Translating Discovery to Therapy

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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.