Joint with: Targeted Delivery of Nucleic Acid Medicines
Precision Genome Engineering: From Laboratory to Clinic
Jan 25–28, 2027 | Keystone Resort, Keystone, CO, United States
Scientific Organizers:
Erik J Sontheimer and Leonela Amoasii
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Jan 25–28, 2027 | Keystone Resort, Keystone, CO, United States
Scientific Organizers: Erik J Sontheimer and Leonela Amoasii
Erik J Sontheimer, PhD
University of Massachusetts Medical School
Erik J. Sontheimer, Ph.D., is a Professor in the RNA Therapeutics Institute at the UMass Chan Medical School and a leading pioneer in RNA biology and CRISPR-based genome editing. His research has defined the molecular mechanisms of RNA-guided nucleases and advanced their translation into precise, safe, and efficient genome engineering tools. He has served as co-chair of the NIH Somatic Cell Genome Editing Program Steering Committee and the National Cancer Institute Board of Scientific Counselors and co-founded Intellia Therapeutics. He has extensive experience organizing international scientific meetings and mentoring early-career investigators across the genome editing and RNA biology communities.
Leonela Amoasii, PhD
Leonela Amoasii, Ph.D., is a molecular biologist and gene-editing scientist with over 15 years of experience in translational genetics, genome engineering, and cell and gene therapy. She was a founding scientist at Vertex Pharmaceuticals, developing CRISPR-based therapies for Duchenne muscular dystrophy. She contributes to translational programs integrating precision genome editing, delivery, and automation to enable scalable genetic medicine platforms. Her research bridges mechanistic discovery with in vivo therapeutic validation and clinical translation. Dr. Amoasii has presented at major international meetings and is deeply committed to mentoring and fostering inclusive participation in science.
Available Formats:
Supported by:
Livestream
In Person
On Demand
***Meeting program subject to change.
Available Formats: = Livestream = In Person = On DemandMerchandise Options
Registration Options
Monday, January 25, 2027
Fundraising
Registration
4:00–8:00 PM
Welcome Mixer
6:00–8:00 PM
Tuesday, January 26, 2027
Breakfast
7:00–8:00 AM
Welcome Remarks (Joint)
8:00–8:10 AM
Keynote Address (Joint)
8:10–9:00 AM
Charles Gersbach, Duke University
AAV-Delivered Epigenome Editors
AAV-Delivered Epigenome Editors
Viral Delivery of Genome Editors and Genetic Medicines (Joint)
9:00–11:30 AM
Beverly L. Davidson, Children’s Hospital of Philadelphia and University of Pennsylvania
AAV Capsid Engineering for Broad CNS Distribution
AAV Capsid Engineering for Broad CNS Distribution
Sonia Vallabh, Broad Institute
Development of Medicines to Treat Prion Diseases: A Personal Journey
Development of Medicines to Treat Prion Diseases: A Personal Journey
Pascaline Aimé-Wilson, Regeneron Pharmaceuticals
Antibody-based Retargeting Platforms for Delivery of Genetic Medicines to the Central Nervous System
Antibody-based Retargeting Platforms for Delivery of Genetic Medicines to the Central Nervous System
Short Talk(s) Chosen from Abstracts
Coffee Break
9:30–9:50 AM
Panel Discussion: Lessons Learned So Far: From Concept to Clinic
11:40–12:40 PM
Poster Setup
12:40–1:00 PM
On Own for Lunch
12:40–2:30 PM
Poster Viewing
1:00–10:00 PM
Symposia Spotlight 1
2:30–4:30 PM
Short Talks Chosen from Abstracts
Coffee Available
4:30–5:00 PM
Next-Generation Editing Tools: Continuing Innovation to Push the Limits of Genome Engineering
5:00–7:00 PM
