REVOLUTIONIZING GENE THERAPY ADMINISTRATION FOR OPHTHALMIC DISORDERS
8:00 am Chair’s Opening Remarks
8:05 am Progressing Multiple Gene Therapy Paths for Avellino Corneal Dystrophy
Synopsis
- Pursuing 2 paths of therapy to treat TGFBI corneal dystrophies: mutant allele specific SIRNA and CRISPR editing
- First topical SIRNA therapy for the eye
- CRISPR gene editing “Dual Cut” approach
8:35 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
8:55 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
9:15 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
9:35 am Q&A Panel Discussion
10:05 am Speed Networking
FROM RARE TO COMMON OPHTHALMIC DISORDERS: DELIVERING LARGE GENES TO THE RETINA
10:35 am Morning Break
11:20 am Preclinical Validation of a Novel Modifier Ocular Gene Therapy for the Prevention of Geographic Atrophy in Patients with Dry Macular Degeneration
Synopsis
- Oculogenex has Developed a Gene Therapy that Prevents Apoptosis and Increases Antioxidant Defenses in Murine Dry AMD Models
- Preclinical Evidence Demonstrates Photoreceptor Protection and Increased
Electroretinogram Amplitudes
11:30 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
11:50 am Q&A Panel Discussion
12:20 pm Lunch Break
MEASURING GENE EXPRESSION IN THE EYE: DEFINING ENDPOINTS & OUTCOME MEASURES
1:20 pm Panel Discussion
Synopsis
Hosted by MERIT
1:50 pm Short Break
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