2016 Archived Content

With its complex structure and the breadth of ocular disorders, the eye presents unique challenges to drug discovery. Cambridge Healthtech Institute’s fourth 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. A special focus will be on gene therapy, stem cell therapies, and treatments outside of the well-established anti-VEGF therapies. The event will cover a broad range of diseases including but not limited to glaucoma, wet and dry age‐related and diabetic macular degeneration, retinopathy and retinitis pigmentosa.

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September 20-21 Conference: Targeting Ocular Disorders

September 21-22 Conference: Advances in Gene Editing and Gene Silencing - Part 2

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Tuesday, September 20

7:00 am Registration Open and Morning Coffee


8:05 Chairperson’s Opening Remarks

Jason Slakter, CEO, Ohr Pharmaceutical, Inc.

8:20 Translating Genomics and Stem Cell Technology into Therapy for Major Blinding Diseases

Kang ZhangKang Zhang, M.D., Ph.D., Professor of Ophthalmology, Chief of Ophthalmic Genetics at University of California San Diego

Stem cell research shows great promise in modeling disease in vitro, and for treating blinding degenerative diseases of the eye, including age-related macular degeneration (AMD) and glaucoma. Limbal stem cell (LSC) deficiency causes corneal surface disease, which leads to blindness in millions of people world-wide. We established an in vitro feeder-free LSC expansion/3-D corneal differentiation system and show transplantation of LSCs in animal models and humans can replenish the LSC pool and restore corneal transparency. We have also shown that lanosterol is essential for prevention of protein aggregation and can reverse cataract. We also demonstrated regeneration of a functional human lens using endogenous stem cells, with gain of visual function, in human infants with cataract. Furthermore, epigenetics contributes to the progression of aging and age related diseases. The recent advances in genetic and stem cell therapies of the eye will allow for identification of the high risk patients for personalized intervention and treatment in the near future.

8:50 Palucorcel for the Treatment of Geographic Atrophy in AMD

James BaldassarreJames S. Baldassarre, M.D., Head, Clinical Development, Janssen

Human umbilical tissue cells (hUTC; palucorcel) produce a number of trophic factors which may favorably influence various retinal cell types in AMD and have the potential to reverse visual impairment. They appear to be well tolerated when confined to the subretinal space, but accurate delivery has been a technical challenge. This talk will discuss the potential of palucorcel in GA, and the evolution of a novel subretinal delivery approach.

9:20 Stem Cell-Derived Retinal Pigment Epithelium for Treatment of Retinal Degeneration

Irina Klimanskaya, Ph.D., Senior Director of Translational Development, Astellas Institute for Regenerative Medicine (AIRM)

Health and efficiency of photoreceptor cells depend on Retinal Pigment Epithelium (RPE) which plays an essential role in photoreceptor maintenance. RPE malfunction can result in photoreceptor loss and subsequent blindness. RPE generated from human embryonic stem cells is the first differentiated derivative of pluripotent cells that entered clinical trials for age-related macular degeneration and Stargardt’s disease. Development of this cell replacement therapy will be discussed.

9:50 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing


10:35 AAV2 Rep-1 Gene Therapy in the Treatment of Choroideremia

David FellowsDavid Fellows, CEO, NightstaRx

Choroideremia is an X linked recessive retinal disease that leads to blindness. Nightstar’s lead program AAV2 Rep-1, administered through sub-retinal injection has been evaluated in a series of phase I/II trials in the treatment of choroideremia. The current status of the AAV2 Rep-1 program will be discussed.

