8:00 am Registration

8:45 am Chair’s Opening Remarks

  • Chad Jackson Director, Preclinical Translational Research Program, Foundation Fighting Blindness

Developing Clinically Validated Novel Candidate Endpoints for Dry AMD, iAMD & GA

9:00 am Targeting Lipids in Early & Intermediate AMD by the Oil Spill Strategies

  • Christine Curcio Professor, Ophthalmology & Director, Age-Related Maculopathy Histopathology Lab, University of Alabama at Birmingham

9:30 am Spotlight on The Prima Bionic Vision System: Improving Visual Acuity in Patients with Advanced Dry AMD

  • Ralf Hornig Director of Clinical Affairs , Pixum Vision


  • Take a look at the work Pixium Vision, a bioelectronics company specialized in neuromodulation are doing to create a world of bionic vision for those who have lost their sight
  • Understand PRIMA a brain machine interface with a retinal implant which helps patient suffering from dry AMD regain artificial visual perceptions
  • Understand how entering in the pivotal study, and demonstrating promising clinical data, shows how the Prima device allows simultaneous Perception of Prosthetic and Natural Vision
  • Explore patients demonstrating Landolt acuity of 1.17 ± 0.13 pixels and with electronic magnification of up to a factor of 8, demonstrate acuity in the range of 20/63 − 20/98

10:00 am Defining Visual Impairment and Clinically Relevant Endpoints in Intermediate AMD: The MACUSTAR Project

  • Sergio Leal Director, Global Clinical Leader Ophthalmology, Bayer


  • Identify hurdles for clinical development in intermediate AMD
  • Learn about MACUSTAR, an IMI (Innovative Medicines Initiative) project aiming to characterize visual functional deficits, structural changes and longitudinal progression in early and intermediate AMD
  • Discuss the industry perspective on the MACUSTAR project and its potential to support regulatory evaluations and patient access in intermediate AMD

10:30 am Virtual Speed Networking


Grab a coffee from the comfort of your own kitchen and jump into this 1:1 networking session to make new connections, exchange virtual business cards and maybe even see a friendly face or two!

Preclinical & Translational Development

De-risking Future Drug Candidates: Eliminating Obstacles with Preclinical Animal & In-Vitro Models

11:00 am Overcome Challenges in Developing In-Vivo Models for Dry-AMD


  • Explore how the development of dry-AMD therapy is hampered by a lack of translatable animal models
  • Since AMD involves the macula, discuss how one major challenge of relying on animal models is lacking in this feature
  • Examine how relevant aspects of human disease can be recapitulated in an animal model and may provide a path forward

11:30 am Discuss a Novel Animal Model of GA that is Translatable to Human Disease


  • Hear a brief background of the model as delivered using custom optics
  • Explore structural, functional, and behavioral/psychophysics data as compared with early-stage AMD patients
  • Examine the advantages of this approach to evaluate potential therapeutics to treat AMD/GA

Reduce Toxic By-Product Accumulation: Reduce DHA peroxidation

12:00 pm Breaking the Vicious Circle of AMD Progression with Oxidation-Resistant DHA


  • Explore how retinal OSE proliferation can turn the idling alternative complement pathway into a pathologic response as demonstrated by the impaired ability of the Factor H AMD risk variant CFH 402H to neutralize retinal OSEs
  • Examine a custom-designed allylic deuteriumreinforced analogue of DHA (D-DHA) which represents a novel approach to preventing retinal LPO and its downstream effects on AMD progression
  • Discuss the specific advantages of D-DHA for treating dry AMD (oral delivery, preclinical safety, target engagement and pleiotropic mechanism of action)

Clinical Development

Photoreceptor & RPE Loss: Stem Cell-Based Therapies Learnings & Updates from Clinical Case Studies

11:00 am A Further Spotlight on OpRegen – A RPE Transplant for Dry AMD with GA

  • Brian Culley Chief Executive Officer, Lineage Cell Therapeutics


  • Reiterative how a transplant of RPE cells can be both robust and endured by patients with dry-AMD
  • Understand further the benefits of OpRegen RPE cells in specific patients with dry-AMD, as well as primary indication of RPE-photoreceptor complex regeneration (GA reduction)
  • Evaluate how RPE cell suspensions can be reasonably scaled to marketable quantities

11:30 am Spotlight on Phase 1/2a Clinical Trial of a Bio-Engineered Implant for GA


  • Analyse the design of implant as a tissue replacement therapy
  • Understand the delivery of the implant to the subretinal space
  • Take a look at phase 1/2a clinical trial design and results
  • Examine the next steps in clinical development

