REVOLUTIONIZING GENE THERAPY ADMINISTRATION FOR OPHTHALMIC DISORDERS
8:00 am Chair’s Opening Remarks
8:10 am Rare Vs Common Disorders: Structural & Functional Endpoints for Clinical Trials in AMD & Diabetic Retinopathy
Synopsis
• Evaluating functional endpoints for clinical trials
• Highlighting regulatory considerations in endpoint selection
• Exploring novel and emerging endpoints
8:30 am Evaluating the Promise of Suprachoroidal Delivery for Retinal Expression
Synopsis
• Benefits of suprachoroidal gene therapy administration: avoids risks of surgery, favorable posterior segment distribution and office based to enhance access to care
• Exploring AAV preclinical and clinical studies with suprachoroidal delivery
• Discussing the immune response profile associated with suprachoroidal delivery
8:50 am Approaching Commercialization Hurdles: Other Treatments Available, Pricing & Reimbursement
Synopsis
• Launching & Maximizing the Commercial Success of an IRD Gene Therapy
• Looking for the needle in the haystack – IRD landscape
• Commercial opportunities and challenges when launching an Ocular rare disease therapy: access, genetic diagnosis and patient identification
• Key learnings for upcoming launches
9:20 am Q&A Panel Discussion
9:40 am Speed Networking
10:10 am Morning Break
FROM RARE TO COMMON OPHTHALMIC DISORDERS: DELIVERING LARGE GENES TO THE RETINA
11:00 am Preclinical Challenges with Preclinical Models, Measuring Genetic Expression & Variability of Transfer
Synopsis
• Talk details to follow
11:20 am Session Reserved
Synopsis
• Talk details to follow
11:40 am The Single AAV-minigene Approach for Delivery of Large Genes to the Retina • AAV vectors are efficacious for gene delivery to the retina
Synopsis
• Several retina specific genes are too large for AAV vectors
• Mini-genes have been created that are functional and fit in traditional AAV vectors
12:00 pm Q&A Panel Discussion
12:30 pm Lunch Break
MEASURING GENE EXPRESSION IN THE EYE: DEFINING ENDPOINTS & OUTCOME MEASURES
2:00 pm Measuring Transduction Efficiency in Preclinical Ocular Models
Synopsis
• Talk details to follow
2:20 pm Outcome Measures for Clinical Trials In Inherited Retinal Degenerations
Synopsis
• New imaging modalities can provide information about retinal structure and function in patients with inherited retinal degenerations
• Structural measures correlate with measures of visual function
• Imaging retinal structure and function can be used to monitor disease progression and response to genetic and other therapies for patients with inherited retinal degenerations
2:40 pm Defining Novel Endpoints for IRDs
Synopsis
• Development of a novel functional vision endpoint
• OCT Ellipsoid zone as a structural indicator of function
• Microperimetry as a marker of retinal function and sensitivity
3:00 pm Q&A Panel Discussion
3:30 pm Afternoon Break
CLINICAL PROGRESS IN RARE & COMMON OPHTHALMIC DISORDERS
4:00 pm A Patient’s Perspective: Jake’s Story & Q&A Session
4:20 pm Gene Therapy for GUCY2D Leber Congenital Amaurosis (LCA1)
Synopsis
• Early signs that subretinal delivery of AAV-GUCY2D is safe and effective in GUCY2D LCA1 patients
• Significant improvements in both rod- and cone-mediated vision
• No ocular inflammation
4:40 pm Comparing Clinical Progress with Different Administration Routes
Synopsis
• Evaluating clinical progress in Wet AMD and DR
• Exploring the differences in challenges between subretinal and suprachoroidal delivery
5:00 pm Q&A Panel Discussion
5:30 pm Chair’s Closing Remarks
5:45 pm Scientific Poster Session
