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 and other specialized partners, we have systematically progressed through increasingly rigorous stages of evaluation to identify the strongest therapeutic candidates.

Discovery and Lead Optimization

Approximately 80 ASO candidates were designed and screened against regulatory regions of the NAA15 gene.

Through multiple rounds of testing, we identified a group of lead candidates demonstrating:

• Robust NAA15 mRNA upregulation

• Reproducible activity across independent experiments

• Dose-dependent biological responses

• Consistent increases in NAA15 protein production

Demonstrating reproducible protein restoration represents a major milestone for the program and provides strong evidence that our ASOs are producing the therapeutic molecule needed by cells.

Current Stage: Human CRISPR Disease Model

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

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

These studies are measuring:

• NAA15 mRNA restoration

• NAA15 protein restoration

• Dose-response characteristics

• Relative performance across all twelve candidates

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