With its complex structure and the breadth of ocular disorders, the eye presents unique challenges to drug discovery. Cambridge Healthtech Institute’s Fifth Annual Targeting Ocular Disorders conference provides a platform to discuss novel targets and disease pathways, the latest drug delivery methods, and the most promising emerging therapies for both front and back of eye disorders. A special focus will be on gene therapy, especially optogenetics, and treatments outside of the well-established anti-VEGF monotherapies. Attendees will be able to hear leading experts in ocular drug delivery discuss the pros and cons of various drug delivery methods. The event will cover a broad range of diseases including but not limited to glaucoma, wet and dry age‐related and diabetic macular degeneration, and noninfectious uveitis.

Final Agenda

Day 1 | Day 2

Wednesday, September 27

11:50 am Conference Registration Open

12:35 pm Plenary Keynote Program

(visit http://www.DiscoveryOnTarget.com/Plenary-Keynotes for complete details)

2:00 Refreshment Break in the Exhibit Hall with Poster Viewing

Gene Editing & Gene Therapy Breakthroughs for Ocular Disorders

2:45 Welcome Remarks

Lee Yuan, Conference Director, Cambridge Healthtech Institute

2:50 Chairperson’s Opening Remarks

Bo Liang, Ph.D., President, R&D, IVIEW Therapeutics, Inc.

2:55 Lentiviral Gene Therapy for Ocular Disease

Scott Ellis, Ph.D., Head, Early Development, Oxford BioMedica

Oxford BioMedica’s gene therapy for Parkinson’s disease (ProSavin®) was the first ever lentiviral gene therapy directly administered in man, and its shared LentiVector® gene therapy platform is the basis of three ocular gene therapies currently under clinical evaluation as well as several earlier-stage programs in development. This platform has consistently demonstrated an excellent safety profile and uniquely has been shown to mediate robust and durable therapeutic gene expression in the eye in patients over several years. This talk will review our previous experience and current plans using the LentiVector® platform in the development of gene therapies for chronic ocular diseases.

3:25 Intravitreal Gene Therapy for Dry AMD

Jay S. Duker, M.D., Director, New England Eye Center; Professor and Chairman, Department of Ophthalmology, Tufts Medical Center, Tufts University School of Medicine; Founder, Hemera Biosciences

Dry age related macular degeneration (AMD) represents a significant cause of visual loss in the elderly but lacks an approved therapy. Inhibiting the complement system locally within the eye shows promise as a therapeutic intervention. Hemera Biosciences’ lead product, HMR59, is an AAV2 based gene therapy delivered intravitreally that blocks membrane attack complex (MAC) through the local production of soluble CD59. HMR59 is currently being tested in a Phase I clinical trial in eyes with severe dry AMD and geographic atrophy (GA).

3:55 Development of Sustain Release Povidone Iodine Ophthalmic Drop through Novel in situ Gel Formulation

Bo Liang, Ph.D., President, R&D, IVIEW Therapeutics, Inc.

IVIEW developed a long-acting povidone iodine (PVP-I) ophthalmic drop IVIEW-1201 for the treatment of active infections of the conjunctiva and cornea by bacteria, mycobacteria, virus, fungus, or amoebic causes. There is currently no broadly effective therapy that treats all causes of infection and nothing is approved for the treatment of viral conjunctivitis. This represents a massive unmet need in ophthalmology. The novel in-situ gel formulation IVIEW-1201 where the effective concentration of PVP-I is maintained by the equilibrium between solution PVP-I and the gel bound components results in a long lasting, less toxic pharmacological effect.

