# OFF-TARGET RESOURCE CORE

> **NIH NIH U19** · CHILDREN'S HOSP OF PHILADELPHIA · 2024 · $719,196

## Abstract

PROJECT SUMMARY
Off-target editing analyses will be so fundamental to the development of our therapeutic leads, regardless of the
disease treated—whether phenylketonuria (Lead Project 1), hereditary tyrosinemia type 1 (Project 2), or
mucopolysaccharidosis type I (Project 3)—and so integral to any IND applications that ultimately emerge from
the Projects, that we are proposing an Off-Target Resource Core that will serve all three Research Projects.
This Resource Core will provide services that are directly aligned with the preclinical studies that are
recommended by the U.S. Food and Drug Administration (FDA) Draft Guidance for Industry on Human Gene
Therapy Products Incorporating Human Genome Editing, namely: (1) identification of on-target and off-target
editing activity, including the type, frequency, and location of all off-target editing events, taking into account
human genetic variation; (2) assessment of genomic integrity, including chromosomal rearrangements, large
insertions, or large deletions; and (3) evaluation of the biological consequences associated with off-target editing.

## Key facts

- **NIH application ID:** 10896257
- **Project number:** 5U19NS132301-02
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** Xiao Wang
- **Activity code:** U19 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $719,196
- **Award type:** 5
- **Project period:** 2023-08-01 → 2028-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10896257

## Citation

> US National Institutes of Health, RePORTER application 10896257, OFF-TARGET RESOURCE CORE (5U19NS132301-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10896257. Licensed CC0.

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