Mission

Our big mission is to find a cure for NKX6-2 by creating a gene therapy, while sharing our work and connecting doctors and families around the world to help patients with similar conditions.

Pioneering Targeted Gene Therapy

Our primary goal is to develop a life-changing gene therapy for NKX6-2 related leukodystrophy. This pioneering research focuses on the precision targeting of oligodendrocyte cells, the specialized components of the central nervous system responsible for the formation and maintenance of the myelin sheath. By repairing this vital protective layer for the nerves, we aim to stop the condition from progressing and help children regain their movement and independence.

Advancing Science and Innovation

We are committed to sharing trusted resources and cutting-edge research to spark medical breakthroughs. While our initial focus is NKX6-2, the insights gained regarding myelin repair and oligodendrocyte health have the potential to support and accelerate scientific development for a wide range of related neurological conditions. We hope to contribute towards innovation and driving discovery that benefits the broader medical community.

A Global Hub for Collaboration

We believe that progress happens faster when we work together. Our platform serves as a vital bridge, connecting families navigating rare diagnoses with world-class scientists and researchers. We are dedicated to building a strong community that will attract the pharmaceutical partners needed to bring these new treatments to life, creating a space where knowledge and hope are shared at every step.