Event Program
November 20-21, 2025 | San Diego + Virtually
Dedicated to cutting-edge solutions for targeted delivery, this event is your chance to engage with pioneering research, overcome tissue-specific challenges, and shape the future of medicine by driving the field of gene editing forward.
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Discover Cutting-Edge Solutions: Delve into advanced strategies for targeted delivery to key tissue types, including HSCs, the liver, CNS, muscle, and immune cells.
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Gain Translational Insights: Learn from real-world lessons and emerging technologies that can directly inform your research and accelerate your work from the lab to the clinic.
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Connect and Collaborate: Build meaningful connections with top researchers and clinicians in an intimate environment designed for knowledge exchange and networking.
Register to Attend
Day 1 - Thursday, Nov. 20
Registration, Poster Check-in, Breakfast | 7:30 – 8:30 a.m.
Welcome, Opening Remarks | 8:30 – 8:35 a.m.
Session 1: HSCs | 8:35 – 10:05 a.m.
Break 1 | 10:05 – 10:50 a.m.
Sponsored Presentation | 10:15 –10:35 a.m.
Session 2: Muscle | 10:50 – 11:50 a.m.
Lunch + Exhibits | 11:50 a.m. – 12:50 p.m.
Session 3: Immune Cell | 12:50 – 1:50 p.m.
Break 2 | 1:50 – 2 p.m.
Session 4: CNS | 2 – 3 p.m.
Break 3 | 3 – 3:45 p.m.
Sponsored Presentation | 3:20 – 3:40 p.m.
Session 5: Abstracts (15 minutes each) | 3:45 – 5 p.m.
Poster Viewing + Reception | 5 – 6:30 p.m.
Day 2 - Friday, Nov. 21
Breakfast | 7:30 – 8:30 am.
Session 1: Liver | 8:30 – 9:45 a.m.
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LNP-mediated editing for treatment of hypercholesteremia (updates on clinical trials)
Troy Lister, PhD – Verve Therapeutics
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LNP-mediated base editing for AATD and GSD1a
a.m.y Simon, MD – Be a.m. Therapeutics
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Personalized liver-directed corrective gene editing
Rebecca Ahrens-Nicklas, MD, PhD – Children’s Hospital of Philadelphia
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Group Q&A: 15 minutes
Break | 9:45 – 10:30 a.m.
Sponsored Presentation | 9:55 – 10:15 a.m.
Session 2: Concluding Panel | 10:30 – 11:40 a.m.
- Kiran Musunuru, MD, PhD – University of Pennsylvania
- Hans Peter Kiem, MD, PhD – Fred Hutch Cancer Center
- Sonia Vallabh, PhD – Broad Institute
- Cecilia Cotta Ramusino, PhD – Tessera Therapeutics
Closing Remarks | 11:40 a.m. – Noon
Ready to Register?
Register Now
Goals + Takeaways
The goals of this conference are to:
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Highlight cutting-edge scientific and clinical advancements in the delivery of gene editing in vivo, focusing on challenges and topics relevant to academic researchers in basic science
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Foster knowledge exchange, collaboration, and networking through scientific presentations, abstracts/poster sessions, and informal discussions
By the end of this conference, you’ll walk away with:
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A deeper understanding of how in vivo gene editing delivery is advancing across key tissue types (HSCs, liver, CNS, muscle, and immune cells), and lessons learned from progress
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Insights into how researchers are overcoming tissue-specific delivery challenges
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Emerging strategies to boost targeted gene editing efficiency, specificity, and durability in vivo
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Lessons from the translational pipeline that can inform your research
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New opportunities for collaboration and cross-disciplinary exchange