# Translating Discovery to Therapy

> **NIH NIH R13** · UNIV OF NORTH CAROLINA CHAPEL HILL · 2021 · $23,000

## Abstract

The lives of hundreds of thousands of people in the United States are affected by diverse and
devastating conditions involving tissues derived from ectoderm. As an example, ectodermal
dysplasias (ED) represents >100 rare genetic disorders characterized by defects in skin, hair,
nails, teeth, glands (sweat, ophthalmic, respiratory, mammary, etc.), and other derivatives of
ectoderm. Founded in 1983, the National Foundation for Ectodermal Dysplasias (NFED) is the
sole USA and the most established international advocacy group dedicated to EDs. The NFED
has a strong commitment to research and has steadfastly supported research and advocacy to
improve the health and quality of lives of individuals affected by EDs. While advances in
diagnostics and understanding the etiopathology of genodermatoses have markedly advanced,
therapies to address morbidity and mortality for these diverse rare diseases have lagged.
This application requests support for an international conference "Translating Discovery to
Therapy" to be held in Charlotte NC October 21-24, 2021. We will bring together researchers
and scientist from across the globe who are leading investigators in the areas of tissue
regeneration, wound healing, stem cell research, gene therapy and other cutting-edge
technologies and approaches. The goal is to markedly impact the direction and speed of
translating discovery to novel therapeutics directed at addressing the morbidity and mortality
associated with rare disease affecting tissues of ectodermal origin. Bringing investigators
together at this conference will stimulate new collaborations and research directions that focus
on filling critical knowledge gaps and the development of new treatments and therapies for the
diverse populations impacted by these conditions. The conference structure will feature plenary
sessions and focused talks followed by breakout sessions to specifically address the issues of
collaborative research, gaps in knowledge hindering advancement to therapy. We have set
short term and long term goals that include targeting a 1 to 2 year timeline for a therapeutic
deliverable and a long term goal to develop new collaborations and interested investigators to
ensure a sustained effort directed at novel treatment development.
This conference is highly relevant as it addresses a critical health care need and will bring new
directions and new investigators and clinicians with diverse expertise together with a focused
mission. We believe this "moonshot" approach will result in new health care approaches that
can effectively address the significant morbidly and mortality associated with hereditary
conditions involving ectodermal tissues.

## Key facts

- **NIH application ID:** 10318315
- **Project number:** 1R13DE031165-01
- **Recipient organization:** UNIV OF NORTH CAROLINA CHAPEL HILL
- **Principal Investigator:** John T. Wright
- **Activity code:** R13 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $23,000
- **Award type:** 1
- **Project period:** 2021-08-04 → 2022-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10318315

## Citation

> US National Institutes of Health, RePORTER application 10318315, Translating Discovery to Therapy (1R13DE031165-01). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10318315. Licensed CC0.

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