Phase I/II readiness

Developing groundbreaking CDKL5 AAV9 gene therapy. Help us reach $3.5M for first-in-human trials.

Every dollar advances a therapy designed to restore CDKL5 protein and give children back crucial milestones. We have raised $200K toward a $3.5M launch goal—join the movement.

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Fuel the science

raised of needed to activate IND-enabling studies.

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UPCOMING WEBINAR

Gene Therapy for CDKL5:
The History, Progress & Next Steps

Sunday, October 26, 2025

9:00 - 10:00 AM CDT

The US FDA has provided positive feedback + IND suggestions for gene replacement therapy for CDKL5. Join us to get the latest on definitive work being done and next steps!

Rachel Bailey, PhD Lead Researcher

Jainu Jogani Parent Advocate

Hosted by: American Society of Gene + Cell Therapy & Child's Cure Genetic Research

View Details & Register

Families powering the journey

What is CDKL5 gene therapy?

Our AAV9 vector delivers a working CDKL5 gene directly to neurons—the equivalent of sending a healthy gene passenger aboard a precise molecular ship. The goal: restore CDKL5 protein production and stabilize brain function for children living with CDKL5 Deficiency Disorder (CDD).

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 it is promising

Gene therapy success in rare diseases inspires a CDKL5 breakthrough. Preclinical studies show broad neuron uptake and symptom improvement when the vector is delivered intracerebroventricularly.[1][5]

≥50% transduction across mouse cortex with restored behavior at 3e11–5e11 vg/g brain.[1]
UX055 studies demonstrate motor and cognitive rescue in CDKL5 models and corrected hyperexcitability in brain organoids.[2]
Families worldwide are rallying—Connect CDKL5 now spans 500+ patients in 45+ countries.[3]

Take action now

Pledge your support, start a micro-fundraiser, or host a community event. Together we can unlock the capital required for IND submission and Phase I/II enrollment in Q2 2026.

Ways to Get Involved Join the Updates List

How AAV9 carries hope

Think of AAV9 as a boat engineered to dock at neurons. Once inside, the healthy CDKL5 gene starts expressing protein that supports synaptic development and stabilizes circuits.

Key evidence

[1] Molecular Therapy, 2024: i.c.v AAV9 delivery transduced ≥50% of neurons with improved motor behavior and dendritic structure.
[2] Ultragenyx UX055 pipeline: AAV9-CDKL5 restored motor and cognitive performance in preclinical models; corrected hyperexcitability in organoids.
[3] IFCR, Connect CDKL5: 500+ patients, 45+ countries linked through Centers of Excellence and resource libraries.
[4] CGTLive, 2024: Parent-led coalitions accelerating Child's Cure Genetic Research and pre-IND preparation.
[5] PubMed 39033321: Sustained CDKL5 expression and reduced pathology following vector delivery.