Wings of the First

Fund the First Treatment for CLN3 Batten Disease—And Help Hope Take Flight

At the Vanguard Clinical Rare Disease Foundation, we’re not just developing therapies—we’re reengineering the system that failed to deliver them fast enough. We believe the process is the product. And right now, we’re building one of the most urgent and hopeful clinical trials in rare disease: VCRDF-CLN3, a first-in-child precision therapy for CLN3 Batten disease.

It begins with two brothers—ages 5 and 11—facing a fatal diagnosis with courage and grit.

They are the first. But they cannot be the last.

✨ What We're Launching

In a collaborative effort including scientists and physicians at the University of Michigan and the University of North Carolina, Chapel Hill, VCRDF is advancing an antisense oligonucleotide (ASO) therapy that targets the most common CLN3 Batten mutation: a ~1-kilobase deletion.

CLN3 Batten disease is a rare, fatal genetic disorder that primarily affects children, causing progressive loss of vision, motor skills, speech, and cognitive abilities. As the disease advances, it leads to seizures, mobility challenges, and premature death, often in the teen years. There is currently no cure, and treatment options are extremely limited. We are here to change that, starting with the development of this investigational therapy designed to give children with CLN3 a fighting chance.

This is not a research concept. It’s an opportunity—ready for clinical action.

Our work builds directly on Zebronkysen, the first-ever individualized ASO developed for CLN3 Batten, which showed early promise. Zebronkysen was developed for specifically for twin girls who are the only ones known to have their specific mutation. That program gave one family hope—and helped pave the roadmap for what’s possible now: broader application, faster execution, and a model built to scale.

We’ve already received FDA feedback confirming alignment with our plan to treat these two children under the Agency’s N-of-1 Guidance.

The path is cleared. We have the road map in hand. We have the dream team assembled and ready to go.

All that’s missing is the funding to launch.

🧬 Why It Matters

This is not a study for the future. It’s a treatment for today.

Your donation will:

💊 Manufacture the ASO drug specifically designed for CLN3 Batten Disease
📋 Support regulatory safety studies required to initiate dosing
🏥 Fund treatment for the first two children—two brothers, one chance
🔬 Enable expansion of the IND to allow more CLN3 children to access this therapy

Each dollar fuels real, immediate action—and builds a future beyond the first two.

💙 Driven by Urgency. Powered by Love.

Our team includes leading scientists, clinical experts, and rare disease parents—people who know what’s at stake and move with urgency because they must. As the parents of the boys receiving the first doses said:

“When we learned there might be a chance to help our boys—not years from now, but soon—we felt hope again.”

🕊️ What’s Next

The science is ready.
The children are waiting.
Now we need you.

This trial starts with two—but it’s built to lift many more.

Join us. Fund the first treatment for CLN3 Batten disease.
And help hope take flight.

Our 501(c)(3) Status
Vanguard Clinical Rare Disease Foundation (VCRDF) has filed for 501(c)(3) tax-exempt status with the IRS and is currently awaiting approval. We anticipate receiving confirmation soon, but the need for action is too urgent to wait.

Every day matters for the children and families we serve. Our mission is to accelerate treatment options for ultra-rare pediatric diseases—conditions that are often overlooked and underfunded. By supporting us now, you are helping bring life-changing therapies closer to the patients who need them most.

Your contribution today enables us to continue this critical work, and once our tax-exempt status is officially granted, it will be retroactively applied to all qualifying donations made during this pending period.

Thank you for standing with us.

Hope Takes Flight

By Vanguard Clinical Rare Disease Foundation

Hope Takes Flight

Fund the first treatment for CLN3 Batten Disease—and open the door for more.

Team Members