4:25 Refreshment Break in the Exhibit Hall with Poster Viewing

5:00 Development of an Intravitreal AAV-Based Treatment for Wet Age-Related Macular Degeneration

Mehdi Gasmi, Ph.D., CTO & CSO, Adverum Biotechnologies

5:30 Optogenetic Therapy for Retinal Dystrophies

Anne Douar, Ph.D., Project Director, GenSight Biologics

Optogenetics aims at transferring a gene encoding for a light-sensitive molecule to restore photosensitivity in retinal cells that are still wired into the inner retina layers. The GS030 treatment combines such gene therapy based approach in combination with a photo-stimulating device to potentiate the biologics’ activity. Translational research has allowed to demonstrate the proof of concept in rodent and non-human primate models and to establish the safety profile of the product, paving the way to the first in human clinical trial currently in preparation.

6:00 A CRISPR Medicine Approach for Treating Leber Congenital Amaurosis Type 10

Gerald F. Cox, M.D., Ph.D., CMO, Editas Medicine

Leber congenital amaurosis type 10 (LCA10) is a rare infantile-onset retinal dystrophy caused by autosomal recessive mutations in the CEP290 gene. In photoreceptors, CEP290 is required for cilia formation, maintenance of outer segments, and cell viability. The most common LCA10 mutation, c.2991+1655A>G, creates a cryptic splice acceptor site in intron 26 that causes missplicing and leads to a non-functional protein. CRISPR/Cas9 is a genome editing tool that cuts and modifies DNA at precise locations. This new technology is being applied to correct the underlying common genetic defect in LCA10, with the goal of providing a durable treatment that restores vision to patients.

6:30 Close of Day

Day 1 | Day 2

Thursday, September 28

7:30 am Registration Open

Gene Editing & Gene Therapy Breakthroughs for Ocular Disorders

9:00 Chairperson’s Remarks

Naj Sharif, Ph.D., FARVO, FBPhS, Executive Director; Head, Global Alliances & External Research, Santen, Inc.

9:05 Investigational Gene Therapy for Inherited Retinal Dystrophies Due to Biallelic Mutations in RPE65

Daniel Chung, D.O., Clinical Ophthalmic Lead, Sparks Therapeutics

This presentation is a review of the latest results from a Phase III, open-label, randomized, controlled trial evaluating the safety and efficacy of AAV2-hRPE65v2 (SPK-RPE65) to treat inherited retinal dystrophies caused by biallelic mutations in the RPE65 gene, following adeno-associated virus mediated gene transfer to the retina.

Novel Targets & Disease Pathways

9:35 Novel Glaucoma Drugs and Devices: Paving Paths towards Retinoprotection

Naj Sharif, Ph.D., FARVO, FBPhS, Executive Director; Head, Global Alliances & External Research, Santen, Inc.

Whilst prostaglandin FP-receptor agonists are mainstay current therapeutics used to treat ocular hypertension (OHT) associated with primary open-angle glaucoma (POAG), a number of novel new drugs are on the horizon awaiting FDA approvals. There has also been a recent revolution in the development and clinical utility of minimally-invasive-glaucoma-surgery (MIGS) devices to lower intraocular pressure (IOP) for managing OHT/POAG. However, since reduction of IOP does not always reduce or prevent vision loss from POAG, the holy-grail of neuroprotective therapy, alone or in conjunction with IOP lowering, still represents an achievable goal. These aspects will be addressed in this presentation.

10:05 Plasma Kallikrein Inhibitors for the Treatment of Diabetic Retinopathy/Diabetic Macular Edema

Timothy Shiau, Ph.D., Senior Scientist, Chemistry, Verseon

Topically-applied plasma kallikrein inhibitors are a promising mechanism of slowing, stopping, or reversing diabetic macular edema. We present a series of small molecule, nanomolar inhibitors of plasma kallikrein and progress toward in vivo efficacy in a diabetic retinopathy model following topical dosing.

