Development of an NAA15 Antisense Oligonucleotide (ASO)

Sweet Geej Foundation is developing a mutation-agnostic antisense oligonucleotide (ASO).

Our development strategy follows a rigorous, data-driven translational pathway used throughout biotechnology: identify active molecules, validate activity in disease-relevant human models, and advance the strongest candidates into preclinical safety studies.

CRISPR Disease Model Validation

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

ASO candidates are currently being evaluated in this disease-relevant model to determine which molecules are most effective for NAA15 haploinsufficiency.

Validation in Patient-Derived Neurons

Following completion of the CRISPR disease model studies, the top-performing ASOs will be evaluated in GG's patient-derived induced pluripotent stem cell (iPSC) neurons.

Testing will include:

• Glutamatergic neurons

• GABAergic neurons

• Functional neuronal activity

Lead Candidate Selection and Preclinical Advancement

The lead candidate(s) will then advance into IND-enabling preclinical development, including formal safety, toxicology, pharmacology, and manufacturing activities required to support a first-in-human clinical trial.