Scientific Ecosystem
Where We Are Now: Advancing Toward Targeted Therapy
The Sweet Geej initiative is currently focused on the design and early development of a precision antisense oligonucleotide (ASO) therapy for GG, a child with NAA15 haploinsufficiency. Our goal is to increase expression from her healthy copy of the NAA15 gene, supporting neurological development and functional regulation.
This phase represents a critical bridge between scientific understanding and therapeutic action.
Building the Foundation for ASO Development
We are actively collaborating with academic and industry partners to design and evaluate ASO candidates tailored to GG’s genetic profile.
This work includes:
ASO design and optimization informed by NAA15 gene regulation
Early screening strategies to identify candidates that increase functional gene expression
Cross-disciplinary scientific input across genomics, neuroscience, and translational medicine
This work is being conducted with urgency, while maintaining the rigor required for an N-of-1 therapeutic program.
Leveraging Patient-Derived Biology
A central component of this effort is the use of GG’s patient-derived induced pluripotent stem cell (iPSC) line.
These cells allow us to model disease-relevant biology in human neurons and related cell types, enabling:
Measurement of gene and protein expression
Evaluation of cellular function and signaling
Assessment of biological response to ASO candidates
This approach grounds development in GG’s own biology rather than relying solely on generalized models.
Generating Preclinical Evidence
We are currently generating foundational preclinical data to guide next steps, including:
Functional and expression-based readouts in neuronal systems
Early indicators of biological response and feasibility
Data to support future regulatory and clinical planning
A Translational Path Forward
This work is designed to move thoughtfully from molecular insight to therapeutic readiness. While centered on GG, the knowledge gained may also inform broader understanding of NAA15-related conditions and haploinsufficiency-driven neurodevelopmental disorders.