Explore the Agenda
8:00 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
Engineering Early Market Access Wins to Facilitate Commercial Success
9:00 am Panel Discussion: Surveying Current Investor Sentiment in Gene Therapy to Strengthen Your Funding Strategy
- Understanding what drives interest and what’s driving hesitation across the field
- Discussing the role of policy, regulation, and public trust in shaping long-term investment
- Reviewing the various sources of funding that exist for companies beyond VCs
10:00 am Starting With the End in Mind to Optimize Gene Therapy Development for Long-Term Impact
- Designing clinical trials and programs with commercialization and patient access in mind from the start
- Defining the right target patient and unmet need to build a strong, scalable value proposition
- Maximizing long-term benefits by prioritizing early decisions on formulation, administration, and cost reduction
10:30 am Pursuing Novel Funding Models for Ultra Rare Diseases
- Highlighting creative funding strategies that can sit alongside traditional grant or venture capital models
- Defining what foundations look for when making strategic investments and ensuring progress even if a company steps back
- Showcasing collaborative approaches that keep pipelines alive by piecing together diverse funding sources
11:00 am Morning Break & Networking
12:00 pm AAV-TAZ Gene Therapy for Barth Syndrome
- Reviewing efforts to translate AAV gene therapy for Barth syndrome, in partnership with the Barth Syndrome Foundation
- Optimizing the preclinical Barth syndrome model and the AAV-TAZ therapeutic candidate vector.
12:30 pm Preclinical Development of an AAV-Based Gene Therapy for FRRS1L Epileptic Encephalopathy
- Gaining insight into cutting-edge AAV9-mediated FRRS1L gene therapy development for a devastating epileptic encephalopathy
- Learning how dose-dependent efficacy and molecular restoration in knockout models support the therapeutic potential of FRRS1L replacement
- Discovering how these findings inform IND-enabling studies and the broader advancement of gene therapy for neurodevelopmental disorders
1:00 pm Lunch & Networking Break
Strengthening Clinical Translation Through Robust Analytical Development & Preclinical Studies
2:00 pm Outlining the Journey from Adult Approval to Pediatric Approval
- Designing preclinical studies that demonstrate efficacy and durability in young animal models
- Building the regulatory and clinical evidence needed to progress safely and confidently from adult to younger patient populations
- Understanding challenges including toxicity, immunology, and redosing capabilities
2:30 pm The Role & Implementation of AAV Immunogenicity Assays in Gene Therapy Clinical Trials
- Importance of detecting pre-existing AAV antibodies to ensure safety and efficacy in gene therapy trials
- Determination of thresholds for AAV immunogenicity assays through pre-clinical and clinical studies
- Implementation of validated AAV cut-offs in patient selection strategies
3:00 pm Understanding Comparability Through Deeper Product Characterization
- Using more innovative tools to demonstrate a deep understanding of your product to regulators
- Fully understanding the function of potency assays to streamline the comparability journey
- Assessing different techniques and ways of overcoming common challenges
3:30 pm Afternoon Break & Networking
4:00 pm Designing Smarter Trials with External Controls & the Use of Digital Twins
- Investigating how external controls and natural history data can support trial design when internal controls are not feasible
- Evaluating strategies for rare disease trials where there is variability of progression which needs to be quantified
- Understanding how trial design choices impact speed, cost, and regulatory success across biotech and pharma settings
Driving Rare Disease Trial Success Through Community Partnership & Innovative Controls
4:30 pm Panel Discussion: Connecting with Communities to Build Trust in Clinical Research
- Building trust through transparent education that supports long-term trial participation
- Translating complex gene therapy concepts into clear, accessible materials for families and caregivers
- Sharing real-world strategies that improve recruitment, retention, and patient satisfaction
5:15 pm Navigating Placebo Control Arms in Rare Disease Clinical Trials
- Weighing the pros and cons of placebo versus historical controls in small patient populations
- Understanding evolving regulatory guidance and recent approvals granted without control arms
- Addressing competitive pressures and leveraging shared natural history studies to strengthen trial design