8:50 am Chair’s Opening Remarks

DEVELOPING AN EFFECTIVE CLINICAL TRIAL TO SATISFY REGULATORY BODIES

9:00 am Understanding the FDA Regulatory Perspective on Gene Therapies for Ophthalmic Disorders

Synopsis

  • Discussing the current FDA guidelines released on gene therapies for retinal disorders
  • Understanding the FDA perspective and how this is expected to evolve in the near future
  • Highlighting key milestones to reach from a regulatory perspective and the requirements needed to proceed to the next stage of the pipeline

9:30 am Creating a Bespoke Endpoint That is Meaningful to the Patient

Synopsis

• Beyond visual acuity: developing endpoints that are meaningful to patients and regulatory bodies
• Measuring functional vision vs. visual function
• The role of patient-reported outcome measures
• Designing and validating novel endpoints

10:00 am Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy (LHON): Key Learnings from a Clinical Development Program

Synopsis

• Understanding how to link together clinical trial design and regulatory requirements
• Exploring pathways to fulfil both EMA and FDA requirements
• Developing a clinical trial for LHON: key takeaways to learn from

10:30 am Morning Break

OPTIMIZING CLINICAL TRIAL DESIGN & IMPLEMENTATION

11:15 am MonCvONE: Visual Function Evaluation for Clinical Research

Synopsis

  • Functional versatility – One instrument does it all
  • Vision psychophysics, eye tracking and vision electrophysiology
  • Full visual field and photometric range
  • Comparison of function to structure

11:30 am Determining the Right Site for Your Gene Therapy Trial

Synopsis

• Understanding unique needs of the trial and cross-checking against potential trial sites
• Exploring relevant importance of both ophthalmic expertise and gene therapy expertise
• Investigating scaling capabilities of different sites
• Discussing collaboration between the sponsor, site, and investigator

12:00 pm Utilizing Natural History Studies to Inform Clinical Trial Design

  • Todd Durham Vice President, Clinical & Outcomes Research, Foundation Fighting Blindness

Synopsis

• Creating a consortium of retinal specialists to collect and analyze vital natural history information
• Highlighting the value of better understanding of disease progression to trial design
• Providing a practical context to trial design

12:30 pm PANEL DISCUSSION: Evaluating the Current Commercial Landscape in Light of a Recent Ophthalmic Approval

Synopsis

Exciting work is being done to develop gene therapies to treat a variety of ophthalmic disorders, and there is already a retinal gene therapy on the market. This leads to key questions surrounding reimbursement, access to therapies, and effective commercialization and product differentiation of gene therapies to come. This panel discussion will address various topics surrounding the postapproval
landscape, including:
• Durability of gene therapies in the eye and relative impacts on pricing plans
• Differentiation of products which are treating the same indication
• Enabling patient access to products and the unique factors at play depending on the rarity of the disease
• Impacts of Luxturna serving as a benchmark for future approvals in the ophthalmic space

1:00 pm Lunch Break

2:00 pm Exploring Innovative Partnerships to Enable Gene Therapy Manufacturing for Rare Ocular Disorders

Synopsis

• Investigating current manufacturing challenges and leveraging unique opportunities in gene therapy development for rare ocular disorders
• Developing innovative partnerships with academia and industry to decrease costs, de-risk gene therapies, and overcome obstacles in development

EXPLORING IMMUNE RESPONSE IN THE EYE

2:30 pm Deep Dive: Evaluating Immunogenicity in the Eye: Best Approaches & Practices

Synopsis

There has been much debate over the relative ‘immunoprivilege’ of the eye compared to other organs being targeted by gene therapy. It’s clear from a regulatory perspective that immune response triggered by administration of gene therapies is a key consideration, and so working to better predict, monitor and mitigate immune response in the eye will be key to developing a successful product.

Join this comprehensive session, delivered as a mixture of presentations and panel discussion Q&As, to gain a better understanding of the challenge of immune response in the eye and how to overcome it.

Nagendra Chemuturi, Senior Investigator, Novartis

3:30 pm Afternoon Break

DISCOVERING THE NEXT GENERATION OF GENE THERAPIES TARGETING NOVEL DISEASES & CELL TYPES

4:00 pm Intravitreal HMR59 (AAVCAGsCD59) for the treatment of Age-related Macular Degeneration (AMD)

  • Adam Rogers Founder & Chief Executive Officer, Hemera Biosciences

Synopsis

  •  HMR59 is an AAV2 gene therapy administered as intravitreal injection in an office setting
  • Soluble CD59 (sCD59) is the transgene product and blocks complement at membrane attack complex (MAC)
  • Two phase 1 trials have enrolled 42 subjects with doses up to 1.071x1012vg
  • HMR-1001 evaluated 17 subjects with advanced dry AMD with 24 months follow-up
  • HMR-1002 evaluated 25 eyes treated with new onset wet AMD treated with anti-VEGF at Day 0 and HMR59 at Day 7. Eyes were retreated with anti- VEGF based on prespecified criteria over 12 months. With a mean 10.1 months follow-up
  • Hemera is starting a phase 2 dry AMD clinical trial at 25 U.S. sites

4:30 pm Exploring Next-Generation Therapeutics: The Promise of Gene Editing

  • Qin Liu Assistant Professor of Ophthalmology, Harvard Medical School

Synopsis

• Exploring the use of various gene editing technologies to treat inherited eye diseases
• Investigating the unique challenges associated with gene editing compounded with challenges of gene therapy
• Discussing recent data highlighting innovations of gene edited gene therapies to treat ophthalmic disorders

5:00 pm Chair’s Closing Remarks

5:10 pm Close of Conference