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.

  • Discover Cutting-Edge Solutions: Delve into advanced strategies for targeted delivery to key tissue types, including HSCs, the liver, CNS, muscle, and immune cells.

  • 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.

  • 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. 

  • In vivo LNP-mediated editing in HSCs
    Cecilia Cotta R a.m.usino, PhD – Tessera Therapeutics 

  • Efficient and liver-detargeted in vivo multiplex gene editing of human HSPCs  
    Luca Biasco, PhD – nChroma Bio 

  • Targeted HDAd-mediated editing in HSCs 
    Chang Li, PhD – University of Washington 

  • Group Q&A: 15 minutes 

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. 

  • In utero editing of muscles with LNPs 
    Bill Peranteau, MD – Children’s Hospital of Philadelphia 

  • AAV-mediated epigenetic editing therapy for FSHD 
    Daniel Hart, PhD – Epicrispr Biotechnologies 

  • Group Q&A: 10 minutes 

Lunch + Exhibits | 11:50 a.m. – 12:50 p.m. 

Session 3: Immune Cell | 12:50 – 1:50 p.m.

  • In vivo targeting of immune cells with LNPs
    Haig Aghajanian, PhD – Capstan Therapeutics 

  • In vivo targeting of immune cells with lentiviral vectors  
    Hans-Peter Kiem, MD, PhD – Fred Hutchinson Cancer Center 

  • Group Q&A: 10 minutes 

Break 2 | 1:50 – 2 p.m. 

Session 4: CNS | 2 – 3 p.m. 

  • AAV-mediated epigenetic editing for prion disease 
    Sonia Vallabh, PhD – Broad Institute 

  • Brain-wide genome editing via STEP-RNPs for treatment of Angelman syndrome 
    Yong-Hui Jang, MD, PhD – Yale 

  • Group Q&A: 10 minutes 

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.

  •  LNP-mediated editing for treatment of hypercholesteremia (updates on clinical trials)
    Troy Lister, PhD – Verve Therapeutics 

  • LNP-mediated base editing for AATD and GSD1a
     a.m.y Simon, MD – Be a.m. Therapeutics 

  • Personalized liver-directed corrective gene editing 
    Rebecca Ahrens-Nicklas, MD, PhD – Children’s Hospital of Philadelphia 

  • 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?

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Goals + Takeaways

The goals of this conference are to: 

  • 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  

  • 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: 

  • 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

  • Insights into how researchers are overcoming tissue-specific delivery challenges

  • Emerging strategies to boost targeted gene editing efficiency, specificity, and durability in vivo

  • Lessons from the translational pipeline that can inform your research

  • New opportunities for collaboration and cross-disciplinary exchange

2025

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