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

  • Tara Moore Professor of Personalized Medicine, Ulster University; Chief of Research & Innovation, Avellino

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

  • Nadia Waheed Associate Professor in Ophthalmology Tufts University Medical School, Chief Medical Officer Gyroscope Therapeutics

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

  • Thomas Ciulla Vitreoretinal Medicine & Surgery, Chief Medical Officer & Chief Development Officer , Clearside Biomedical Inc.

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

  • Nadia Waheed Associate Professor in Ophthalmology Tufts University Medical School, Chief Medical Officer Gyroscope Therapeutics
  • Thomas Ciulla Vitreoretinal Medicine & Surgery, Chief Medical Officer & Chief Development Officer , Clearside Biomedical Inc.
  • Stavros Ntogiako Head of Luxturna & Gene Therapy, Novartis
  • Tara Moore Professor of Personalized Medicine, Ulster University; Chief of Research & Innovation, Avellino

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 Explore Platform, Posters & Discussion Session

2:20 pm Outcome Measures for Clinical Trials In Inherited Retinal Degenerations

  • Jacque Duncan Professor, Clinical Ophthalmology, University of California San Fransisco

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

  • Jacque Duncan Professor, Clinical Ophthalmology, University of California San Fransisco
  • Daniel Chung Chief Medical Officer , SparingVision

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)

  • Shannon Boye Founder & Chief Scientific Officer , Atsena Therapeutics

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