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Targeting Ocular Disorders - Day 2

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2014 Archived Content

Tuesday, October 7

8:15 am Morning Coffee


8:45 Chairperson Remarks

Eric Furfine, Ph.D., CSO, Eleven Therapeutics

8:50 A Synthetic Chemically Modified siRNA Targeting Caspase-2 as a Therapeutic Agent for Ocular Neuroprotection

Elena Feinstein, M.D., Ph.D., CSO, Quark Pharmaceuticals

Caspase-2 is a pro-apoptotic gene specifically expressed and activated in retinal ganglion cells following acute optic nerve injury or intraocular pressure increase. Intravitreal administration of QPI1007 (a syntehticsiRNA targeting caspase-2) demonstrated local RNA interference, lack of inflammatory effects and excellent preclinical and clinical safety profiles. Neuroprotective activity of QPI1007 has been demonstrated in five preclinical animal models involving RGC loss. In human Phase I trials in NAION patients, a single intravitreal injection of QPI1007 within 2 weeks following disease onset prevented further visual deterioration.

9:20 Nanobodies® as Next-Generation Therapeutics for Ocular Applications

Tony De Fougerolles, Ph.D., CSO, Ablynx NV

Next-generation ophthalmic therapeutics must aim at being superior to the currently available agents with regards to efficacy and improved drug delivery. We show here that Nanobodies deliver these properties and present a novel class of highly potent and specific drug candidates for the treatment of eye disorders.

9:50 Local Inhibition of Cytokine Signaling to Treat Anterior and Posterior Ocular Disorders

Eric Furfine, Ph.D., CSO, Eleven Therapeutics

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 potential for local treatment diabetic macular edema. Finally, a novel soluble receptor inhibitor of cytokines IL-17A and IL-17F was engineered for the local treatment of uveitis. Both IL-6- and IL-17-targeted drugs were designed and engineered for intravitreal administration.

10:20 Coffee Break in the Exhibit Hall with Poster Viewing

10:50 Topical Aganirsen in Eye Diseases: From Phase III Results in the Cornea to Retinopathies - A New Revolution

Eric Thorin, Chief Development Officer, Gene Signal International SA

The antisense aganirsen inhibits the expression of Insulin Receptor Substrate-1 (IRS-1). The phase III showed that its topical application induces regression of pathological corneal neovascularization and reduces the need for corneal transplantation. Topical aganirsen reaches the retina, which is at the basis of its clinical development for the treatment of AMD and DME. Aganirsen should become the first therapy active by topical application for the treatment of angiogenesis-based retinopathies.

11:20 NLRP3 Inflammasome Activation in Retinal Pigment Epithelial Cells by Lysosomal Destabilization: Implications for Age-Related Macular Degeneration

Bruce R. Ksander, Ph.D., Associate Professor, Ophthalmology, Schepens Eye Research Institute/Massachusetts Eye and Ear

NLRP3 upregulation occurs in the RPE during the pathogenesis of advanced AMD, in both geographic atrophy and neovascular AMD. Destabilization of RPE lysosomes induces NLRP3 inflammasome activation, which may contribute to AMD pathology through the release of the proinflammatory cytokine IL-1β and through caspase-1-mediated cell death, known as “pyroptosis.”

11:50 Maximizing Chances of Success in the Clinic:  The Role of Animal Models in the Development of Ocular Therapeutics
Claire M. Gelfman, Ph.D., Director, Pre-Clinical Services, Ora 
Achieving proof-of-concept in animal models of ophthalmic disease is a commonly desired prerequisite for advancing drugs into the clinic. The choice of endpoints for evaluation should mirror, when possible, those that will be evaluated in the clinic. In this presentation, we will review several models that recapitulate specific features of human ophthalmic disease, and discuss the integration of that information into the larger clinical development plan in order to maximize chances of success. 

12:20 pm Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

1:50 Chairperson’s Remarks

Sue Washer, President and CEO, Executive, AGTC


1:55 AAV-Based Gene Therapy for Genetic Ocular Diseases

Sue Washer, President and CEO, Executive, AGTC

The utility of AAV-based gene therapy for the treatment of ocular disorders has been validated in human clinical trials for Leber congenital amaurosis and wet AMD. Given their safety and persistent expression, AAV vectors are an optimal approach to providing long-lasting treatment for genetic eye diseases such as archromatopsia, X-linked retinoschisis, and X-linked retinitis pigmentosa. Current investigational AAV-based therapies for these indications will be discussed.

2:25 Porous Poly({varepsilon}-caprolactone) Scaffolds for Retinal Pigment Epithelium Transplantation

Magali Saint Geniez, Ph.D., Assistant Scientist, Schepens Eye Research Institute; Assistant Professor, Harvard Medical School

Retinal pigment epithelium (RPE) transplantation is a promising strategy for the treatment of dry age-related macular degeneration. However, previous attempts at subretinal RPE cell transplantation have experienced limited success due to poor survival on aged or diseased Bruch’s membrane. To improve the clinical outcome of RPE transplantation, we are developing biomimetic and biocompatible scaffolds able to not only support RPE survival but also significantly improve cell morphology, gene expression and function.

2:55 Stem Cell-Based Therapeutic Strategies for Understanding and Treating Ocular Surface Failure

Sajjad Ahmad, M.D., Consultant Ophthalmic Surgeon, St. Paul’s Eye Unit, Royal Liverpool University Hospital

The cornea is the clear front of the eye and its clarity is vital for visual function. The corneal surface is maintained by stem cells and these fail in the disease of limbal stem cell deficiency resulting in painful blindness. Stem cell-based cellular therapies are increasingly being used to treat patients with this debilitating disease. I have developed an animal-free culture system for expanding human limbal stem cells that has been used to successfully treat patients with limbal stem cell deficiency. In addition, using in vitro disease models, I am currently investigating the role of the limbal stem cell microenvironment in contributing to disease progression so that therapeutic strategies can be developed earlier on in the disease process.

3:25 Interim Analysis of the Safety and Preliminary Efficacy of Human Neural Stem Cells (HuCNS-SC) in Geographic Atrophy (dry AMD)

Joel Naor, M.D., M.B.A., M.Sc., Vice President, Clinical Development, Ophthalmology, StemCells, Inc.

A multi-center dose-escalation Phase I/II trial ( NCT01632527) for patients with the dry form of AMD is investigating the safety and preliminary efficacy of 200,000 and 1 million cell doses administered to the subretinal space of the study eye. Post-transplant assessments include tests of visual acuity, fluorescein angiography, spectral domain ocular coherence tomography (OCT), microperimetry, multifocal electroretinography, and contrast sensitivity. Interim results confirm the safety and tolerability of HuCNS-SC cell transplantation into the subretinal space and show slowing of disease progression and improvement in visual function / contrast sensitivity.

3:55 Decellularized Retina Matrix: Natural Substrates for Human Retinal Progenitor Cells

Joydip Kundu, Ph.D., Associate Research Scientist, Department of Chemical Engineering, Northeastern University

Development of a biomimetic platform that is permissive to hRPC (human retinal progenitor cell) survival, differentiation, and integration within the host retina to restore vision. Decellularized retina (decell-retina) mimics the environment of retina, and thus we hypothesize will promote hRPC survival, attachment, migration, differentiation and integration into the host retina. We explored the prospect of decell-retina based substrates as delivery vehicle of hRPCs to subretinal space for retinal repair.

4:25 Closing Remarks

4:30 Close of Conference

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