Every day, children are born with rare genetic disorders for which no treatment exists.
Not because solutions are impossible, but because these diseases are often too rare to attract attention, funding, or coordinated development.

For families, this means waiting.
Waiting without answers. Waiting without options.

The Gene2Cure Translational Gene Therapy Summit 2026 in Barcelona

During the Gene2Cure Translational Gene Therapy Summit 2026, our focus will be on our two programs: PUS3 syndrome and GAND syndrome. Researchers working on these two diseases will talk about the biology of the disease, but also about the development of gene therapy.

Today, gene therapy has the potential to treat diseases that were once considered untreatable.
But for ultra-rare conditions, development is still slow, fragmented, and often dependent on individual efforts.

You are welcome to join the conference or supporting it with a donation. Your support will help us:

organize and accelerate real therapy programs
build collaborations that reduce duplication and delays
and move promising treatments closer to clinical trials

Because for children with rare diseases, time is critical. And we believe progress happens faster when people work together. Sharing knowledge, being transparent about challenges and focusing on what matters most - getting treatments to patients.

If you believe that no disease is too rare to matter,
we invite you to be part of this work.

Gene2Cure Foundation Translational Gene Therapy Conference 2026 (PUS3 and GAND syndrome)

Advancing treatments for children with rare genetic disorders