Retinal disorders including age related macular degeneration, diabetic retinopathy, glaucoma and inherited retinal generations are major causes of blindness in the world. With an increase in life expectancy and growing diabetes epidemic, the socioeconomic burden of retinal disorders is expected to enormous. Rate limiting steps in the development of treatments for retinal disorders include identification of new therapeutic agents as well as new approaches to deliver these agents to the tissues of the back of the eye. With the identification of new therapeutic agents such as siRNA, miRNA, and gene therapies, the need for the development of novel delivery systems also escalates. Several nucleic acid therapies may fail due to the lack of availability of appropriate delivery systems that can a) protect the therapeutic agent from degradation by nucleases, b) allow enhanced cellular entry, c) minimize off-target effects, and d) offer prolonged delivery in treating chronic retinal disorders. Even with successful protein therapeutic agents such as ranibizumab, frequent injections over several years might be detrimental to retinal health. This limitation can potentially be overcome through the development of long term delivery approaches for protein drugs. For small molecule drugs, noninvasive delivery to the back of the eye has yet to become a clinical reality. Further, sustaining the delivery of small water soluble molecules to the back of the eye even by invasive approaches is a major challenge. In order to expedite the translation of new small molecule, protein, and nucleic acid therapeutic agents from the bench to the bedside, the purpose of this conference is to share cutting edge science based on drug product development principles among a diverse group of participants, including representatives from academia, industry, and regulatory agencies. The audience is expected to be drawn from a diverse group of individuals including those representing minorities and traditionally underrepresented communities in science careers. The two day conference will include oral as well as poster presentations. Financial support provided for this conference will be helpful in recruiting top scientists as well as participants. In addition, registration fee waivers and travel awards will be provided in order to encourage participation by minorities and underrepresented communities. Based on the presentations and discussions at the conference, a white paper will be prepared within 3 months after the conference and submitted for publication in a timely manner in an ophthalmology journal.

Public Health Relevance

Blinding retinal disorders have a major socioeconomic impact. A rate limiting step in advancing new therapeutic agents from bench to bedside is the delivery to the affected target cells. Each class of therapeutic agents poses unique problems in developing clinically usable delivery systems. Most recently, siRNA based therapies have met with limited success potentially due to their off-target effects on the cell surface. Such therapeutic agents should enter the cell, while avoiding interactions with some cell surface receptors. In order to expedite the clinical translation of new therapeutic agents, the purpose of this conference is to present cutting edge science based approaches for developing new drug products for treating back of the eye diseases.

National Institute of Health (NIH)
National Eye Institute (NEI)
Conference (R13)
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Special Emphasis Panel (ZEY1-VSN (02))
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Shen, Grace L
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University of Colorado Denver
Schools of Pharmacy
United States
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Rowe-Rendleman, Cheryl L; Durazo, Shelley A; Kompella, Uday B et al. (2014) Drug and gene delivery to the back of the eye: from bench to bedside. Invest Ophthalmol Vis Sci 55:2714-30