Program

Ready to Register?

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

Registration, Poster Check In, Breakfast: 7:30am – 8:30am

Welcome, Opening Remarks: 8:30 a.m. – 8:35 a.m.

Session 1 (Invited Speakers): 8:35 a.m. – 10:05 a.m. HSC’s

 Invited Speaker 1: 25 minutes Cecilia Cotta Ramusino, PhD (Tessera Therapeutics)
- Talk Topic: In vivo LNP-mediated editing in HSCs
 Invited Speaker 2: 25 minutes Luca Biasco, PhD (nChroma Bio)
- Talk Topic: Efficient and liver-detargeted in vivo multiplex gene editing of human HSPCs
Invited Speaker 3: 25 minutes Chang Li, PhD (University of Washington)
- Talk Topic: Targeted HDAd-mediated editing in HSCs
Group Q&A: 15 minutes

Break 1: 10:05 – 10:50 a.m.

·       Sponsored Session: 10:15AM –10:35 AM

Session 2 (Invited Speakers): 10:50 AM – 11:50 PM Muscle

 Invited Speaker 1: 25 minutes Bill Peranteau, MD (CHOP)
- Talk Topic: In utero editing of muscles with LNPs
Invited Speaker 2: 25 minutes Daniel Hart, PhD (Epicrispr Biotechnologies)
- Talk Topic: AAV-mediated epigenetic editing therapy for FSHD
Group Q&A: 10 minutes

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

Session 3 (Invited Speakers): 12:50 – 1:50 p.m. Immune Cell

 Invited Speaker 1: 25 minutes Haig Aghajanian, PhD (Capstan Therapeutics)
- Talk Topic: In vivo targeting of immune cells with LNPs
Invited Speaker 2: 25 minutes Hans-Peter Kiem, MD, PhD (Fred Hutch)
- Talk Topic: In vivo targeting of immune cells with lentiviral vectors 
Group Q&A: 10 minutes

Break 2: 1:50 – 2:00 p.m.

Session 4 (Invited Speakers): 2 – 3 p.m. CNS

Invited Speaker 1: 25 minutes Sonia Vallabh, PhD (Broad Institute)
- Talk Topic: AAV-mediated epigenetic editing for prion disease
Invited Speaker 2: 25 minutes Yong-Hui Jang, MD PhD (Yale)
- Talk topic: Brain-wide genome editing via STEP-RNPs for treatment of Angelman syndrome
Group Q&A: 10 minutes

Break 3: 3 – 3:45 p.m.

Breakfast: 7:30 – 8:30 am..

Session 1 (Invited Speakers): 8:30 – 9:45 a.m. Liver

 Invited Speaker 1: 20 minutes Troy Lister, PhD (Verve Therapeutics)
- Talk Topic: LNP-mediated editing for treatment of hypercholesteremia (updates on clinical trials)
Invited Speaker 2: 20 minutes Amy Simon, MD (Beam Therapeutics)
- Talk Topic: LNP-mediated base editing for AATD and GSD1a
Invited Speaker 3: 20 minutes Rebecca Ahrens-Nicklas, MD, PhD (CHOP)
- Talk Topic: Personalized liver-directed corrective gene editing
Group Q&A: 15 minutes

Break: 9:45 – 10:30 a.m.

 Sponsored Session: 9:55 – 10:15 a.m.

Session 2 (Concluding Panel): 10:30 – 11:40 a.m.

Closing Remarks: 11:40 a.m. – 12 p.m.