Helpful science. For KCNQ2.
Violehealth funds gene therapy research and builds translation pipelines for children with KCNQ2 mutations — starting with one little girl named Violetta.
KCNQ2 Target Locus Mapping
Characterizing PIP₂ binding pocket dynamics to understand R325 locus mutations.
Learn moreAAV9 Vector Delivery Capsids
Building tissue-specific viral vectors optimized for central nervous system neurons.
Learn morePatient-Derived iPSC Assays
Validating therapeutic restoration using patch-clamp electrophysiology.
Learn moreUnified Patient Registry
Connecting KCNQ2 families globally to assemble clinical-trial-ready patient cohorts.
Learn moreMajor Gifts & Wires Portal
Supporting Donor-Advised Funds (DAFs), securities transfers, and direct wires.
Learn moreScience Resource Library
Open-access diagnostic reports, sequence summaries, and patient case files.
Learn moreThe KCNQ2 Gene Therapy Pipeline
Tracking translational milestones from initial locus modeling to clinical trial filings.
PHASE 01
Target Locus Mapping
Structure characterization of PIP₂ binding domain at the KCNQ2 R325 locus.
PROGRESS
100%
PHASE 02
Vector Design (AAV9)
Constructing tissue-specific AAV capsids for target neuronal delivery.
PROGRESS
100%
PHASE 03
In Vitro Validation
IPSC patient-derived neuron transfection and patch-clamp electrophysiology.
PROGRESS
70%
PHASE 04
In Vivo Seizure Model Studies
Validating safety, channel expression density, and phenotype restoration in mice.
PROGRESS
15%
PHASE 05
IND-Enabling Trials
Final toxicology audits and filing Investigational New Drug (IND) documents with FDA.
PROGRESS
0%
Latest updates
Understanding KCNQ2: An Awareness Primer
KCNQ2 is one of the most common causes of neonatal genetic epilepsy. Here is what families and supporters should know.
Read article →Our First Grant Cycle Opens Soon
Violehealth is preparing its first investigator-initiated grant cycle focused on the KCNQ2 PIP2-binding pathway.
Read article →Violehealth Foundation Is Founded for Violetta
A father turns an impossible diagnosis into a mission to fund gene therapy research for children with KCNQ2 mutations.
Read article →