11:05 Novel Optogenetic Approach to Vision Restoration in Retinal Degenerative Conditions

Sean AinsworthSean Ainsworth, CEO, RetroSense

RetroSense is developing optogenetic approaches to partial vision restoration in retinitis pigmentosa and potentially dry age-related macular degeneration. The company’s lead product, RST-001, is currently in Phase I/II clinical studies. Optogenetics refers to genetic delivery of optically-sensitive proteins, i.e., opsins. RetroSense is applying the approach to retinal neurons, which remain intact after photoreceptor degeneration, enabling them to directly respond to light stimuli and send a visual signal to the brain. The safety and efficacy of the approach has been well established in several animal models. RetroSense has secured exclusive intellectual property rights from Wayne State University and Massachusetts General Hospital covering the use of a broad range of opsins in vision restoration. These patents, combined with a portfolio of complementary intellectual property make RetroSense the world leader in optogenetics for vision restoration.

11:35 Novel Gene Therapy to Treat Diabetic Retinopathy and Wet Macular Degeneration

R Michael BurrR. Michael Burr, MS, MBA, Vice President, Product Development, iVeena, Inc.

iVeena has three gene therapy pipeline products in preclinical development. Flt23k nanoparticles for intravenous delivery and AAV2-Flt23k for sub-retinal delivery regress CNV in mice and monkeys. Comp-ANG1 is a soluble construct of angiopoetin-1 targeting the tie-2 receptor that prevents vision loss and restores vascular function in diabetic mice. iVeena intends to do first-in-human work with these products in 2017.

PharmOptima12:05 pm Ligand Binding Assay Development and Troubleshooting

Phil Zaworski, MS, Senior Research Scientist, Biochemistry, PharmOptima LLC

Novel protein therapeutics for treating ocular disorders require the development of ligand binding assays to monitor drug concentrations in biological matrices. Methods of quantitation for these therapeutics include both functional assays and standard sandwich immunoassays. Assays capable of differentiating between free and bound drug will be described.

Celanese12:20 Driving Ophthalmic Drug Delivery Innovation with EVA

Kelsey Achenbach, Strategic Marketing Manager, Healthcare, Celanese

12:35 Session Break

12:45 Luncheon Presentation (Sponsorship Opportunity Available) or Lunch on Your Own

1:25 Refreshment Break in the Exhibit Hall with Poster Viewing


2:05 Chairperson’s Remarks

Ken Mandell, M.D., Ph.D., President and CEO, LayerBio, Inc.

2:15 Gene Therapy for Retinal Diseases

Abraham ScariaAbraham Scaria, Ph.D., Senior Scientific Director, Ophthalmology, Genzyme, a Sanofi Company

Over the past two decades several studies have demonstrated the utility of Adeno associated viral (AAV) vectors for gene delivery to the retina based on the non-pathogenic nature of AAV and due to its ability to transduce a variety of different cell types in the retina. Different routes of AAV delivery to retina and the use of different AAV serotypes lead to transduction of different cell types. Results will be presented from studies aimed at developing gene therapies for wet-AMD and LCA-1.


2:45 Novel Drug Delivery Platform and Polymer for Treating Ocular Diseases

Barbara WirostkoBarbara Wirostko, M.D., CMO, EyeGate Pharmaceuticals, Inc.

EyeGate is a late-stage specialty pharmaceutical and drug delivery company focused on developing and commercializing therapeutics and drug delivery mechanisms for treating diseases of the eye. Our technologies address two of the most prevalent issues in ophthalmic care; the existing lack of patient adherence and patient safety. We are committed to advancing our delivery technology and therapeutic candidates with a goal of bringing new, potentially superior treatment options to patients who will most likely benefit. EyeGate recently acquired Jade Therapeutics developing a novel crosslinked Hyaluronic acid polymer platform. HA has a long history of being an ocular lubricant, capable of wound healing systemically, provides an extracellular matrix for stem cell delivery and regenerative medicine, and can deliver drugs in a sustained release manner.

Oculis3:15 Nanoparticle Eye-Drops Deliver Drugs to Retina in Human Patients

Stefansson_EinarEinar Stefansson, M.D., Ph.D., FARVO, Chairman, Co-Founder, Oculis

Oculis’ solubilizing nanoparticle (SNP) eye drops deliver drugs to retina in animals and humans. Four clinical trials have shown effect in DME and posterior uveitis. Various SNP eye drops have been successfully tested for anterior and posterior segment use.

