Program

This program will be updated as speakers and sessions are confirmed.

Ready to Register?

Registration opens in early July!

Program

Day 1 - Nov. 20, 2025

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.

Sponsored Session: 3:20 – 3:40 p.m. 

Session 5 Abstracts: 3:45 – 5 p.m.

Abstract 1: 15 minutes 

Abstract 2: 15 minutes 

Abstract 3: 15 minutes 

Abstract 4: 15 minutes 

 Abstract 5: 15 minutes 

Poster Viewing/Reception: 5 – 6:30 p.m. 

Day 2 - Nov 21, 2025

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.

• Participants TBD 

Closing Remarks: 11:40 a.m. – 12 p.m.