# Chemically controlling chromatin to treat Friedriech's Ataxia

> **NIH NIH R41** · EPIGENOS BIOSCIENCE, INC. · 2020 · $251,236

## Abstract

We recently developed a technology that combines catalytically inactive dCas9 with bifunctional
chemicals that link endogenous host cellular machinery to specific genes targeted with dCas9.
We have demonstrated this technology can significantly increase gene expression at multiple
endogenous mammalian genes in a chemically dependent manner. Here we propose to apply
this technology to the mono-allelic rare disease with no current cure, Friedreich’s Ataxia. This
disease is caused by a GAA expansion in the first intron of the frataxin gene which leads to
polymerase pausing and lower gene expression. This proposed project will evaluate if targeting
the frataxin gene with our combination of protein and chemical activator can restore expression
levels of frataxin to a point which reverses pathology of this disease in a modern model of
Friedreich’s Ataxia. We will use patient derived Friedreich’s Ataxia models in specific assays that
intricately measure mitochondria function and metabolic activity that is typically dysregulated in
patients that is the hallmark of this human disease. This work has the potential to lead to new
treatment paradigms for individuals afflicted by this aggressive disease.

## Key facts

- **NIH application ID:** 10009926
- **Project number:** 1R41TR003245-01
- **Recipient organization:** EPIGENOS BIOSCIENCE, INC.
- **Principal Investigator:** Nathaniel A. Hathaway
- **Activity code:** R41 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $251,236
- **Award type:** 1
- **Project period:** 2020-04-01 → 2022-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10009926

## Citation

> US National Institutes of Health, RePORTER application 10009926, Chemically controlling chromatin to treat Friedriech's Ataxia (1R41TR003245-01). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10009926. Licensed CC0.

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