Explore the Agenda
8:00 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
9:00 am Panel Discussion: Scaling Clinical Operations to Support Gene Therapies for Larger Populations
- Highlighting the limitations of current small-batch methods in meeting the needs of larger patient populations
- Understanding the unique manufacturing, financial, and regulatory challenges associated with working on common disease indications
- Anticipating the scientific and operational shifts needed to make gene therapy viable for high-prevalence conditions
10:00 am Gene Therapy for Dry AMD Using AAV to Improve Durability & Compliance
- Complement Inhibitors clinically validated for treatment of dry AMD
- AAV vectors can potentially transform treatment from monthly injections to a single long-lasting intervention
- Intravitreal delivery using novel AAV capsid
10:30 am Morning Break & Networking
Creating Viable Market Strategies for Gene Therapies in Common Disorders
11:30 am Case Study: Building an Economically Viable Path for Gene Therapy in Osteoarthritis
- Leveraging novel vectors to reduce cost of goods and create greater economic value
- Understanding how proactive payer engagement and early market education can pave the way for smoother commercialization
- Exploring how cross-functional collaboration and continuous learning have shaped a resilient development strategy capable of navigating scientific, clinical, and economic hurdles
12:00 pm Building Smart Pricing Strategies to Secure Reimbursement for Gene Therapy in Common Conditions
- Understanding how payers evaluate value when multiple therapies exist for the same disease
- Learning how coverage decisions, rebates, and clinical trade-offs impact pricing outcomes
- Exploring how to align internal teams on pricing strategy early to avoid reimbursement delays and maximize launch success
12:30 pm Lunch & Networking Break
Pinpointing the Right Targets & Tools to Make Gene Therapy Viable for More Widespread Conditions
1:30 pm Roundtable Discussion: Identifying Clear Biological Targets to Make Gene Therapy Work for Common Diseases
- Exploring whether common diseases like cardiovascular disorders, diabetes, and obesity have gene therapy-suitable targets
- Understanding how biology can guide modality choice and clarify the path forward for gene therapy
- Using real disease examples to assess when gene therapy offers a viable and differentiated approach
2:15 pm Evaluating the Right Modalities to Deliver Gene Therapy at Scale in Common Diseases
- Comparing gene therapy to small molecules and biologics in terms of tractability and therapeutic reach
- Investigating whether advanced modalities like AAV, mRNA, or LNPs can meet the demands of high-prevalence diseases
- Delving into use cases to understand where gene therapy may offer unique value or face limitations