A Mission Born From Love. Fueled By Science.
Violehealth.org funds gene therapy research for children with KCNQ2 loss-of-function mutations — specifically targeting the PIP₂ binding pathway at the R325 locus — with the goal of clinical translation of functional KCNQ2 supplementation therapy within a generation.
Fund
Investigator-initiated research grants targeting the science that does not yet exist for these variants.
Connect
Bring together families, clinicians, and researchers working on KCNQ2 loss-of-function disorders.
Accelerate
Bridge lab findings toward clinical trials — turning mechanism into therapy.
Why Now
The tools have caught up to the need. Advances in AAV delivery, patient-derived iPSC neuron models, and gene-editing precision mean that loss-of-function channelopathies like KCNQ2 are, for the first time, realistic therapeutic targets. The opportunity — and the urgency — has never been greater.
The Gap We Fill
The R325M variant has never been formally published. No functional characterization exists. No animal model. No targeted therapy. Violehealth exists to close that gap.
Working Together
We acknowledge the vital work of the KCNQ2 Cure Alliance and the broader research community. We collaborate, we don't compete. Our specific focus is gene therapy for loss-of-function variants at the PIP₂ binding site.
Leadership & Governance
Our foundation is governed by philanthropic stewards and advised by leading scientists at the forefront of genetic medicine.
Board of Directors
Founder & President
Philanthropic Director
Violehealth.org
Finance & Governance Trustee
Treasury & Audit Chair
Global Health Philanthropy Partners
Legal Counsel & Trustee
Secretary & Compliance
Life Sciences Law Advisory
Scientific & Medical Advisory Board
SAB Advisory Position
Lead Gene Therapy Advisor
Harvard Medical School / BCH
SAB Advisory Position
Neurophysiology Specialist
Children's Hospital of Philadelphia
SAB Advisory Position
Clinical Genetics Chair
Stanford Medicine / Lucile Packard