Help Fund the Cure for SCN2A
Every donation fuels critical research moving us closer to the first ever gene therapy cure for SCN2A-Related Disorders.
SCN2A is a gene critical to brain communication and development. Mutations in this gene can have life-altering neurological effects.
In fact, SCN2A is one of the leading single-gene causes of genetic epilepsy, autism spectrum disorder, and intellectual disability.
That’s why we exist. We’re funding the world’s first gene therapy cure for SCN2A-related disorders, to address the condition at its genetic source.
Together, We’ve Raised:
towards a gene therapy cure for SCN2A
About Us: The Story Behind Cure SCN2A
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.
What is SCN2A?
SCN2A is a gene essential for normal brain signalling and development. Mutations in this gene can significantly alter how a child’s brain develops and functions.
SCN2A-related disorders are associated with:
Seizures
Developmental delay
Intellectual disability
Autism spectrum disorder
Movement disorders
Cortical visual impairment
Dysautonomia
Our Path to a Cure
We’ve teamed up with one of the world’s leading gene therapy experts to develop the first gene therapy specifically designed for SCN2A.
This program targets the condition at its genetic source, using a proven delivery strategy with the potential to restore brain communication and reduce neurological complications.
Our goal: first-in-human dosing by 2030.
Donate Today.
All funds raised support our active gene therapy project and the work required to develop the first ever gene therapy treatment for SCN2A.
Every contribution counts.
Looking to make a larger donation?
Click here for details.
"We don’t all get to change the world, but we all get the chance to change someone’s world.”
Subscribe to follow our mission
Stay up to date on upcoming events, updates on the gene therapy project and our family’s journey as we work towards the first ever gene therapy cure for SCN2A.