Visual Cycle Modulation

12:00 pm Targeting the Visual Cycle Through Vitamin A Modification


  • Dry AMD treatment with minerals, antioxidants, and other natural compounds.
  • Current challenges and therapeutic approaches for the treatment of
  • Geographic Atrophy: drug delivery, animal models, study designs
  • Visual cycle: to slow or not to slow? Use of ALK-001 as vitamin A replacement therapy for the treatment of dryAMD or Geographic Atrophy
  • Leonide Saad, President & Chief

12:30 pm Networking Lunch

Examine Novel Approaches: Inflammasome Inhibition & HtrA1 Inhibitor

1:30 pm On the Horizon – HtrA1 Inhibition in the Treatment of GA


  • Strong human genetic evidence associates ocular HtrA1 overexpression with geographic atrophy and all neovascular forms of AMD
  • Growing understanding of how HtrA1 substrates and products play important roles in the pathogenesis of AMD
  • Compelling preclinical and clinical evidence for role of HtrA1 in AMD
  • HtrA1 pathway is non-overlapping with VEGF and is independent of complement risk factors

2:00 pm Spotlight on GR39821- Phase 1 Single & Multiple-Dose Safety Study of Anti-High Temperature Requirement A1 (HtrA1) in GA


  • Therapeutic Hypothesis for Targeting HtrA1
  • Review of GR39821 Phase 1 open-label, multicenter, single-and multiple-dose study of anti-HtrA1 in patients with GA secondary to AMD
  • Review ocular specific enzyme activity-based antiHtra1 PD assay

2:30 pm How do Inflammasomes, Key Molecular Regulators, Play a Vital Role in Dry AMD?

  • Paul Ashton President & CEO, Inflammasome Therapeutics


  • Examine inflammasome activators in dry-AMD
  • Explore inflammasome activation inhibitors in pre-clinical models
  • Evaluate novel, recent clinical data
  • Discuss the therapeutic strategy for inflammasome inhibition in dry-AMD

Examining the Growing Need for Validated Biomarkers & Endpoints for Dry-AMD Clinical Research & Trials

1:30 pm Optimize Novel Imaging Biomarkers & Endpoints for Dry AMD Trials


  • Review current FAF and OCT biomarkers for AMD progression
  • Discuss endpoints for assessing efficacy in current GA trials
  • Explore novel OCT-A biomarkers for use in clinical research and clinical trials

2:00 pm Enhance Novel Clinical Trial Endpoints for Trials in GA


  •  Examine Structural versus Functional endpoints in GA Trials
  • Review current endpoints for registration
  • Explore the role of microperimetry as an emerging endpoint
  • Analyze novel endpoints for clinical trials in GA
  • Discuss the use of artificial intelligence in GA endpoints of the future

2:30 pm Opportunities for Personalized Medicine – Automated Risk Classification for Progression in AMD & Utilizing Machine Learning Enhanced OCT Feature Extraction

  • Justis Ehlers Associate Professor of Ophthalmology, Cleveland Clinic Lerner College of Medicine


  • Explore OCT-based biomarkers linked to AMD progression to subfoveal geographic atrophy
  • Discuss the potential automated solutions for image interrogation and characterization for quantification of these biomarkers
  • Examine the potential risk classification system using these imaging biomarkers for clinical trial enrichment, treatment decision-making, and prognostication

3:00 pm Afternoon Networking Break

3:30 pm Panel Discussion & Presentation: The Future Landscape of Dry-AMD Therapies

  • Jeffrey Heier Director, Retina Service Director, Ophthalmic Consultants of Boston (OCB)
  • Baruch Kuppermann Director, Co-Director, Professor & Chair - Ophthalmology, UC Irvine
  • Dhaval Desai Senior Vice-President & Chief Development Officer, IVERIC Bio
  • Peter Kaiser Professor of Ophthalmology, Cleveland Clinic Lerner College of Medicine
  • SriniVas Sadda Professor of Ophthalmology, UCLA
  • Sobha Sivaprasad Professor & Consultant , Ophthalmologist Moorfields Eye Hospital
  • Jeff Willis Senior Medical Director, Genentech


  • Further analysis of the treatment landscape for Dry-AMD/GA – What’s on the horizon?
  • Examine patient treatment journey with the emerging new treatment landscape
  • Examine how clinicians/developers can support and deal with treatment burden
  • Explore clinical trial development and the continued impact of the pandemic on on-going clinical trials

4:30 pm Chair’s Closing Remarks

  • Chad Jackson Director, Preclinical Translational Research Program, Foundation Fighting Blindness

4:45 pm End of Summit