Day One

• 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

• Exploring how an innovative, validated process leveraging fixed-bed bioreactors with the option to integrate perfusion technology is advancing adherent manufacturing for gene therapies
• Gaining deeper insights into cell line kinetics and optimizing viability, health, and harvest timing – a level of process control often difficult to achieve with traditional suspension batch methods
• Discovering how this approach translates into cleaner upstream material, higher product volumes, and improved full capsid percentages for AAV viral vectors
• Learning how these advancements enable faster development, more efficient manufacturing, and higher quality products, ultimately accelerating market access for gene therapies, particularly for rare diseases

• 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

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.

• 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

• 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

• 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

• Transient, B cell directed immunomodulation regimen using anti-mCD20+antimBAFF effectively depleted B cells in the blood
• Anti-mCD20+anti-mBAFF reduced formation of anti-AAV IgM and IgG and allowed liver-directed AAV redosing in a subset of animals
• Redosing was possible using the same AAV gene therapy vector at clinically relevant doses

• 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

• 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 influences the ability to target previously inaccessible disease sites

• 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

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.

• 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

• 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

• 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 capsids for CNS, muscle, and ophthalmic therapeutics