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Targeting Ocular Disorders - Day 2

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Friday, Sept 27, 2013

8:00-8:25am Morning Coffee


8:25 Opening Remarks

Rogier Smits, Ph.D., Managing Director, Medicinal Chemistry, Griffin Discoveries BV

8:30 P529: A First-in-Class Dual Dissociative TORC1/TORC2 Small Molecule Drug Inhibitor of the PI3K Pathway

David Sherris, Ph.D., CEO, Paloma Pharmaceuticals, Inc.

P529 has been examined in two Phase I clinical trials for age-related macular degeneration. Results from these trials indicate preliminary activity and a lack of toxicity. Of particular interest, P529 displayed a depot affect with a long resident half-life in the eye.

9:00 Bi-Specific Aptamer-Based Antagonists of VEGF and PDGF for the Treatment of Age-Related Macular Degeneration

Nebojsa Janjic, Ph.D., CSO, R&D, SomaLogic, Inc.

The distinguishing feature of SOMAmers is the presence of protein-like functional groups typically involved in protein-protein and drug-protein interactions, but absent in conventional aptamers. We have identified SOMAmers for PDGF-B with composition distinct from previously reported aptamers to PDGF-B, including Fovista. We have further shown that VEGF and PDGF SOMAmers can be readily assembled into bi-specific regents that maintain full binding affinity and potency of the individual reagents. With the recent clinical validation PDGF-B as a target for therapeutic intervention in AMD when combined with VEGF inhibitors, such bi-specific reagents represent an exciting new direction for development of novel AMD therapeutics.

9:30 Antihistimines in Ocular Allergy: The Promise of H1R/H4R Dual-Action Antagonists

Rogier Smits, Ph.D., Managing Director, Medicinal Chemistry, Griffin Discoveries BV

Histamine has been known to play an important role in ocular allergy. Up-to-date antihistamines have been developed to block only the histamine H1 receptor subtype (H1R), despite the fact that four histamine receptor subtypes have been discovered. In this presentation we will discuss the role of the newest member of this receptor family, the H4R. We will also show that histamine H1R/H4R dual-action antagonists are superior in an in vivo model of ocular allergy to the golden standard olopatadine. H1R/H4R dual-action antagonists represent a new generation of antihistamines that combine anti-allergic and anti-inflammatory action.

10:00-10:30 Coffee Break with Exhibit & Poster Viewing


10:30 Antisense Oligonucleotides: A Close Look at a Topic that Makes Sense

Eric Thorin, Ph.D., Chief Development Officer, Gene Signal International, SA

Antisense oligonucleotides are small biological molecules benefitting from simplified rational drug design, scalability and cost. Gene Signal is developing aganirsen, a topically active anti-angiogenic antisense oligonucleotide that limits the pathological proliferation of neovessels on the cornea in patients, and the pathological neovascularisation of the retina in non-human primates. The recent FDA approval of mipomersen (Isis Pharmaceuticals/ Genzyme), an antisense oligonucleotide that inhibits apolipoprotein B100, the recent demonstration of the efficacy of ISIS 333611 in patients with amyotrophic lateral sclerosis, and miravirsen's encouraging results in chronic hepatitis C virus genotype 1 infection should strengthen the antisense field of research and development.

11:00 Development of Novel Small Molecule Inhibitors Targeting Atypical PKC to Prevent Retinal Edema

David A. Antonetti, Ph.D., Department of Ophthalmology and Visual Sciences, Kellogg Eye Center, University of Michigan

Macular edema remains a leading cause of visual loss in a number of retinal diseases. Research has demonstrated atypical PKC (aPKC) acts as a common signaling molecule for vascular permeability induced by VEGF, as well as TNF, CCL2 and thrombin. Novel, specific aPKC inhibitors effectively prevent combined VEGF and TNF induced permeability in vivo and may provide an effective means to treat a wide range of retinal diseases.

11:30 Targeted Nanoparticle Therapy using Intraceptor Inhibition of Choroidal Neovascularization

Michael Burr, Ph.D., Research Scientist, Moran Eye Center, University of Utah

Here, we show that a single intravenous injection of targeted, biodegradable nanoparticles delivering a recombinant Flt23k intraceptor plasmid homes to neovascular lesions in the retina and regresses CNV in primate and murine AMD models. Moreover, this treatment suppressed subretinal fibrosis, which is currently not addressed by clinical therapies. We found no evidence of ocular or systemic toxicity from nanoparticle treatment. These findings offer a nanoparticle-based platform for targeted, vitreous-sparing, extended-release, nonviral gene therapy.

12:00 pm Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own


1:45 Chairperson's Remarks

Eric Furfine, Ph.D., President of R & D, Eleven Biotherapeutics

1:50 Lens Epithelium-Derived Growth Factor Fragment (LEDGF1-326), a Novel Therapeutic Protein: Biosynthesis, Characterization, and Efficacy in Retinal Degenerative Diseases

Uday B. Kompella, Ph.D., Director, Kompella Lab, University of Colorado Anschutz Medical Campus

We identified lens epithelium derived growth factor fragment (LEDGF1-326) as a novel protein therapeutic. We biosynthesized, purified, and characterized LEDGF1-326. Eight weeks after single intravitreal injection in Royal College of Surgeon (RCS) rats, LEDGF1-326 increased the b-wave amplitude significantly from 9.4 ± 4.6 to 57.6 ± 8.8 μV for scotopicelectroretinogram (ERG) and from 10.9 ± 5.6 to 45.8 ± 15.2 μV for photopic ERG. LEDGF1-326 significantly increased the retinal outer nuclear layer thickness from 6.34 ± 1.6 to 11.7 ± 0.7 μm. LEDGF1-326 is a potential new therapeutic agent for treating retinal degenerative diseases.

2:20 Inhibition of Cytokine Signaling to Treat Ocular Diseases

Eric Furfine, Ph.D., President of R & D, Eleven Biotherapeutics

2:50 Treatment for Dry Form AMD in a New Mouse Model

Haoyu Mao, Ph.D., Department of Molecular Genetics and Microbiology, University of Florida College of Medicine

By developing a new dry form AMD mouse model, a new treatment with a potential therapeutic reagent is investigated by up to 4 months daily treatment. Monthly monitor of ERG, OCT and fundus showed improved retinal function with treatment comparing with that of control saline group.

3:10  Concluding Remarks 

3:30  Close of Conference




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