affected by NF2.

🎯 The Goal:

Our target is $400,000, and we're confident we can reach it with your support! Share this fundraiser with your network, and consider donating today. Let's create a brighter future for everyone affected by NF2!

💞 Join Us on Giving Tuesday:

Save the date - November 28, 2023! This Giving Tuesday, let's make a difference together. Please help us get closer to finding a cure for NF2. We're all in this fight together, and your support means the world to us! If you'd like to have your fundraiser that you can share with your friends and family, contact us at rebeca-chajon@nf2biosolutions.org.

🤗 Donate, Share, and Make a Difference!

Every action you take brings us closer to a world without NF2. Join us on Giving Tuesday, share this fundraiser, and donate what you can. Let's profoundly impact the lives of those who need it most.

Thank you for being a part of our journey, and together, we can unlock a brighter, healthier future for all. 💪💙