09:00 – 12:00 | Workshop A
Navigating Novel Ophthalmic Endpoint Development & Regulatory Validation to Accelerate Disease Therapeutic Approvals
Synopsis
Novel endpoint development and validation are major challenges in ophthalmic gene therapy, with regulatory acceptance crucial for approval. Traditional vision measures often fall short in rare, slow-progressing diseases, while patient-reported outcomes add variability. Join us in this workshop to identify effective endpoints and regulatory strategies to increase regulatory approval.
Key Discussion Points:
- Exploring early disease signs and endpoint selection strategies to optimize regulatory timelines for gene therapy trials
- Examining microperimetry and FST endpoint validation challenges to address real-world implementation barriers in inherited retinal diseases
- Comparing FDA and EMA regulatory perspectives on functional endpoints to navigate divergent approval requirements effectively
- Discussing novel endpoint acceptance and clinical meaningfulness criteria to advance ophthalmology-specific gene therapy development pathways
12:00 pm LUNCH BREAK & NETWORKING
01:00 – 04:00 | Workshop B
Leveraging AI for Capsid Engineering to Optimize Delivery & Advance Next-Generation Ophthalmic Gene Therapies
Synopsis
AI integration in gene therapy requires strategic wet lab-computational coordination and high-quality datasets. Join this workshop to explore practical machine learning implementation for capsid optimization targeting ILM crossing and production efficiency. Discuss emerging frontiers including payload engineering, inflammatory response mitigation, and laboratory automation. Collaborate on navigating AI disease models and regulatory shifts away from animal testing.
Key Discussion Points:
- Implementing AI-driven capsid engineering to optimize ILM crossing and production efficiency in ophthalmic gene therapy
- Coordinating wet lab experimental design with machine learning pipelines to generate high-quality datasets for effective AI integration
- Exploring payload optimization and inflammatory response mitigation to address key hurdles in gene therapy development
- Evaluating emerging AI applications including laboratory automation and disease models to navigate regulatory shifts away from animal testing