Targeted delivery
Our therapy uses a special carrier to deliver a healthy CDKL5 gene directly to brain cells, reaching key areas like the cortex and hippocampus.
About our CDKL5 AAV9 program
We are a coalition of families, clinicians, and drug developers partnering to deliver a one-time gene therapy that addresses the fundamental deficit driving CDKL5 Deficiency Disorder (CDD). By transporting a functional CDKL5 gene directly into neurons, we aim to restore protein expression, stabilize circuitry, and reclaim developmental potential.
We aren't waiting for a cure for CDKL5, we are making it happen.
How the therapy works
Restoring Hope
Once inside, the gene helps produce a vital protein that supports brain function, reduces seizures, and boosts development for kids with CDKL5 Deficiency Disorder.
Long-Lasting Effect
This treatment is designed to work steadily over time, offering lasting benefits without altering a child's natural DNA.