Ophthalmological pharmacology has historically lacked systematic understanding of disease mechanisms, especially of those for the back of the eye. This is now changing with the discovery of novel targets and pathways paired with a greater understanding of the role of genetics in ocular pathologies.

The third annual Targeting Ocular Disorders will cover some of the most promising emerging therapies in the treatment of ocular diseases. Special focus with be given to age-related macular degeneration and retinopathy. Designated sessions will emphasize the role of precise imaging techniques and animal models in accelerating preclinical development. Recent solutions in drug delivery to the back of the eye will also be discussed.

Final Agenda

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Tuesday, September 22

7:00 am Registration and Morning Coffee


8:00 Chairperson’s Opening Remarks

Eric Furfine, Ph.D., CSO, Eleven Biotherapeutics, Inc.

8:10 Keynote Presentation: New Horizons for Glaucoma Therapy

Naj Sharif, Ph.D., FARVO, Executive Director and Head, Global Biomedical Sciences, Santen, Inc.

Glaucoma and the associated ocular hypertension seriously threaten vision in >60 million patients. Reduction of elevated intraocular pressure (IOP) in glaucoma remains the major treatment option. New classes of IOP-lowering agents worthy of clinical pursuit include NO-donor-PGA conjugates, Rho kinase inhibitors, ACE-2 activators, non-peptide bradykinin agonists, serotonin-2 and EP2 receptor agonists. An overview of such new therapeutics will be presented.

8:40 Use of High Content Screening to Identify Targets for the Treatment of Glaucoma and the Retinal Degenerative Disease

Donald Zack, M.D., Ph.D., Guerrieri Professor of Genetic Engineering and Molecular Ophthalmology, Johns Hopkins University

Phenotypic screening, as opposed to traditional target-based screens, has proven successful in the development of molecular probes and lead molecules for a number of complex diseases. We have taken such a phenotypic approach to identify small molecules that promote the differentiation, function, and survival of murine primary and human stem cell-derived retinal neurons.

9:10 Nitric Oxide-cGMP Signaling as a Therapeutic Target for Glaucoma

Emmanuel Buys, Ph.D., Assistant Professor, Anesthesia, Harvard Medical School; Associate Scientist, Broad Institute of MIT and Harvard

The nitric oxide (NO)-soluble guanylate cyclase (sGC)-cyclic guanosine monophosphate (cGMP) pathway regulates intraocular pressure (IOP). Preclinical and clinical studies have demonstrated the ability of NO to lower IOP (e.g. VESNEO®). The therapeutic potential of targeting NO-cGMP signaling in glaucoma will be reviewed.

9:40 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing

10:25 Local modulation of cytokine signaling to treat anterior and posterior ocular disorders

Eric Furfine, Ph.D., CSO, Eleven Biotherapeutics, Inc.

Cytokines, chemokines, and growth factors mediate anterior and posterior eye diseases. Our lead product, the IL-1 receptor inhibitor EBI-005, was designed and engineered for the topical treatment of dry eye disease and was biologically active in subjects with dry eye disease. In addition, we engineered an IL-6 inhibitor with a long intravitreal half-life with potential for local treatment diabetic macular edema and other posterior ocular disorders.

10:55 Retinal Inflammasome as a Novel Drug Target in Ischemia and Glaucoma

Valery Shestopalov, Ph.D., Professor, Ophthalmology, University of Miami Miller School of Medicine; Bascom Palmer Eye Institute

We discovered robust activation of the inflammasome in the mouse retina with induced glaucoma, which localized primarily to the inner retina. Inactivation of inflammasome core enzymes of upstream regulators showed significant protection in mouse disease models. These experimental evidence support that injury-induced inflammasome induces major neurotoxicity to RGCs and endorses inflammasome pathway as a novel drug target in acute glaucoma.

11:25 Targeting Rho Kinase for the Treatment of Ocular Diseases

Casey Kopczynski, Ph.D., CSO, Aerie Pharmaceuticals, Inc.

I will present the discovery and development of AR-13324, a novel small molecule inhibitor of Rho kinase and Norepinephrine transporter currently in Phase 3 trials for glaucoma. The development of Rho kinase inhibitors for age-related macular degeneration will also be presented.