Rxi Pharmaceuticals3:30 Self-Delivering RNAi Compounds (sd-rxRNA™) for the Treatment of Ocular Disorders

 Byrne_MichaelMichael Byrne, Ph.D., Director, Pharmacology, RXi Pharmaceuticals

RXi developed novel self-delivering RNAi compounds (sd-rxRNA®) with drug-like properties. RXI-109, is in a Phase 1/2 clinical trial for the treatment of subretinal fibrosis in patients with late-stage AMD. An overview of sd-rxRNA delivery and efficacy in the retina and cornea will demonstrate sd-rxRNA’s utility for treatment of ocular disorders.

3:45 Refreshment Break in the Exhibit Hall with Poster Viewing and Poster Competition Winner Announced

4:25 Current Trends in Ocular Drug Delivery

Ken MandellKen Mandell, M.D., Ph.D., President and CEO, LayerBio, Inc.

An overview of current approaches to ocular drug delivery will be presented with examples from various ophthalmic indications, including dry eye syndrome, uveitis, postoperative inflammation, glaucoma and retinal diseases.


Justin HanesJustin Hanes, Ph.D., Lewis J. Ort Professor; Director, The Center for Nanomedicine at the Wilmer Eye Institute, Johns Hopkins University School of Medicine; Founder, Kala Pharmaceuticals and GrayBug Vision

Vision loss due to ocular disease is prevalent and devastating to those affected and their families. The worldwide economic burden is also enormous. This talk will provide an overview of two technologies that are being commercialized as methods to provide more effective drug delivery to the eye: (1) mucus-penetrating particles (MPP) composed of nearly pure drug particles suspended in topical eye drops; MPP are currently being evaluated in Phase II and III clinical trials for treatment of various diseases that affect the front or back of the eye, and (2) non-inflammatory injectable polymer microparticles that provide effective and long-lasting treatments for major eye diseases, including age-related macular degeneration and glaucoma.

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

6:25 End of Day

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Wednesday, September 21

7:30 am Registration Open and Morning Coffee


8:00 Chairperson’s Opening Remarks

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

8:10 Intraocular Implant Technology Providing Sustained Delivery of a Therapeutic Protein to Treat Back of the Eye Disorders

Konrad KauperKonrad Kauper, MSc, Vice President, Core Technology Development, Neurotech Pharmaceuticals, Inc.

Treating diseases of the back of the eye remains a challenge due to not only a lack of existing therapies, but also due to the lack of localized, sustained delivery options. Take for example ciliary neurotrophic factor (CNTF), a member of the IL-6 cytokine family. CNTF has been shown to promote both photoreceptor and retinal ganglion cell survival in laboratory studies and in multiple animal models of disease. However, practical delivery of CNTF to the eye has been a significant challenge due to both the extremely short half-life of this drug as well as the eye’s blood-retina barrier. To address the need for sustained, long-term and localized drug delivery, Encapsulated Cell Therapy (ECT) has been developed. ECT is an intraocular implant utilizing a proprietary, immortalized, human, non-tumorigenic retinal pigment epithelial cell line, genetically engineered to constitutively produce therapeutic proteins for a minimum of two years or longer. This presentation will review the manufacturing, regulatory and pre-clinical development of ECT delivery of CNTF and discuss preliminary data from two completed Neurotech clinical trials. Data from both macular telangiectasias and glaucoma clinical trials suggest that sustained, intraocular delivery of CNTF may arrest retinal disease progression and prevent vision loss in both diseases, providing a novel therapy for patients currently without effective treatment options.


