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 Comparing Epigenetic Options to Overcome AAV Size Constraints & Improve Payload Efficiency
- Surveying the range of epigenetic editing tools available as alternatives to conventional Cas9 to help you identify smaller, more packaging-friendly options for your AAV program
- Contrasting the efficiency, specificity and size profiles of different editing tool to equip you with a clearer framework for selecting the right tool within AAV constraints
- Highlighting real-world applications of compact epigenetic editing tools across gene editing programs to give you practical insight into how these tools are being deployed clinically
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