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Targeting Ocular Disorders - Day 1

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Thursday, Sept 26, 2013

12:30-1:55 Conference Registration


1:55 Welcome by Session Chairperson

Guillaume Pfefer, Ph.D., President & CEO, Kala Pharmaceuticals

2:00 A Special Intraocular Contemplation for New Format Antibody Formulations

Dhananjay Jere, Ph.D., Group Leader, PTDE, Hoffmann-La Roche

Development of protein products to target ocular diseases requires special considerations from the molecular designing aspect and also from the formulation, stability, and regulatory aspect. The protein intended to stay in eye has to be compatible and stable in the intraocular environment. This presentation describes key development aspects of new format antibody formulation to make it apt for the intraocular application

2:30 Intrinsic Toxicity Pathways Affecting Survival of Ganglion Cells in Injured Retina

Val Shestopalov, Ph.D., Associate Professor, Ophthalmology, Bascom Palmer Eye Institute, University of Miami Ophthalmology

Aberrant activation of pannexin-1 membrane channel disrupts resilience of these neurons to ischemic, mechanical and other injuries. Mechanistic insights into pathophysiology of pannexin-1 activation suggest that neuroprotective effect strongly correlates with blocking ionic dysbalance and cytokine production. Our results indicate that pharmacological control of pannexin-1 activation can protect against various types of retinal pathologies triggered by ischemic injury, optic nerve stroke and glaucoma.

3:00 Tear Fluid Analysis as a Window into Ocular Health and Disease

Mark W. Duncan, Ph.D., Professor of Medicine, Anschutz Medical Campus, Unviersity of Colorado Denver

We are exploring the potential of tear fluid analysis, especially of peptides and proteins, in defining ocular health and disease by using both targeted and non-targeted analysis strategies.  Tear samples (n=10) were collected from subjects (n=40) directly onto a polymeric support following photorefractive keratectomy (PRK).  Target proteins were assayed by ELISA (both multiplexed and individual assays) and the samples were also examined by matrix-assisted laser desorption/ionization mass spectrometry (MALDI-MS).  The relationships between the tear constituents and their quantitative changes follow surgery will be discussed.

Kala Pharmaceuticals3:30 Breakthrough Solutions for Ocular Diseases

Guillaume Pfefer, President & CEO, Kala Pharmaceuticals

Kala is building a diversified pipeline of ophthalmic product candidates: a best-in-class treatment of ocular inflammation and a first-of-a-kind, completely non-invasive wet age-related macular degeneration (AMD) treatment that will transform the industry. We’ll look at the role of technology to create powerful therapies with significantly less cost and risk.

3:45 Refreshment Break with Exhibit & Poster Viewing


4:30 Antibody Fragment Pharmacokinetics in the Eye and Implications for Bi-Specific Therapeutics 

Justin Scheer, Ph.D., Senior Scientist, Protein Chemistry, Genentech, Inc.

Although many patients achieve positive vision outcomes in response to ranibizumab, unmet need remains for the majority. We will present recent findings on the ocular pharmacokinetics of various antibody formats and describe advances in our bispecific platform for ophthalmic indication.

5:00 Treating Ocular Disease with Therapeutic Mirror Proteins

Dana Ault-Richie, Ph.D., CEO, Reflexion Pharma

Vascular endothelial growth factor (VEGF) is a protein that when aberrantly expressed stimulates the formation of new blood vessels that contribute to tumor growth and macular degeneration. Reflexion's novel approach uses mirror-image protein technology. Antibodies, like all natural proteins, are "left-handed," meaning that they have a particular orientation in their structure. We have created an unnatural "right-handed" VEGF protein.The beauty of this process is that the "right-handed" end product protein can't be so readily metabolized by the body. Thus lower and less frequent doses are possible, and a reduced side-effect profile (such as immune reactions) is likely.

5:30 Cell-Based Therapies for Ocular Disease

Mohammed El-Kalay, Ph.D., Research and Development, EyeCyte, Inc.

PharmOptima6:00-7:00 Welcome Reception with Exhibit & Poster Viewing



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