Therapeutic Strategy

Restoring What's Missing

GG’s NAA15-related neurodevelopmental disorder is caused by haploinsufficiency: she carries one functional copy of the NAA15 gene, but that single copy doesn't produce enough protein to meet the brain's (or body’s) developmental demands. The goal isn't to repair a mutation — it's to help the body make more of what it already knows how to make.

An Antisense Oligonucleotide Approach

Antisense oligonucleotides (ASOs) are short, synthetic strands of nucleic acid that can be designed to interact with specific regions of a gene's RNA. Sweet Geej Foundation is developing an ASO that targets the 5' untranslated region (5'UTR) of the NAA15 gene — a regulatory stretch of RNA that sits upstream of the protein-coding sequence and helps control how efficiently the gene is translated into protein.

By engaging this regulatory region, our lead ASO is designed to increase NAA15 protein output from the patient's intact gene copy — without altering the DNA itself.

Where We Are

A lead ASO has been identified and validated in GG's patient-derived neurons, where it increased NAA15 protein levels with no adverse effect on cell health. The program is now advancing into IND-enabling preclinical studies in support of a first-in-human clinical trial.