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.

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