Current Program Status

In just six months, Sweet Geej Foundation has rapidly advanced its ASO discovery program from initial sequence design to a human CRISPR disease model—an important translational milestone in the development of a precision therapy for NAA15 haploinsufficiency.

Working in collaboration with La Jolla Labs, iXcells, and in our own lab space led by Michelle Mattson-Hoss, PhD, and other specialized partners, we have systematically progressed through increasingly rigorous stages of evaluation to identify the strongest therapeutic candidates and undergone early safety and tolerability studies.

Current Stage: Human CRISPR Disease Model

The program has now advanced into a CRISPR-engineered human cell model carrying GG's NAA15 mutation.

ASO candidates are currently being evaluated to determine which molecules most effectively restore NAA15 expression in a disease-relevant cellular system.

The results will identify the strongest candidates to advance into GG's patient-derived neurons before entering preclinical safety studies.