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Targeting Ocular Disorders - Day 1

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2014 Archived Content

Monday, October 6

1:00 pm Registration

2:00 Opening Remarks by Chairperson

James Larrick, MD, Ph.D., Co Founder and Managing Director, Panorama Research Institute, Wntgen LLC & Velocity Pharmaceutical Development LLC


2:10 Systemic Administration of Anti-Ab mAb Reduces Retinal Deposition of Abeta and Activated Complement C3 in Age-Related Macular Degeneration Mouse Model 

Ian R. Catchpole, Ph.D., Topical BioPharm Discovery Research and Development, Glaxo-Smith Kline

Cfh-/- mice were treated systemically in both prophylactic andtherapeutic regimes with an anti-Ab monoclonal antibody (mAb), 6F6, todetermine the effect on the cfh-/- retinalphenotype. Prophylactic treatment with 6F6 demonstrated a dose dependent reduction in the accumulation of both Aband activated C3 deposition. SerumAb levels after systemic administration of 6F6 show accumulation of Ab in the periphery suggestive of a peripheral sinkmechanism. In summary, anti-Ab mAb treatment can partially prevent or reverse ocular phenotypes of the cfh-/- mouse. The data support this therapeutic approach in humans potentially modulating two key elements in the pathogenesis of AMD – Ab and activated, complement C3.


2:40 Oral Therapies for Retinal Degeneration: Case Study of ALK-001

Leonide Saad, Ph.D., CEO, Alkeus Pharmaceuticals, Inc.

Safely attaining biologically active sustained amounts of a compound in the retina is notoriously difficult. Here we present how compounds delivered orally can find their way to the retina. We further discuss the science behind ALK-001, an oral investigational new drug designed to inhibit the formation of toxic by-products thought to be responsible for major retinal degenerations such as AMD, Stargardt, Best disease or autosomal recessive Retinitis Pigmentosa.

3:10 Wnt Pathway Inhibition for Diabetic Retinopathy

James Larrick, MD, Ph.D., Co-Founder and Managing Director, Panorama Research Institute, Wntgen LLC & Velocity Pharmaceutical Development

Activation of the Wnt pathway via LRP6 cell surface signaling contributes to the severe retinal/choroidal neovascularization observed in Vldlr-/- mice. Elevated Wnt signaling is also observed in human retinas of patients with diabetic retinopathy. We identified a novel murine monoclonal antibody that antagonizes LRP6 activity which demonstrates significant activity in numerous animal models of retinal disease. A humanized form of this antibody may be a novel therapy for diabetic retinopathy.

3:40 MANF - A Novel Neurotrophic Factor for the Treatment of Retinal Disorders
Roman Urfer, Ph.D., Chief Development Officer, NeuroAssets Sarl
Mesencephalic astrocyte-derived neurotrophic factor is the prototype of an emerging family of neurotrophic factors with evolutionary highly conserved structure and function. MANF is expressed in response to cellular stress and during retinal development. MANF's activity in genetic and pharmacolocical models of retinal disorders will be presented.

3:55 Refreshment Break in the Exhibit Hall with Poster Viewing

4:25 Nanoliter Scaled Immunoassays: Enabling Biomarker Assay Development in Low Volume Ocular Samples
Robert A. Durham, Ph.D., Director, Field Applications North America, Gyros US, Inc.
Biotherapeutic approaches that target diseases of the eye pose a bioanalytical challenge due to the small sample volume of relevant matrices in the target organ. To this end we have evaluated the feasibility of using a nanoliter-scaled immunoassay on the Gyrolab for pharmacokinetic and biomarker assays to support ocular diseased projects. This presentation will review data from Biomarker and PK measurements made in vitreous humor, and tear samples.  The results show that the Gyrolab platform enables development of low volume assays for PK and biomarker measurements in the eye.


4:55 Combination Therapy in Diseases of Retinal Origin: A New Paradigm  

David Sherris, Ph.D., CSO, RestorGenex, Corp.

Neovascular retinal and subretinal diseases as in age-related macular degeneration and proliferative diabetic retinopathy occur through a cascade of events over time. Numerous cytokines have shown involvement throughout the disease process. Anti-VEGF technologies inhibit edema and hemorrhage due to their ability to reduce vascular permeability and to some extent inhibit neovascularization. However, VEGF is not the only cytokine in these pathologies. Here we will discuss the influence of a cytokine storm in back-of-the-eye diseases with the example of P529, a first-in-class, allosteric, dual dissociative inhibitor of the TORC1 and TORC2 complexes within the PI3K/Akt/mTOR pathway in combination with anti-VEGF.

5:25 Welcome Reception in the Exhibit Hall with Poster Viewing

6:25 Close of Day

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