RESEARCH UPDATES
RESEARCH UPDATES
GAND Gene Therapy Development Roadmap
We are pleased to present the official development roadmap for the GAND gene therapy program. This document outlines the scientific strategy, funding secured, planned milestones, and the pathway toward a first-in-human clinical trial between 2025 and 2028.
2025–2028 Program Development & Funding Plan
PROGRAM HIGHLIGHTS
→ $580,478 secured in funding
→ Phase 1 discovery program initiated
→ Mouse model development underway
→ Preclinical validation planned for 2026
→ IND-enabling studies targeted for 2027
→ First-in-human clinical trial preparation underway
Every contribution moves the GAND gene therapy program closer to clinical translation. This roadmap reflects our long-term commitment to developing the first disease-modifying treatment for children living with GAND syndrome.
December 2025 Research Progress Report
We are proud to share the first official research progress report for the GAND gene therapy program.
This report outlines the scientific progress achieved in 2025, including construct development, cell testing, translational planning, and next research milestones toward clinical development.
Key Research Highlights
→ 15 therapeutic constructs successfully developed
→ Initial neuronal cell testing completed
→ Two lead candidates selected for translational studies
→ Patient-derived organoid testing planned
→ GAND mouse model development initiated
→ Translational studies expected through 2026
Every step forward in this program is made possible through the support of families, researchers, clinicians, and donors who believe that children with GAND syndrome deserve a future with treatment options.