OUR MISSION

Our mission is to enable the development of the first effective treatment for GAND and to improve the quality of life for children and families living with this diagnosis.

This project is deeply personal. It is built around real children, real families, and real stories.

It represents a collaboration between science and determination — between experts and families who are not willing to accept that rarity is a reason to stop searching for solutions.

Many rare diseases remain without treatment because research is complex, time-consuming, and financially risky. This is why the support of the community, donors, and partners is essential, not only financially, but as a statement that every child matters.

WHAT IS GENE THERAPY?

Gene therapy is one of the most advanced areas of modern medicine. Instead of focusing only on symptoms, it aims to address the underlying cause of disease — the genetic code itself.

In rare genetic conditions like GAND, gene therapy represents one of the most promising paths toward a long-term solution.

HOW DOES IT WORK?

Our bodies function based on instructions encoded in our genes. When a gene does not work properly, it can disrupt development and normal biological processes. Gene therapy aims to correct or bypass this problem, most commonly by delivering a functional copy of the gene into the cells or restoring its activity.

This is typically done using specially engineered viral vectors, which act as a delivery system. These vectors are modified so they cannot cause disease, but can safely carry genetic information to the target cells.

Gene therapy for GAND syndrome is a one-time treatment that delivers a healthy copy of the gene to the cells with viral vectors. The majority of patients with a GAND syndrome have one mutated gene that causes loss-of-function of the protein called beta-catenin. Gene replacement therapy is a type of therapy where a functional copy of GATAD2B is delivered to a patient’s cells through adeno-associated virus (AAV) delivery.

WHAT THIS MEANS FOR GAND?

For GAND, the goal of gene therapy is to restore or replace the function of the GATAD2B gene that is not working correctly.

If successful, this approach could slow or stop disease progression, improve developmental outcomes, and significantly enhance quality of life.

While the development process is complex and requires time, research, and resources, advances in gene therapy already show that this approach is scientifically realistic.

WHY THIS APPROACH MATTERS?

In recent years, gene therapy has already led to breakthroughs in other rare diseases. This demonstrates that meaningful progress is possible, even in conditions once considered untreatable, like for patients with Spinal Muscular Atrophy (SMA) or Juvenile Parkinsonism (AADC defficiency).

For families affected by GAND, gene therapy represents more than science — it represents hope. The possibility that the future will not be defined only by limitations, but also by progress, collaboration, and action.

HOW YOU CAN HELP?

Consider donating to this cause as with GAND patients every day matters. Every day that passes, is a day these children will never get back. Every donation matters, every dollars counts.

Join us in this fight for a better future for children with GAND syndrome.

Fundraising for GAND gene therapy program

Team Members