Explore the Agenda
8:00 am Registration & Networking
8:55 am Chair’s Opening Remarks
Advancing Commercial Viability & Therapeutic Durability by Overcoming Scalability & Immunogenicity Barriers in Gene Editing Therapeutics
9:00 am Fireside Chat: Transitioning Gene Editing Therapeutics from Bespoke Treatments to Platform Approaches for Broader Disease Indications & Commercial Sustainability
- 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
9:45 am Overcoming Vector & Cargo Immunogenicity to Improve Therapeutic Persistence in Patients
- 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
10:15 am Morning Refreshment Break & Networking
Maximizing AAV Payload Capacity to Enhance Gene Editing Therapeutic Efficacy
11:15 am Delivering Prime Editing Within AAV to Unlock Precise In Vivo Gene Editing
- 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
11:45 am Roundtable Discussion: Addressing Immunogenicity & Safety Concerns to Enable Durable Gene Editing Therapeutic Outcomes & Regulatory Approval
- Identifying immunogenic responses to CRISPR components and developing mitigation strategies to prevent immune rejection and enable sustained therapeutic efficacy
- Implementing safety monitoring frameworks to detect genotoxicity, off-target effects, and immune activation ensuring patient safety throughout clinical development
- Navigating regulatory expectations for immunogenicity assessment and long-term safety follow-up to accelerate approval pathways while maintaining durability standards
12:45 pm Lunch Break & Networking
1:45 pm Showcasing Epigenetic Editing Strategies to Overcome AAV Size Constraints & Improve Payload Efficiency
- 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
Strengthening Clinical Translation to Validate Gene Editing Therapeutic Outcomes
2:15 pm Advancing Site-Specific Genome Integration Delivering Curative Hemophilia A Therapies Through IND-Enabling Translational Development
- 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
2:45 pm Afternoon Break & Refreshments
3:15 pm Translating Genetic Insights by Optimizing Sickle Cell Therapies from Bench to Bedside to Achieve Clinical Success
- 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
3:45 pm Applying Genome Editing to Xenotransplantation to Advance Human Compatible Organ Solutions
- 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