Speaker to be Announced
Erik J Sontheimer, University of Massachusetts Medical School
Next-Generation Genome Editing Platforms
Next-Generation Genome Editing Platforms
Weixin Tang, University of Chicago
Reprogramming Deaminase Specificity for Precise Base Editing
Reprogramming Deaminase Specificity for Precise Base Editing
Short Talk(s) Chosen from Abstracts
Social Hour with Dinner
7:00–8:00 PM
Posters
7:30–10:00 PM
Wednesday, January 27, 2027
Breakfast
7:00–8:00 AM
Machine-Learned Molecules: AI-Driven Design in Genome Engineering & Delivery
8:00–11:00 AM
Hiroshi Nishimasu, RCAST, The University of Tokyo
Engineering Programmable Transposases
Engineering Programmable Transposases
Peter Cameron, Profluent Bio
Personalized Gene Editing at Scale: An AI-Driven Approach
Personalized Gene Editing at Scale: An AI-Driven Approach
Martin Pacesa, University of Zurich
Computational Design of Next Generation Gene Editing Tools
Computational Design of Next Generation Gene Editing Tools
Jonathan S. Gootenberg †, Harvard Medical School
Talk Title to be Announced
Talk Title to be Announced
Short Talk(s) Chosen from Abstracts
Coffee Break
9:00–9:20 AM
Poster Setup
11:00–1:00 PM
On Own for Lunch
11:00–3:00 PM
Poster Viewing
1:00–10:00 PM
Career Roundtable
3:00–4:30 PM
Coffee Available
4:30–5:00 PM
Editing with Confidence: Evaluating Safety, Specificity, and Genomic Integrity
5:00–7:00 PM
Maria Mirotsou, Scribe Therapeutics
Advancing In Vivo CRISPR CasX-Based Therapies to Target Key Dyslipidemia Drivers
Advancing In Vivo CRISPR CasX-Based Therapies to Target Key Dyslipidemia Drivers
Shengdar Q Tsai, St. Jude Children's Research Hospital
Off-Target Profiling for Base and Prime Editing
Off-Target Profiling for Base and Prime Editing
Marcello Maresca, AstraZeneca
Enhancing Prime Editing Platforms for in vivo Applications
Enhancing Prime Editing Platforms for in vivo Applications
Short Talk(s) Chosen from Abstracts
Social Hour with Dinner
7:00–8:00 PM
Posters
7:30–10:00 PM
Thursday, January 28, 2027
Breakfast
7:00–8:00 AM
Redefining Editors: From CRISPR Biology to Recombination and Integration
8:00–11:00 AM
Jason D. Fontenot, Stylus Medicine
Talk Title to be Announced
Talk Title to be Announced
Sangsu Bae, Seoul National University
Precise ADAR Base Editors and Prime Editor Enhancers
Precise ADAR Base Editors and Prime Editor Enhancers
Ying Zhang, Wuhan University
Talk Title to be Announced
Talk Title to be Announced
Short Talk(s) Chosen from Abstracts
Coffee Break
9:00–9:20 AM
On Own for Lunch
11:00–2:30 PM
Symposia Spotlight 2
2:30–4:30 PM
Short Talks Chosen from Abstracts
Coffee Available
4:30–5:00 PM
In vivo Genome Editing via Non-Viral Delivery (Joint)
5:00–6:00 PM
Birgit C Schultes, Intellia Therapeutics Inc.
mRNA/LNP based Gene Editing
mRNA/LNP based Gene Editing
Rebecca Ahrens-Nicklas, Children's Hospital of Philadelphia
Personalized Therapeutic Genome Editing for Hepatic Inborn Errors of Metabolism
Personalized Therapeutic Genome Editing for Hepatic Inborn Errors of Metabolism
Closing Keynote Address (Joint)
6:00–6:50 PM
Eric N. Olson, University of Texas Southwestern Medical Center
From Mutations to Medicines: The Journey to Genetic Cures for Muscle and Heart Disease
From Mutations to Medicines: The Journey to Genetic Cures for Muscle and Heart Disease
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
6:50–7:00 PM
Social Hour with Dinner
7:00–8:15 PM
Cash Bar
8:00–11:00 PM
Entertainment
8:00–11:00 PM
Friday, January 29, 2027
Departure
12:00–11:59 PM
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