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 Developing Proprietary AAV Capsids to Enable Para-retinal Administration Across Multiple Retinal Indications
Synopsis
- Demonstrating capsid AAV204 viability in non-human primates to establish the periretinal route of administration for retinal gene therapy
- Advancing X-linked retinoschisis program with capsid AAV204 data in mice to support progression into large animal studies
- Platforming the capsid and delivery route combination to expand therapeutic applications across multiple retinal indications including osimbotan and optic atrophy
10:30 am Morning Refreshment Break & Speed Networking
Analyzing Clinical Strategy for Prevalent Ophthalmic Therapies to Accelerate Regulatory Approval
11:30 am Presenting Phase 2 Clinical Data on Adverum Biotechnologies WetAMD lxo-vec Therapy to Show Progress of Ophthalmic Gene Therapy
Synopsis
- Iterating on capsid structure and dosing protocols to achieve the optimal efficacysafety balance while minimizing adverse events
- Leveraging regulatory pathways and collaborative agency relationships to accelerate development timelines
- Working strategically within FDA frameworks to demonstrate compelling efficacy data that meets regulatory expectations
12:00 pm 4D-150 Intravitreal Gene Therapy: Phase II & Phase III Data
Synopsis
- Understanding the advantages of intravitreal injection platform to appreciate superior delivery method over competing approaches
- Examining the unbelievably earth-shatteringly safe Phase III wet AMD trial data 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 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
1:00 pm Lunch Break & Networking
1:30 pm
Advancing Preclinical Model Selection & IND-Enabling Study Design to Accelerate Gene Therapy Clinical Translation
2:30 pm Navigating Preclinical to Clinical Translation of AXV-101 to Accelerate Gene Therapy Development
Synopsis
- Selecting optimal animal models and securing regulatory approval to establish robust preclinical foundations across FDA and MHRA requirements
- Conducting dose-ranging studies achieving therapeutic windows comparable to other therapies to ensure safe and effective human translation
- Overcoming mouse-to-human dosing challenges and regulatory hurdles to successfully initiate clinical trials with promising early safety data
3:00 pm Showcasing Nonclinical Efficacy & Toxicology Data Packages to Demonstrate IND Submission Readiness for Ophthalmic Gene Therapy Development
Synopsis
- Demonstrating comprehensive in vitro and in vivo efficacy studies to establish robust proof-of-concept data for IND approval
- Translating efficacy findings from rodent models to non-human primates to validate therapeutic potential in different species
- Understanding how complementary preclinical models address individual limitations to build complete nonclinical data packages
3:30 pm Afternoon Break & Refreshments
4:00 pm Leveraging Transgenic Animal Models for Robust In Vivo Validation of Therapeutic Targets in Inherited Retinal Diseases
Synopsis
- Choosing disease-specific knockout mouse models to accurately mirror human genetic pathology and provide relevant efficacy data
- Collaborating with pre-clinical partners experienced in retinal gene delivery to optimize vector administration protocols
- Collecting longitudinal functional and histological evidence of therapeutic benefit to build compelling proof-of-concept data supporting regulatory confidence
4:30 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