# MANUFACTURING OF PLASMID AND VECTOR AND GLP DOSE-ESCALATION TOXICOLOGY STUDIES

> **NIH NIH N01** · BATTELLE CENTERS/PUB HLTH RES & EVALUATN · 2024 · $131,706

## Abstract

Currently, the National Institute of Neurological Disorders and Stroke (NINDS), via its URGenT network,
is funding a cooperative agreement for the manufacturing of an AAV9 vector, plasmids, and GLP
dose-escalating studies as a potential therapy the severe and progressive genetic neurological disorder,
Aspartylglucosaminuria (AGU). The goals of the URGenT cooperative agreement are to manufacture the vector, plasmid, and initial GMP batch.
Specifics of the process will come during the tech transfer described in the base task. The final product
qualification and characterization testing will include assays of sterility, endotoxin, purity, identity,
activity/potency, vector genome titer, capsid particle titer, appearance, and genomic structure. Upon
establishment of the vectors, and optimal characterization, this project will progress to IND-enabling toxicology studies to support the IND.

## Key facts

- **NIH application ID:** 11172202
- **Project number:** 75N95022D00021-P00001-759502300003-1
- **Recipient organization:** BATTELLE CENTERS/PUB HLTH RES & EVALUATN
- **Principal Investigator:** Amanda Burnaugh
- **Activity code:** N01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $131,706
- **Award type:** —
- **Project period:** 2023-09-30 → 2026-09-29

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11172202

## Citation

> US National Institutes of Health, RePORTER application 11172202, MANUFACTURING OF PLASMID AND VECTOR AND GLP DOSE-ESCALATION TOXICOLOGY STUDIES (75N95022D00021-P00001-759502300003-1). Retrieved via AI Analytics 2026-05-28 from https://api.ai-analytics.org/grant/nih/11172202. Licensed CC0.

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