Explore the Agenda
Workshop A
9:00 am Navigating Regulatory IND Packages to Accelerate Genome Editing Therapeutics into the Clinic
Securing regulatory approval for novel genome editing modalities presents unique challenges, with unclear expectations around preclinical pharmacology, toxicity and analytics from agencies. Join this workshop to explore how late-stage and commercial companies are structuring IND packages, bridging preclinical and clinical data, and collaborating directly with regulators. Gain robust strategies to accelerate your program into the clinic.
Workshop highlights:
- Examining how late-stage companies structure IND packages for novel genome editing modalities, helping you build stronger regulatory submissions faster
- Identifying which preclinical outcomes most reliably predict clinical success, enabling you to design more robust studies that satisfy regulatory scrutiny
- Exploring commercial-stage regulatory strategies across new genome editing tools, giving you a roadmap to navigate approval pathways with greater confidence
- Engaging regulators directly in panel discussion to surface industry best practices, empowering you to foster more productive agency relationships for your program
12:00 pm Lunch Break & Networking
Workshop B
1:00 pm Advancing Extrahepatic Delivery to Improve Safety & Targeting for Genome Editing Therapeutics
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