Day Two

• Identifying strategic, regulatory, and technical barriers limiting gene editing therapeutics to rare, single-patient applications and exploring pathways to expand into larger patient populations
• Examining how industry leaders are navigating manufacturing challenges, clinical development efficiency, and regulatory alignment to make genome editing commercially viable across multiple indications
• Discussing the organizational capabilities, investment priorities, and platform design decisions required to transition from boutique therapies to scalable, multi-disease treatment models

• Examining how immune responses to vectors and cargo drive blood clearance of gene editing therapies to help you identify strategies that improve therapeutic persistence
• Exploring approaches to reduce immune-mediated clearance during repeat dosing to equip you with practical solutions for maintaining therapeutic efficacy over time
• Assessing how leading companies are engineering vectors to evade immune recognition to give you real-world insights into overcoming immunogenicity in your program

• Showcasing how prime and RT editing technologies can be packaged within AAV to give you insight into a uniquely precise approach to in vivo gene editing
• Unpacking the engineering challenges of fitting prime editing systems within AAV size constraints to help you understand the innovations enabling this delivery approach
• Highlighting how AAV-delivered prime editing compares to conventional CRISPR approaches to equip you with a broader perspective on next-generation in vivo editing options

• Reviewing engineering efforts on compact gene expression modulation systems (GEMs) for durable epigenetic activation within single-AAV constraints
• Highlighting therapeutically relevant proof-of-concept applications of gene activation tools that enable AAV-based approaches, providing practical insight into their translational potential

Redirecting gene editing vectors away from the liver whilst maintaining effective dosing remains one of the field’s most pressing delivery challenges. Join this workshop to explore real-world applications of reducing LNP and AAV liver tropism, enhancing tissue-specific targeting and investigating exosomal delivery approaches, giving you practical strategies to improve both safety and therapeutic efficacy.

Workshop highlights:

• Exploring real-world approaches to reducing LNP and AAV liver tropism, equipping you with strategies to improve the safety profile of your therapeutics
• Examining methods for enhancing tissue-specific targeting of delivery vectors, enabling you to direct therapies more precisely to the right disease area
• Investigating exosomal delivery as an emerging alternative to viral and lipid vectors, giving you a broader toolkit for extrahepatic genome editing applications
• Balancing safety and effective dosing in extrahepatic delivery design, helping you develop therapeutics that meet regulatory expectations without compromising efficacy

• Demonstrating durable factor VIII expression enabling potentially curative one-time hemophilia a treatment strategies
• Establishing precise genome editing minimizing off-target activity supporting confident regulatory advancement toward clinical translation
• Leveraging proprietary metagenomics platforms expanding scalable therapeutic applications across secreted protein deficiencies beyond hemophilia

• Anchoring preclinical programs on human genetic insights and the critical 60/40 hemoglobin ratio to optimize therapeutic development
• Optimizing therapeutic strategies by aligning preclinical endpoints with target product profiles to achieve meaningful clinical outcomes
• Achieving consistent 60/40 hemoglobin ratios in clinic, reducing painful vaso-occlusive crises to meet registrational endpoints

• Investigating how genome editing is being used to make porcine organs compatible with humans to give you insight into one of the most pioneering clinical translation applications in the field
• Mapping the regulatory and safety considerations specific to xenotransplantation programs to help you understand the unique challenges of translating genome editing into organ compatibility
• Highlighting real-world progress in xenotransplantation to equip you with a broader perspective on how genome editing is expanding the boundaries of therapeutic application