Explore the Agenda
8:00 am Check-In & Coffee
8:50 am Chair’s Opening Remarks
Building Confidence & Clarity in the Present Regulatory Landscape to Accelerate Progress
9:00 am Advancements at the MHRA to support ATMP Development
9:30 am Navigating Regulatory Setbacks & Rebuilding Confidence in Gene Therapy Programs
- Learning from both setbacks and successes to accelerate the field as one
- Exploring real-world examples of programs that pivoted and re-engaged with regulators
- Sharing lessons learned from teams that had to change endpoints and still move forward
10:00 am Session reserved for
10:30 am Morning Break & Speed Networking
As this community reunites, this session will provide valuable networking time with your peers, and new faces in the gene therapy space, enabling you to forge new and lasting connections.
Preclinical & Translational
Chair:
Driving Safety & Success with Differentiated Gene Therapy Platforms
11:30 am Leveraging Platform Technology for a Differentiated Gene Therapy Approach
- Developing a high purity product profile that reduces total viral load while maximizing efficacy with a novel construct
- Pivotal trial designed with external controls to show differentiation
- Implementing a proactive, short-course immune modulation regimen designed to improve safety outcomes
12:00 pm Improving Probability of Success Through Multiple Platform Testing
- Animal vs human-derived platforms for gene therapy efficacy and relevance
- Vector genome element screening: Plasmid or AAV?
- What’s your true titre/dose? Dose extrapolation – the ocular story
Manufacturing & CMC
Chair:
Advancing Safe & Targeted Gene Delivery with Engineered AAV Capsids
11:30 am Improving Precision & Safety in Gene Therapy Through Engineered AAV Capsids
- Investigating how engineered capsids increase efficiency and safety
- Evaluating design strategies that help avoid immune responses to repeat dosing
- Understanding how capsid can enable gene therapy for muscle, brain, and other organs
12:00 pm Engineered AAVs for Safe & Therapeutic Delivery to CNS, Muscle, & Eye
- Our shared challenge: therapeutic, broadly applicable, and safe gene delivery
- Leveraging the power of pooled in vivo screens and AI-guided sequence design
- Next-generation AAV cap
12:30 pm Lunch & Networking Break
Preclinical & Translational
Enabling Extrahepatic Delivery & Broader Organ Access with Innovative Gene Editing Tools
1:30 pm Leveraging CRISPR Technologies for Wider Organ Delivery
- Highlighting how smaller CRISPR enzymes open access to tissues that other delivery systems cannot reach
- Exploring the potential for enzymes to be packaged into more flexible delivery vehicles
- Investigating how enzyme size influence
2:00 pm A Gene Delivery System that can Efficiently Modify Smooth Muscle Cells & Pericytes of the Vasculature
- Utilizing CRISPR-based and gene addition therapeutics approach to target the smooth muscle lining blood vessels
- Paving a way for future gene therapies to target indications linked to this
Manufacturing & CMC
Integrating Strategy & Innovation to Deliver Next-Generation Gene Therapies
1:30 pm Revolutionizing Manufacturing to Make Gene Therapies Affordable
- Forecasting next-generation technologies that could dramatically reduce production costs
- Pinpointing remaining scientific and technical barriers to scalable manufacturing
- Aligning innovation timelines with clinical and commercial readiness to support faster adoption
2:00 pm Walking the Walk: CMC & Operational Strategy
- What can we learn from mature drug developers and biotechs
- How to manage manufacturing capacity and partners
- Developing fit for purpose frameworks from virtual companies to pharma
2:30 pm Afternoon Break & Poster Session
This is your opportunity to contribute to the conversation and share your cutting-edge research with this community, while discovering exciting work carried out by your peers.
Advancing Clinical Trial Design with Biomarker & CDx Flexibility
3:30 pm Roundtable Discussion: Designing Flexible Trials to Withstand Changing Biomarker Expectations
- Identifying intermediate endpoints that could bridge the gap between early biomarker data and long-term clinical outcomes
- Evaluating the unique implications for rare disease programs where long-term outcomes are harder to measure
- Anticipating the impact of a move toward clinical outcome-based approvals on development timelines and investment strategies
4:15 pm Anticipating Diagnostic Demands to Avoid Costly Delays in Gene Therapy Development
- Clarifying the evolving FDA expectations for companion diagnostics in gene therapy programs
- Identifying strategies to mitigate delays caused by late-stage CDx requirements
- Understanding how regulatory ambiguity impacts patient selection and program timelines