Cure SCN2A was founded to do one thing: Fund the world’s first gene therapy cure for SCN2A.
Who We Are
Cure SCN2A began after our son Jack was diagnosed with a rare SCN2A mutation. When we were told there was no treatment for his condition, we had 2 choices:
Do nothing.
Or fight for a cure.
We chose to fight.
We are Melissa & Scott Tindal, or better known as Jack’s parents.
From the very beginning, Jack’s development seemed to come a lot harder than others. He struggled to feed, some milestones came late, but most never came at all. We watched some skills disappear and progress stall. The gap between Jack and other children widened more and more each day. After years of uncertainty, a Failure to Thrive, Global Developmental Delay and Autism Spectrum Disorder diagnosis later, we still couldn’t find an explanation behind all of his challenges.
Shortly before Jack’s 3rd birthday, genetic testing finally gave us one: SCN2A - a rare gene mutation affecting brain development. While it explained everything we were experiencing, it came with words no parent ever wants to hear: “There is no cure.”
Jack’s diagnosis changed everything. We’d lost count of how many times someone had told us of someone they knew that didn’t talk until they were 4. Or suddenly said a sentence out of nowhere. Or was simply a “late bloomer”. Instead, we had to face the reality that for us it would likely be different. We would likely never hear our son’s voice. That he would likely require lifelong care. That he would likely face a number of complex medical issues throughout his childhood. And most devastating of all, that his condition may even be life limiting.
In the past 12 months since receiving Jack’s SCN2A diagnosis, his challenges have continued. Jack experienced his first seizures, he was diagnosed with a movement disorder, and at 4 years of age is he still non-verbal with the developmental ability of a 9-12 month old. We were even advised that we should just focus on trying to keep him happy, advice that hit us surprisingly hard. That perhaps, progress may never come.
Unfortunately, children like Jack are often dismissed as “too rare to care”. Their conditions affect too few people to attract meaningful investment, leaving families without answers and children without options. The burden often falls on families like ours to raise the funds required to develop a treatment for their child. But rarity should never determine whether a child is worthy of research, treatment, or hope.
The reality is, this is just our story. There’s millions of other children fighting a similar battle and facing a similar reality. In fact, it is estimated that “rare” diseases collectively affect as many as 400 million people worldwide. But for some, like Jack, treatments can be developed.
We have decided if there is currently no path forward, we would build one. Not only for Jack, but for the thousands of other children living with this condition.
The science is ready. Breakthroughs are already happening for many other genetic conditons. We are raising funds to accelerate a gene-therapy cure for SCN2A. Not someday, but urgently, while it can still change children’s futures.
And with your help, we know we can do this.
About the Charity
Our Vision.
A future where SCN2A no longer defines a child’s life. Where it’s treatable. Manageable. Curable.
Our Mission.
Raise the funds required to develop a cure for LOF SCN2A through a bespoke gene-therapy project.
Our Path to a Cure.
A project has been initiated in partnership with the Gene2Cure Foundation, utilising a dual AAV gene-therapy approach designed to deliver a functional copy of the SCN2A gene to affected brain cells, targeting the root cause of the disorder. The project is estimated to take 4 years, and cost $5-10 million.
Read more about the project here.
Cure SCN2A is a registered not-for-profit organisation governed by a volunteer Board. Funds raised are applied in line with our charitable purpose, supporting gene therapy research and the operational work required to deliver it. We are committed to transparency, accountability, and ensuring every donation is used responsibly.
Transparency & Governance
Watch our Story
Get to know Jack and our SCN2A journey.
Donate to the Project
All funds raised support our active gene therapy project and the work required to develop the first ever gene therapy treatment for SCN2A.
Not someday - but urgently.
Every contribution counts