10:35 Coffee Break in the Exhibit Hall with Poster Viewing and Poster Competition Winner Announced

11:20 Plasma Kallikrein Inhibition as a VEGF-Independent Treatment for Diabetic Macular Edema

Edward P. Feener, Ph.D., Co-Founder and CSO, KalVista Pharmaceuticals

This talk will present the scientific and clinical rationale for targeting plasma kallikrein as a novel VEGF-independent treatment for diabetic macular edema (DME). Increased plasma kallikrein levels have been identified in vitreous from DME patients. Preclinical studies have demonstrated that plasma kallikrein induces retinal edema via a VEGF-independent mechanism. KalVista has discovered and developed an intravitreally administered plasma kallikrein inhibitor, KVD001. The Company has successfully completed its first-in-human study in patients with DME and is preparing for Phase II studies.

11:50 Late Breaking Presentation

12:20 pm Sponsored Presentation (Opportunity Available)

12:50 Session Break

1:00 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:50 Refreshment Break in the Exhibit Hall with Poster Viewing

2:35 Chairperson’s Remarks

Sharon Klier, Vice President, Ophthalmology, Medical, Quark Pharmaceuticals

2:40 Novel Injectable Products for Treatment of Ocular Diseases

Ming Yang, Ph.D., Director, Research, Graybug Vision

Graybug Vision is developing novel products for the treatment of people with ocular diseases. The company’s proprietary injectable products are designed to enable less frequent administration and to reduce the burden of treatment for patients with ocular diseases. The company’s lead product, GB-102, consists of an approved receptor tyrosine kinase inhibitor, sunitinib malate, that inhibits multiple pathogenic angiogenesis pathways known to be involved in the choroidal neovascularization, the cause of neovascular AMD (nAMD). Graybug Vision has incorporated sunitinib in its novel delivery system to allow a potential twice per year injection, dramatically reducing the current burden of treatment for patients and physicians. Graybug Vision has also developed a library of compounds to treat glaucoma, a leading cause of progressive, irreversible vision loss worldwide, by lowering intraocular pressure alone or in combination with neuroprotection when injected twice per year into the subconjunctiva.

3:10 Sustained Micro-Dose Drug Delivery with Injectable Inserts: Current Status and Emerging Applications

Dario A. Paggiarino, M.D., Vice President, CMO, pSivida

Sustained linear-release, micro-dose delivery of antiviral and anti-inflammatory agents has evolved over the years from surgically implanted to injectable miniaturized inserts. The same technology showing long-term clinical benefit in past approved products is now being developed in the delivery of new agents and ocular conditions for which micro-dosing would be key in providing extended control of disease pathophysiology.

3:40 Session Break

3:55 Intracanalicular Inserts for Drug Delivery – Applications in Managing Ophthalmic Diseases

Amar Sawhney, Ph.D., Executive Chairman, Ocular Therapeutix

Intracanalicular inserts may prove to be a promising modality for non-invasive placement of drug products to deliver courses of therapy for several ophthalmic diseases in the anterior segment or on the ocular surface. Dextenza is being developed to deliver an entire 30-day course of preservative-free steroid therapy for pain and inflammation following ocular surgery with a single insertion. Additionally, Ocular Therapeutix is evaluating OTX-TP (travoprost insert) for the treatment of glaucoma and ocular hypertension that could provide therapy for up to 3 months. This talk will explore the design, development, and clinical data surrounding these drug product candidates and other sustained release product candidates in the Ocular Therapeutix pipeline.

4:25 PANEL DISCUSSION: Pros and Cons of Ocular Drug Delivery Methods

Moderator:
Elias Reichel, M.D., Professor and Vice Chair, Tufts University School of Medicine; Director, Vitreoretinal Diseases and Surgery Service, New England Eye Center; Founder, Hemera Biosciences

Panelists:

Ming Yang, Ph.D., Director, Research, Graybug Vision

Amar Sawhney, Ph.D., Executive Chairman, Ocular Therapeutix

Dario A. Paggiarino, M.D., Vice President, CMO, pSivida

The panelists will discuss key considerations when determining the right ocular drug delivery method for their products and challenges that they faced. There will also be general discussion about the pros and cons of different delivery methods for the eye.

4:55 Close of Conference

Day 1 | Day 2