8:00 am Registration & Breakfast
8:55 am Chair’s Opening Remarks
Optimizing Ocular Delivery Method Selection & Retinal Targeting Confirmation to Maximize Therapeutic Retention & Visual Outcomes
9:00 am Mastering Gene Therapy Delivery Routes to Optimize Patient Outcomes & Clinical Success
Synopsis
- Comparing subretinal, intravitreal, and suprachoroidal delivery approaches to determine the most effective route for specific gene therapy applications
- Demonstrating advanced surgical techniques through live operative footage to provide hands-on insights into precise subretinal gene therapy injection procedures
- Evaluating pros and cons of each delivery method to guide evidence-based decision-making for optimal therapeutic outcomes
9:30 am Utilizing Suprachoroidal Delivery Technologies to Optimize Gene Therapy Targeting
Synopsis
- Evaluating when Suprachoroidal Delivery should be used to determine optimal ophthalmic gene therapy integration
- Accessing available delivery technologies and platforms to enable effective suprachoroidal therapeutic administration
- Implementing confirmation methods and monitoring techniques to validate successful suprachoroidal space delivery
10:00 am Panel Discussion: Evaluating the Interplay between Capsid Design & Route of Administration to Maximise Efficacy and Mitigate Adverse Effects
Synopsis
- Exploring innovative capsid engineering approaches to improve stability and reduce immunogenicity for safer delivery
- Comparing administration routes and their impact on biodistribution to minimize patient risk while maintaining efficacy
- Integrating capsid properties with delivery strategies to achieve controlled exposure and sustained therapeutic effect
10:30 am Morning Refreshment Break & Speed Networking
Analyzing Clinical Strategy for Prevalent & Rare Ophthalmic Therapies to Accelerate Regulatory Approval
11:30 am Pioneering Intravitreal Gene Therapy for Wet AMD: 7 Years of Clinical Experience from Phase 1 to Phase 3
Synopsis
- Examining key learnings from early-stage intravitreal gene therapy studies and how these learnings impacted late-stage study design
- Highlighting key efficacy and safety data from the phase 1 OPTIC and phase 2 LUNA studies
- Exploring an end-to-end journey of one of the first patients dosed with Ixo-vec
12:00 pm 4D-150 Intravitreal Gene Therapy: Phase II Data
Synopsis
- Understanding the advantages of intravitreal injection platform to appreciate superior delivery method over competing approaches
- Examining the safety of 4D-150 from the Phase II wet AMD trial to recognize breakthrough safety profiles in gene therapy
- Exploring the scientific evidence behind customized capsids to understand why 4DMT has solved safety challenges where nobody else has
12:30 pm Introducing the 3rd Generation iCare MAIA Microperimeter
Synopsis
- What is Microperimetry and How is it Used in Both Clinic and Research
- iCare MAIA concept – What makes the iCare MAIA Microperimeter Different
- Introducing the 3rd Generation MAIA and Discussing the Technical Advantages
12:40 pm Lunch Break & Networking
1:45 pm Vision Restoration via Mutation-Agnostic Optogenetic Therapy: ZM-02 Opportunity
Synopsis
- Introducing ZM-02 optogenetic therapy enabling light-sensitive protein expression to restore vision to patients with severe retinal degeneration
- Delivering a mutation-agnostic approach allowing treatment across genetic profiles to broaden access for diverse patient populations
- Showcasing first-in-human clinical data demonstrating safety and functional vision improvement to validate ZM-02’s therapeutic potential
Advancing Preclinical Model Selection & IND-Enabling Study Design to Accelerate Gene Therapy Clinical Translation
2:15 pm Targeting Geographic Atrophy in Age-Related Macular Degeneration to Slow Disease Progression
Synopsis
- Targeting complement pathway dysfunction through subretinal gene delivery to slow progression of geographic atrophy lesions in dry AMD patients
- Achieving meaningful reduction in lesion growth rates to demonstrate proof-ofconcept for complement modulation approaches
- Identifying critical treatment timing windows and patient characteristics to predict optimal therapeutic response in advanced AMD stages
2:45 pm Afternoon Break & Refreshments
3:15 pm De-risking Translation in Rare Ophthalmic Gene Therapy: The Axovia Experience
Synopsis
- Leveraging a disease-specific animal model, single-species toxicology, and dose-ranging studies to assess tolerability and efficacy
- Navigating an adaptable regulatory pathway, pivoting from US to UK-based Phase 1 trials, with a registrational trajectory toward BLA submission
- Driving patient access through close PAG engagement and optimizing recruitment strategies
3:45 pm Developing AAV Gene Therapies for BEST1-Associated Inherited Retinal Disease That Ensures IND Success
Synopsis
- Engineering of a safe, efficacious RPE selective AAV vector for delivery of BEST1 in dominant and recessive forms of the disease
- Proof of concept in cellular and large animal models of BEST1
- Development of clinical endpoints for a P1/2 BEST1 clinical trial