Drug and Gene Delivery to the Back of the Eye: From Bench to Bedside

• Type of Material: Article
• Authors: Cheryl L. Rowe-Rendleman, Omar Consulting Group LLC; Shelley A. Durazo, University of Colorado Anschutz Medical Campus; Uday B. Kompella, University of Colorado Anschutz Medical Campus; Kay D. Rittenhouse, Pfizer Inc; Adriana Di Polo, University of Montreal; Alan L. Weiner, DrugDel Consulting LLC; Hans Grossniklaus, Emory University; Muna I. Naash, University of Oklahoma Health Sciences Center; Alfred S. Lewin, University of Florida; Alan Horsager, University of Southern California; Henry Edelhauser, Emory University
• Language: English
• Date: 2014-04-01
• Publisher: The Association for Research in Vision and Ophthalmology Inc
• Publication Version: Final Published Version
• Copyright Statement: Copyright 2014 The Association for Research in Vision and Ophthalmology, Inc.
• License: Creative Commons Attribution 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1167/iovs.13-13707
• Title of Journal or Parent Work: Investigative Ophthalmology and Visual Science
• Volume: 55
• Issue: 4
• Start Page: 2714
• End Page: 2730
• Grant/Funding Information: Supported by the Association for Research in Vision and Ophthalmology (ARVO); the National Eye Institute Grant 1R13EY021956-01 (UBK); Pfizer; grants from Bausch & Lomb (Rochester, NY, USA); Ora (Andover, MA, USA); and Santen USA (Emeryville, CA, USA).
• Keywords: ADENOASSOCIATED VIRUS; DIPEPTIDE PRODRUGS; retina; gene therapy; RPE65 MUTATIONS; Life Sciences & Biomedicine; Ophthalmology; Science & Technology; VISUAL FUNCTION; PHARMACOKINETICS; drug delivery; nanoparticle; MOUSE; THERAPY; LEBER CONGENITAL AMAUROSIS; translational medicine; COMPACTED DNA-NANOPARTICLES; RETINAL GANGLION-CELLS
• Research Categories: Biology, Genetics; Health Sciences, Opthamology

Abstract

The ARVO 2012 Summer Eye Research Conference (SERC 2012) on “Drug and Gene Delivery to the Back of the Eye: From Bench to Bedside” was held June 15 and 16, 2012, at the University of Colorado Anschutz Medical Campus in Aurora, Colorado. The SERC provided a diverse group of approximately 150 scientists and physicians representing industry and academia from 14 countries with a unique opportunity to explore the latest approaches to drug and gene delivery to the posterior segment of the eye. Unlike the 2009 SERC meeting, which focused on novel drug delivery platforms while elucidating the anatomic barriers to reach the posterior segment, 1 the most recent meeting explored strategies for bypassing ocular barriers using novel materials, nanoparticulate delivery systems, and gene therapy. It brought together experts in both ophthalmology and tangentially related areas to discuss the application and inherent technical challenges for translating experimental results from the laboratory bench to dependable medical therapies at the bedside and, where possible, it exemplified findings in ocular models with methods and results gleaned from disciplines outside of ophthalmology. The present review of the SERC provides investigators with tools to navigate these nascent approaches by exploring strategies from key laboratory investigations, drug development specialists, and clinical trials. The 2-day conference comprised the following six sessions: (1) barriers to drug delivery and transporter-guided drug design; (2) drug/gene delivery systems and cell therapies for the eye; (3) pharmacokinetics (PK), pharmacodynamics, and alternative routes of drug delivery; (4) nanotechnology for diagnosis and treatment of posterior eye disease; (5) translation of gene delivery for posterior eye disease; and (6) clinical trials. Rather than being a deliberate summary of each presentation, this review describes the common themes expressed during the six sessions.