The Future of Gene Therapy Starts Here – Fund the Final Step
Donation protected
Gene therapy has the potential to revolutionise medicine, offering the possibility of real treatments for conditions that were once considered untreatable. For children born with genetic disorders, this technology represents more than just hope—it offers the potential to change lives, giving them opportunities for greater independence, mobility, and communication. Scientific breakthroughs have brought gene therapy to the brink of transforming paediatric medicine, but funding remains a critical barrier. Right now, £300,000 stands between a life-changing gene therapy and the children who need it. Without this funding, a treatment that could make a profound difference will remain out of reach, delaying progress not just for one condition, but for the future of gene therapy as a whole.
Our daughter was born with CTNNB1 syndrome. When she was small, particularly in those early months, we believed she was just taking her time, that every baby develops at their own pace. But as the weeks turned into months, we began to notice the small things—milestones that should have come but did not. Her movements were slower, her muscles softer, and the words we longed to hear never arrived. At first, we reassured ourselves. Maybe she was just on her own schedule. Maybe everything would even out. But deep down, an uneasy feeling grew. We watched other children pull themselves up, take their first steps, say their first words, while our daughter remained in a world that seemed to move differently for her. The gradual realisation that something was wrong was not a single moment, but a slow, creeping weight—hope and worry twisting together until the day we heard the words that changed everything: a rare genetic condition.
No parent ever imagines this path. No one prepares for the countless therapies, the medical appointments, the sleepless nights spent searching for answers. And yet, here we are—standing at the edge of a breakthrough that could rewrite her future. A gene therapy exists that could treat the very cause of her condition, offering possibilities we never thought we would have. But like so many medical breakthroughs, it is just out of reach—not because it does not work, but because the funding to take it to clinical trial has not yet been secured
Children with CTNNB1 Syndrome are bright, determined, and full of personality. They find ways to connect, communicate, and experience joy in their own unique ways. But every day, they face challenges that most people never have to think about. A simple movement—lifting a cup, taking a step—can require immense effort. Speech may not come at all, leaving children unable to express their needs, thoughts, or feelings in the ways they want to. Many cannot walk, struggle with coordination, or experience seizures and serious medical complications. Therapies help, but they can only do so much. The underlying cause—a single genetic mutation—remains unchanged.
But for the first time, there is a treatment that could change this.
The First Gene Therapy for CTNNB1 Syndrome Is Ready
A gene therapy has already been developed and manufactured. It is one of the highest-quality products ever produced by its manufacturing partner, and it has been recognised by both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) as a promising therapy.
This could be the breakthrough that children with CTNNB1 Syndrome desperately need. A treatment that does more than manage symptoms—one that could target the root cause.
The only barrier left? Funding.
£300,000 must be raised to move the treatment into clinical trials. Until that happens, the therapy cannot be used, and families remain in a painful waiting game—knowing that something exists but not being able to access it.
A Step Towards Transforming Medicine for Other Genetic Conditions
CTNNB1 Syndrome is just one of thousands of genetic conditions caused by a single-gene mutation. Many, like Angelman Syndrome and Rett Syndrome, share similar challenges: severe movement and communication difficulties, high support needs, and no cure.
Gene therapy offers a real pathway to change. The success of this trial could set the stage for other conditions to follow, opening doors for more research, faster approvals, and a future where genetic disorders can be treated—not just managed.
This is bigger than one condition. It is about proving that gene therapy can and should be an option for children who would otherwise be left with no medical solutions.
Time Is Running Out. The Trial Cannot Move Forward Without This Funding.
A treatment is ready. The research has been done. The approval process is within reach. But none of that matters without funding.
Every delay means more time lost—time when children could be receiving life-changing treatment.
Donations, no matter the amount, bring this one step closer to reality.
Share this, donate, and help make sure that this therapy does not sit on a shelf when it could be changing lives.
Because when a breakthrough like this is possible, it should not be out of reach.
ALL DONATIONS WILL GO DIRECTLY TO THE CTNNB1 FOUNDATION
CTNNB1 Foundation, The Gene Therapy Research Institute
Address: Dalmatinova ulica 5, Ljubljana
Registration Number: 4125878000
Organizer
Amy Monerawela
Organizer