THE EYE: IMMUNE PRIVILEGE & CHALLENGES

8:00 am Chair’s Opening Remarks

8:10 am Landscape of Ocular Gene Therapy Immunogenicity & Vector Strategies to Overcome Inflammation

Synopsis

• Viral vectors elicit dose-dependent intraocular inflammation
• Managing immunogenicity is critical for achieving successful gene therapies
• Vector-based approaches should be utilized to mitigate inflammation

8:20 am Retinitis Pigmentosa to Dry AMD: A Clinical Update on Optogenetic Therapy

Synopsis

• Demonstrating the safety of optogenetic gene therapy in advanced retinal degenerations
• Describing the initial efficacy data
• Positioning such therapies in the spectrum of emerging therapies for retinal degenerations

8:40 am Host Immunity after Suprachoroidal AAV in Nonhuman Primates

  • Glenn Yiu Associate Professor, University of California, Davis

Synopsis

• Highlight novel use of suprachoroidal microneedles for AAV delivery
• Review transgene expression from different routes of AAV administration
• Explain host immune privilege and responses related to retinal gene therapy
• Discuss how AAV biodistribution may impact host immune response

9:00 am Q&A Panel Discussion

  • Glenn Yiu Associate Professor, University of California, Davis
  • Ying Kai Chan Visiting Scholar, Harvard University
  • José-Alain Sahel Chairman & Professor, Ophthalmology, University of Pittsburgh

9:30 am Morning Break

VECTOR DESIGN & NON-VIRAL VECTORS TO IMPROVE RETINAL SELECTIVITY

10:10 am Machine Guided Capsid Engineering for Ocular Diseases

  • Eric Kelsic Chief Executive Officer, Dyno Therapeutics

Synopsis

• Machine-guided capsid engineering represents a new approach to overcome the current challenges of in vivo gene delivery
• This approach combines three advanced technologies: i. next-gen library synthesis, ii. next-gen sequencing, and iii. machine learning
• With this workflow the search for improved capsids can be dramatically accelerated

10:30 am Efficient Gene Therapy Manufacturing for Ocular Diseases

Synopsis

• How to scale an AAV program with efficient manufacturing
• Utilizing adenovirus helper plasmid for AAV production
• Considerations for partnering with a CDMO

10:50 am Non-Viral Vectors for Lower Cost Manufacturing & Immune Mitigation

Synopsis

• Eyevensys technology offers major advantages such as safety, flexibility, low cost and sustained but controllable and transitory protein production
• No limit in gene size and possible multiple protein expression
• Large pipeline for major ocular diseases
• Has shown safety and feasibility in humans and promising signs of efficacy

11:10 am Retinal Gene Therapy: Novel Vector Technologies

Synopsis

• Providing an overview on remaining key challenges in retinal gene therapy and introducing ViGeneron’s approach to address key challenges
• Introducing the vgAAV vector technology, which supports less invasive intravitreal delivery
• Discussing the REVeRT dual AAV technology for efficient delivery of large genes

11:30 am Q&A Panel Discussion

12:00 pm Lunch & Networking

ADDRESSING NEW INDICATIONS WITH NOVEL APPROACHES FOR LONG TERM EXPRESSION & RETINAL DISTRIBUTION

1:00 pm Gene Agnostic Approaches Targeting Later Stage Diseases for Photoreceptor Function

Synopsis

• Investigational Gene Agnostic Approaches to Preserve or Restore Cone Function in Inherited Retinal Disease
• Explore the concept of gene agnostic gene therapy for inherited retinal disease
• Mechanisms for RDCVF and GIRK2 strategies for gene agnostic approaches
• Preclinical data and next steps

1:20 pm Positive Interim Results in Bionic Sight’s Optogenetic Gene Therapy Trial

Synopsis

• Optogenetic gene therapy can target a broad range of retinal degenerative diseases
• Combining the gene therapy with a neural coding device
• Sharing current results
• Discussion of mechanism(s) of action

1:40 pm The Potential of Intravitreal Human Retinal Progenitor Cells (hRPCs) in Inherited Retinal Disorders

Synopsis

• Exploring key unmet medical needs in inherited retinal disorders
• Discussing our intravitreal hRPC development program and recent data
• Reflecting on key learnings from building an advanced therapy organization

2:00 pm Q&A Panel Discussion

2:30 pm Panel: Discussing Pharma Partnering & Collaboration: Challenges & Opportunities

  • Udo Maier Head of Research Beyond Borders Europe, Boehringer Ingelheim
  • Stephen Poor Director of External Opportunities & Translational Biomarkers, Novartis

3:00 pm Afternoon Break

RARE VS COMMON DISORDERS PATIENT POPULATIONS: CLINICAL CHALLENGES & REGULATION

3:15 pm Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy (LHON): Regulatory Considerations: US & Europe Regulators

Synopsis

• LHON disease and its main clinical aspects
• Walking through late stage development of Lumevoq gene therapy
• Discussing regulatory pathways
• Highlighting key learnings

3:35 pm Understanding the Pre-Clinical Regulators’ Expectations for Gene Therapy Trials

Synopsis

• Introducing the regulatory frame
• Proof of concept studies
• Regulatory tox studies
• Risk based approach

3:55 pm Regulatory Considerations for Gene Therapy Development” covering the following topics:

  • Snehal Naik Regulatory Policy Leader, Spark Therapeutics

Synopsis

• What’s different about the regulatory path for gene therapies
• Highlights from FDA’s gene therapy for retinal disease guidance
• Health authority interaction mechanisms and designations that can enable gene
therapy development

4:15 pm Gene Agnostic Approaches for the Treatment of Inherited Retinal Diseases

  • Claire Gelfman Chief Scientific Officer , Foundation Fighting Blindness

Synopsis

• Reviewing therapeutic approaches to treat inherited retinal diseases (IRDs)
• Highlighting the rationale of gene-agnostic approaches to treat IRDs
• Reviewing universal disease mechanisms currently being targeted for treatment

4:35 pm Q&A Panel Discussion

5:00 pm Chair’s Closing Remarks