11:55 Title TBA

Maria Bartolomeo Grant, Ph.D., Marilyn Glick Professor, Ophthalmology, Ophthalmology Center, Indiana University, Eugene & Marilyn Glick Eye Institute

12:25 pm Session Break

12:35 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

1:15 Refreshment Break in the Exhibit Hall with Poster Viewing

1:50 Chairperson’s Remarks

Robert Ferry, Ph.D., Professor, Pediatrics, University of Tennessee Health Science Center

2:00 Nuclear Receptors as Therapeutic Targets for Age-Related Macular Degeneration

Goldis Malek, Ph.D., Associate Professor, Ophthalmology, Duke University School of Medicine

Nuclear receptors are transcription factors that play varied roles during development, aging, and disease. The aryl hydrocarbon receptor (AhR) is a candidate AMD associated receptor identified from screening a nuclear receptor atlas of human retinal pigment epithelial cells. We will discuss the potential role of AhR in AMD development and consequence of targeting AhR activity on pathobiology associated with wet AMD.

2:30 Topical, Beta-Adrenergic Agonist as Novel Approach to Treat Diabetic Macular Edema and Retinopathy (DMER)

Robert Ferry, Ph.D., Professor, Pediatrics, University of Tennessee Health Science Center

Reduced sympathetic tone reproduces DMER phenotype in rodents. Applied topically, a novel, patent-pending, ß-adrenergic agonist safely prevented histologic and functional DMER in diabetic rats. No deleterious effects were detected after repeated topical administration to eyes of legally blind, diabetic humans.

3:00 Sponsored Presentation (Opportunity Available)

3:30 Refreshment Break in the Exhibit Hall with Poster Viewing and Poster Winner Announced


4:10 Utility of in vivo Confocal Microscopy-Based Imaging Endpoints for the Assessment of Ocular Surface Inflammation

Pedram Hamrah, M.D., Assistant Professor, Ophthalmology, Harvard Medical School; Director, Ocular Surface Imaging Center, Massachusetts Eye and Ear Infirmary

Recent studies have shown the role of immune changes in the pathogenesis of dry eye disease (DED) and a sensitive test is needed to quantify the immune response. The purpose of this study is to evaluate the utility of in vivo confocal microscopy changes in corneal and conjunctival inflammation to therapeutic intervention.

4:40 Using Posterior Segment OCT as a Biomarker in Disease

Jay S. Duker, M.D., Professor and Chair, Ophthalmology, Tufts University School of Medicine; Director, New England Eye Center

Optical Coherence Tomography (OCT) is a non-invasive, highly reproducible imaging modality that provides quantitative measurements of retinal anatomy with micron resolution. OCT provides important biomarkers for disease states that permits researchers, clinicians and drug developers to monitor the status of a variety of ocular disease.

5:10 Interactive Breakout Discussion Groups

Lessons learned from MEAD, RIDE, RISE, and Protocol T (Diabetic Retinopathy Clinical Research Network) trials to guide future studies

Host:  Robert Ferry, M.D.., Professor, Pediatrics, University of Tennessee Health Science Center

  • Optimizing outcome measures (e.g., VFQ-25 vs. central macular thickness vs. best corrected visual acuity vs. posterior retinal non-perfusion or other outcomes with respect to prospective glycemic control over 1 year)
  • Successful strategies to maximize trial enrollment and retention (vs. less successful approaches)
  • Which optical coherence tomography is best for DME trials? (e.g., time domain, spatially-encoded frequency domain, time-encoded frequency domain, single point confocal, parallel or full field, smart detector array for parallel OCT)

Pros and cons of developing back-of-the-eye therapeutics, is this really where you want to go?

Host:  David Sherris, Ph.D., President & CEO, GenAdam Therapeutics

  • Target identification and proof of concept
  • Formulation issues
  • Ocular toxicities

Retinal inflammasome as a novel drug target in ischemia and glaucoma

Host:  Valery Shestopalov, Ph.D., Professor, Ophthalmology, University of Miami Miller School of Medicine; Bascom Palmer Eye Institute

  • Molecular targets of retinal inflammasome:  upstream regulators vs caspases vs endproducts
  • Therapeutic effects and systemic toxicity
  • Inflammasome targeting strategy in AMD:  can we apply them to ischemia or glaucoma?

