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
×
-
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
Juan E Hurtado Koolik †, Azalea Therapeutics
Short Talk: Cell-Specific and Genomic Site-Specific Engineering Enables Safe and Potent In Vivo TRAC-CAR T Cell Generation in Mice and Non-Human Primates
Short Talk: Cell-Specific and Genomic Site-Specific Engineering Enables Safe and Potent In Vivo TRAC-CAR T Cell Generation in Mice and Non-Human Primates
Short Talk(s) Chosen from Abstracts
Coffee Break
9:30–9:50 AM
Jessica Langer, Helmsley Trust Introduction
11:30–11:35 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
Pratik Koppikar †, University of Cambridge
Short Talk: Scalable Endogenous Tagging by Pooled Prime Editing for Characterizing Protein Localization
Short Talk: Scalable Endogenous Tagging by Pooled Prime Editing for Characterizing Protein Localization
Jeff Hussmann †, IGI
Short Talk: PMS1-dependent Base Excision Repair is a Cellular Determinant of Prime Editing Outcomes
Short Talk: PMS1-dependent Base Excision Repair is a Cellular Determinant of Prime Editing Outcomes
Rachel Anderson †, UC San Diego
Short Talk: Elucidating the Genetic Mechanisms of Adenine Base Editing Using a Fluorescence-Based Crispri Screen
Short Talk: Elucidating the Genetic Mechanisms of Adenine Base Editing Using a Fluorescence-Based Crispri Screen
Chen Chang †, UT Southwestern Medical Center
Short Talk: Parallel Activation and Interference CRISPR (PAIR) with Sequencing Uncovers DNA Repair Networks Guiding Precision Cell Engineering
Short Talk: Parallel Activation and Interference CRISPR (PAIR) with Sequencing Uncovers DNA Repair Networks Guiding Precision Cell Engineering
Guy Bedford †, Rice Univeristy
Short Talk: Induction of Hysteretic Chromatin States by Crispra Reveals Locus-Specific Encoding of Transcriptional Memory
Short Talk: Induction of Hysteretic Chromatin States by Crispra Reveals Locus-Specific Encoding of Transcriptional Memory
Junming Qian †, Columbia University
Short Talk: Allele-specific and Mutation-agnostic Silencing of Pathological Alleles by CRISPR-IMPRINT
Short Talk: Allele-specific and Mutation-agnostic Silencing of Pathological Alleles by CRISPR-IMPRINT
Garrett R Rettig †, Integrated DNA Technologies
Short Talk: A Programmable UNA Guide Architecture Improves CRISPR Genome-Editing Specificity Across Genomic Loci
Short Talk: A Programmable UNA Guide Architecture Improves CRISPR Genome-Editing Specificity Across Genomic Loci
Kiril Poukalov †, UT Southwestern Medical Center
Short Talk: Halo-LNP Protein Conjugation for Extrahepatic Targeting of Lipid Nanoparticles
Short Talk: Halo-LNP Protein Conjugation for Extrahepatic Targeting of Lipid Nanoparticles
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
Aamir Mir, CRISPR Therapeutics
Talk Title to be Announced
Talk Title 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
Ciro Bonetti †, Regeneron Pharmaceuticals
Short Talk: Enabling CRISPR-Mediated Homology-Directed Repair in Somatic Cells In Vivo
Short Talk: Enabling CRISPR-Mediated Homology-Directed Repair in Somatic Cells In Vivo
Tyler Daniel †, University of Pennsylvania
Short Talk: Precision A3G Base Editors for Disease Modeling and Correction
Short Talk: Precision A3G Base Editors for Disease Modeling and Correction
Social Hour with Dinner
7:00–8:00 PM
Poster Session 1
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
Kaiyi Jiang, Princeton’s Omenn-Darling Bioengineering Institute
Programmable Biology: Designing Molecular Medicine with Generative AI
Programmable Biology: Designing Molecular Medicine with Generative AI
Sherry Gao †, Genentech Inc
Talk Title to be Announced
Talk Title to be Announced
Hailey Wallace †, St. Jude Children’s Research Hospital
Short Talk: Computationally Designed New-to-Nature Tools for Programmable DNA Integration
Short Talk: Computationally Designed New-to-Nature Tools for Programmable DNA Integration
Matthew H Bakalar †, Basecamp Research
Short Talk: In Vivo Generation of CAR-T Cells by Targeted LNP Delivery of AI-Designed Large Serine Recombinases
Short Talk: In Vivo Generation of CAR-T Cells by Targeted LNP Delivery of AI-Designed Large Serine Recombinases
Coffee Break
9:00–9:20 AM
Poster Setup
11:00–1:00 PM
On Own for Lunch
11:00–3:30 PM
Poster Viewing
1:00–10:00 PM
Career Roundtable (Joint)
3:30–4:30 PM
Coffee Available