8:40 Innovation Theory Applied to AMD Therapy Development: The Innovators' DNA, Dilemma, and Prescription

Thomas CiullaThomas A. Ciulla, M.D., MBA, Vice President, Clinical Strategy, Ophthotech

This talk reviews innovation therapy at a personal level, technology level, and health care system level. It reviews the sweeping innovations in AMD therapies over the past two decades and identifies trends that are predicted by innovation theory. It reviews the discovery skills of an innovator, contrasting them with the delivery skills of an executive. It contrasts the differences between sustaining innovation and disruptive innovation, with particular emphasis on AMD therapy development. Finally, it reviews innovation theory as it applies to the health care system as a whole, with emphasis on business model innovation and value networks. It discusses these concepts as they apply to the delivery of AMD therapies.

9:10 Squalamine Lactate Ophthalmic Solution: A Topical Approach to Combination Therapy for AMD

Jason SlakterJason Slakter, CEO, Ohr Pharmaceutical, Inc.

Squalamine lactate is a small molecule multi-target anti-angiogenic agent that inhibits activity of vascular endothelial growth factor (VEGF), platelet-derived growth factor (PDGF), and basic fibroblast growth factor (bFGF). Unlike anti-VEGF agents that require intravitreal delivery due to their extracellular activity against their target molecule, squalamine lactate’s unique intracellular site of action permits a topical, non-invasive delivery approach. Squalamine lactate ophthalmic solution, 0.2% used in combination with ranibizumab demonstrated greater visual acuity benefits (mean change in visual acuity and proportion of patients gaining 3 or more lines in vision) over ranibizumab anti-VEGF monotherapy in patients with treatment naïve neovascularization due to age-related macular degeneration (AMD) in an exploratory Phase II clinical trial. A global Phase III registration program, evaluating the efficacy and safety of squalamine administered topically twice a day in combination with ranibizumab versus ranibizumab monotherapy has been initiated. The studies will enroll a total of approximately 1,300 patients with treatment naïve, neovascular AMD with lesion characteristics that demonstrated the strongest predictive value of treatment benefit with the combination of squalamine and ranibizumab in the phase II study.

9:40 Coffee Break in the Exhibit Hall with Poster Viewing

10:25 Treating Diabetic Eye Disease via Activation of Tie2 by AKB-9778: Results of a Phase II Clinical Study

Mitchell BrigellMitchell Brigell, Ph.D., Vice President, Clinical Development, Aerpio Therapeutics

AKB-9778 is a small molecule inhibitor of vascular endothelial protein tyrosine phosphatase (VE-PTP) that stimulates phosphorylation of Tie2 in vascular endothelial cells. Activation of Tie2 is necessary for vascular integrity, and is reduced by angiopoietin2 (Ang2) in disease states such as diabetic macular edema (DME) and diabetic retinopathy (DR). In this talk I will summarize the DME and DR results of TIME-2, a phase IIa study in 144 patients with DME randomized to receive 3 months of treatment with AKB-9778 monotherapy, ranibizumab monotherapy, or combination AKB-9778 + ranibizumab therapy. Advantages of VE-PTP inhibition over inhibition of Ang-2 will be discussed.

10:55 Single Domain Antibodies Against CXCR4 as a Potential Therapy in Retinal Vascular Disease

Mick FoleyMick Foley, Ph.D., CSO, Biochemistry, AdAlta, Australia

The chemokine receptor CXCR4 has been implicated in a number of ocular diseases including aberrant choroidal neovascularization and it has been suggested that antagonizing this receptor may be a promising therapy for ocular diseases. We have identified a human single domain “i-body” against CXCR4 and shown that upon intravitreal administration, this antagonist can reduce retinal scarring and tissue contraction in a laser induced mouse model of choroidal neovascularization. This is consistent with a potential effect on fibrosis and scarring that occurs following laser damage. The mechanism of action of this novel approach will be discussed.

11:25 Enjoy Lunch on Your Own

2:40 Refreshment Break in the Exhibit Hall with Poster Viewing

3:20 Close of Conference

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