6:10 Welcome Reception in the Exhibit Hall with Poster Viewing

7:15 Close of Day

Day 1 | Day 2 | Short Course | Download Brochure

Wednesday, September 23

7:30 am Registration and Morning Coffee


8:00 Chairperson’s Remarks

Abraham Scaria, Ph.D., Senior Scientific Director, Ophthalmology, Genzyme, a Sanofi Company

8:10 Gene Therapy for Wet-AMD

Abraham Scaria, Ph.D., Senior Scientific Director, Ophthalmology, Genzyme, a Sanofi Company

VEGF antagonists are useful for treating neovascular AMD, however current treatments require chronic intravitreal injections. We have demonstrated long-term efficacy with minimal side effects following a single intravitreal delivery of a gene therapy AAV-sFLT01 (soluble VEGF-R) in rodents and non-human primate models. A Phase I clinical trial is almost completed at four clinical sites in the USA.

8:40 Long Acting Delivery of Antibody Therapeutics to the Back of the Eye

Debby Chang, Ph.D., Associate Scientist, Genentech, Inc., a Member of the Roche Group

Successful macromolecular therapies for treating posterior eye diseases have spurred the development of long acting delivery technologies. The objective is to reduce treatment burden by decreasing frequency of intravitreal injection. Here, we present an overview of ocular delivery systems for long-term delivery of antibody therapeutics. Promising sustained release formulations and implanted devices will be discussed.

9:10 Intraocular Implant Technology Providing Sustained Delivery of Therapeutic Proteins to the Back of the Eye

Konrad Kauper, MSc, Vice President, Core Technology Development, Neurotech Pharmaceuticals, Inc.

While intravitreal therapies targeting VEGF have led to a paradigm shift in the treatment of neovascular AMD, dosing frequency and compliance issues remain. To address the need for sustained, long-term drug delivery, Encapsulated Cell Therapy, an intraocular implant of a proprietary human cell line genetically engineered to constitutively produce therapeutic proteins for two years, may provide a novel treatment solution.

9:40 Coffee Break in the Exhibit Hall with Poster Viewing


10:25 Human Clinical Trials of AAV-Based Gene Therapies for Orphan Ocular Disorders

Stephen W. Potter, MBA, Chief Business Officer, AGTC

AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop AAV-based gene therapy products for severe inherited orphan diseases in ophthalmology. AGTC expects initial data in human subjects for its lead programs, for the treatment of X-linked retinoschisis and achromatopsia, during the second half of 2015.

10:55 Targeting the Inflammasome Signaling in a Mouse Model of a Geographic Atrophy: A Gene Therapy Approach

Cristhian J. Ildefonso, Ph.D., Senior Postdoctoral Associate, Laboratory of Dr. Alfred S. Lewin, Molecular Genetics & Microbiology, University of Florida College of Medicine

Geographic Atrophy (GA) is associated with oxidative stress and inflammation driven by the inflammasome signaling pathway. We developed and characterized AAV vectors deliver secretable and cell-penetrating proteins targeting this pathway. These vectors are being tested in a GA mouse model.

11:25 Enjoy Lunch on Your Own

12:55 pm Plenary Keynote Program

2:40 Refreshment Break in the Exhibit Hall with Poster Viewing

3:25 Close of Conference

Recommended Short Course:  

Wednesday, September 23, 7:00-9:30 pm

SC9: Preclinical Animal Models for Ocular Indications

The goal of the workshop is to provide an introductory overview of current preclinical animal models for ocular indications. The presentations will focus on models for Age-Related Macular Degeneration, Ocular Inflammation and Glaucoma. An overview of the characteristics of different animal models, their pros and cons and potential uses will be discussed.


Andy Whitlock, Ph.D., Senior Director, Pre-Clinical Research & Development, Ophthalmology, Ora, Inc.

Goldis Malek, Ph.D., Associate Professor, Ophthalmology, Duke University School of Medicine

Maria B. Grant, M.D., FARVO, Professor, Ophthalmology, Eugene and Marilyn Glick Eye Institute, Indiana University

Detailed Agenda

Day 1 | Day 2 | Short Course | Download Brochure


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