4:30–5:00 PM
Editing with Confidence: Evaluating Safety, Specificity, and Genomic Integrity
5:00–7:00 PM
Sarah Denny, Scribe Therapeutics
Purpose-Built Genome Editing for Therapeutic Impact
Purpose-Built Genome Editing for Therapeutic Impact
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
Amanda Haupt †, Revvity
Short Talk: Cas-Mediated Deaminase Recruitment Drives Sequence-Independent Off-Target Editing and Informs the Design of High Specificity Modular Base Editors
Short Talk: Cas-Mediated Deaminase Recruitment Drives Sequence-Independent Off-Target Editing and Informs the Design of High Specificity Modular Base Editors
Graham McVicker †, Salk Institute for Biological Studies
Short Talk: Joint Single-Cell Capture of Cas9 Edits and Transcriptomes Reveals Regulatory Effects of On- and Off-Target Editing
Short Talk: Joint Single-Cell Capture of Cas9 Edits and Transcriptomes Reveals Regulatory Effects of On- and Off-Target Editing
Social Hour with Dinner
7:00–8:00 PM
Poster Session 2
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
Gregory J Cost †, Addition Therapeutics
Short Talk: recise, RNA-mediated Insertion of Transgenes Using an R2 Retrotransposase Yields Stable, Durable, and Therapeutic-Level Hepatic Protein Expression, from Mice to NHPs
Short Talk: recise, RNA-mediated Insertion of Transgenes Using an R2 Retrotransposase Yields Stable, Durable, and Therapeutic-Level Hepatic Protein Expression, from Mice to NHPs
Heewhan Shin †, University of Chicago
Short Talk: Exploring New Dimensions of Large Serine Integrase/RDF Systems
Short Talk: Exploring New Dimensions of Large Serine Integrase/RDF Systems
Jesse Kennedy †, Adelaide University
Short Talk: Reincarnation: Mutation Agnostic Knock-Out and Precise Endogenous Replacement with Novel Prime Integrase Editing Tools
Short Talk: Reincarnation: Mutation Agnostic Knock-Out and Precise Endogenous Replacement with Novel Prime Integrase Editing Tools
Samagya Banskota †, Boston University
Short Talk: Engineering Next-Generation Virus-Like Particles for Efficient and Targeted In Vivo Gene Editing
Short Talk: Engineering Next-Generation Virus-Like Particles for Efficient and Targeted In Vivo Gene Editing
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
Lauren Testa †, Innovative Genomics Institute
Short Talk: Development of a Bespoke Adenine Base Editing Therapy for Citrullinemia Type I: Lessons Learned on the Path to the Clinic
Short Talk: Development of a Bespoke Adenine Base Editing Therapy for Citrullinemia Type I: Lessons Learned on the Path to the Clinic
Jackson H Bauer †, University of Pennsylvania
Short Talk: In Utero Gene Editing Corrects the Most Common Non-Deletional Mutation In Α-Thalassemia
Short Talk: In Utero Gene Editing Corrects the Most Common Non-Deletional Mutation In Α-Thalassemia
Quinn T Cowan †, Gladstone Institutes
Short Talk: Mechanisms of Allele-Specific Inactivation to Treat Dominant Disease in Neurons
Short Talk: Mechanisms of Allele-Specific Inactivation to Treat Dominant Disease in Neurons
Tianyi Ye †, Boston Children's Hospital
Short Talk: Durable and Reversible Epigenome Editing of Human Hematopoietic Stem Cells Modulates Platelet Function
Short Talk: Durable and Reversible Epigenome Editing of Human Hematopoietic Stem Cells Modulates Platelet Function
Zaneta Matuszek †, Harvard University
Short Talk: Precise Correction of Pathogenic Repeat Expansions with Prime Editing Ameliorates Disease Phenotypes in Trinucleotide Repeat Disorders
Short Talk: Precise Correction of Pathogenic Repeat Expansions with Prime Editing Ameliorates Disease Phenotypes in Trinucleotide Repeat Disorders
Huanyu Zhou †, Tenaya Therapeutics
Short Talk: Development of TN-501, an AAV-Delivered Gene Editing Therapy for PLN-R14del Cardiomyopathy
Short Talk: Development of TN-501, an AAV-Delivered Gene Editing Therapy for PLN-R14del Cardiomyopathy
Xin "Daniel" Gao †, University of Pittsburgh
Short Talk: Developing Precision Genome Editing for the Treatment of Peroxisomal Disorders
Short Talk: Developing Precision Genome Editing for the Treatment of Peroxisomal Disorders
Juan J Gallardo Pinera †, Washington University in St. Louis
Short Talk: A One-Time Base Editing Therapy for Wolfram Syndrome
Short Talk: A One-Time Base Editing Therapy for Wolfram Syndrome
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
Subscribe for Updates