What is CDKL5 gene therapy?
CDKL5 Deficiency Disorder (CDD) is a rare condition (about 1 in 40,000 children) that can cause developmental delays and seizures when the CDKL5 gene is missing or not working properly.
Our solution: we use a special delivery system called AAV9—like a tiny ship—to bring a healthy CDKL5 gene to a child’s brain cells, helping them grow and stay healthy.
Why it matters: this could give kids with CDD a chance at a better life, bringing hope to thousands of waiting families. Early tests in mice show improvements in movement and thinking, and the approach is designed as a one-time treatment with lasting benefit.
Where we are: we’re in the research phase and planning human trials—your support can help
- Targets the root cause by replacing the missing gene.
- Designed for one-time dosing with durable benefit.
- Builds on proven AAV9 platforms used in SMA and MLD.
Why is it promising
Fixes the Problem at Its Source: This therapy delivers a working CDKL5 gene to brain cells, helping them function properly and tackling the main cause of CDD, not just the symptoms like seizures or delays.
One-Time Treatment: It’s designed to be a single dose using a safe delivery system (AAV9), which could provide long-lasting benefits without needing repeated treatments.
Based on Proven Methods: It uses technology already successful in treating other rare diseases like SMA, making it more likely to be safe and effective.
Shows Great Results in Tests: Early studies in mice show better movement, thinking, and brain health, giving hope that it could help kids with CDD live better lives.
Moving Toward Human Trials: Research is progressing fast, with plans for testing in people soon—support can help make this happen!
Brings Real Hope: This could be a game-changer for kids with CDD, reducing seizures, improving development, and giving families a brighter future.