Precision Genome Engineering: Translating the Human Genome to the Clinic (C1)
March 3-6, 2025  | INEC Killarney Convention Centre, Killarney, KY, Ireland
Fyodor D. Urnov, Claire Booth and Laralynne Przybyla
Scholarship Deadline: Dec. 18, 2024 | Abstract Deadline: Feb. 11, 2025 | Early Registration Deadline: Jan. 15, 2025
* Session Chair † Invited but not yet accepted | Program current as of September 12, 2025 8 AM | For the most up-to-date details, visit https://www.keystonesymposia.org
Monday, March 3, 2025
4:00–8:00 PM Registration INEC Foyer
6:00–8:00 PM Welcome Mixer and Dinner INEC Foyer
Tuesday, March 4, 2025
7:00–8:00 AM Breakfast Individual Hotel
8:00–9:00 AM Welcome and Keynote Address INEC Main Auditorium
  * Claire Booth, University College London
 
  * Fyodor D. Urnov, University of California, Berkeley
 
  * Laralynne Przybyla, Arena BioWorks
 
  Maria Jasin, Memorial Sloan Kettering Cancer Center
Megabase-scale Loss of Heterozygosity Provoked by CRISPR-Cas9 DNA Double-strand Breaks
 
9:00–11:30 AM From Genomes to Targets I: Uncovering New Biology for Target Identification INEC Main Auditorium
  Helen Bateup, University of California, Berkeley
Towards CRISPR Neurotherapetics via Human Brain Organoid Functional Genomics
 
  * Laralynne Przybyla, Arena BioWorks
Dissecting Mechanisms of Human Disease via ex vivo Next-Generation Functional Genomics
 
  Charles Gersbach, Duke University
Decoding and Programming the Regulatory Genome
 
  Erica Bello, Milner Therapeutics Institute
Short Talk: Arrayed CRISPR Screening in Intestinal Epithelial Organoids for Target Identification in Inflammatory Bowel Disease
 
  Jason Swinderman, UCSF
Short Talk: Probe-Based Single-Cell Screening of Synthetic Protein and Genetic Perturbations for Cell Engineering
 
9:30–9:50 AM Coffee Break INEC Foyer
11:30–1:00 PM Poster Setup INEC Foyer
11:30–5:00 PM On Own for Lunch
1:00–10:00 PM Poster Viewing INEC Foyer
3:00–4:30 PM Symposia Spotlight: Late-breaking research presentations selected from abstract submissions INEC Main Auditorium
  * Jennifer E. Adair, Fred Hutchinson Cancer Research Center
Session Chair
 
  * Jason Swinderman, UCSF
Session Chair
 
  Peter Cameron, Profluent Bio
Deep Learning Prediction and Customization of CRISPR-Cas PAMs
 
  T.J. Cradick, HuidaGene Therapeutics
CRISPR/hfCas12Max DNA-Editing Therapy Restores Muscle Function in Duchenne Muscular Dystrophy: From Bench to Bedside
 
  Ashley Flory, St. Jude Children's Research Hospital
CHANGE-Seq-R Massively Parallel Biochemical Assay Paired with Machine Learning to Define Genetic Features Impacting Genome-Wide Activity of CRISPR Editors
 
  Jason D Fernandes, Scribe Therapeutics
Engineering of Next Generation Epigenetic Editors for Enhanced Durable Repression in Non-Human Primates
 
  Robert Torrance, University College London
Functional Restoration of Immune Defects in STAT1 Gain-of-Function Immunodeficiency Following Gene Editing
 
  Ross Wilson, University of California, Berkeley
Highly Efficient In Vivo Editing of Neurons Via Peptide-Mediated Delivery of CRISPR Enzymes
 
4:30–5:00 PM Coffee Available INEC Foyer
5:00–7:00 PM From Genomes to Targets II: Technologies to Enable New Therapeutic Areas INEC Main Auditorium
  * Emma Chory, Duke University
High-Throughput Approaches to Uncover Synergistic Drug Combinations: Automating CRISPR and Beyond
 
  Britt Adamson, Princeton University
The Genetic Landscape of Human DNA Repair: Implications for CRISPR Therapies
 
  Jennifer E. Adair, Fred Hutchinson Cancer Research Center
Nonviral CRISPR Editing of Hematopoietic Stem Cells
 
  Yehui Sun, UT Southwestern Medical Center
Short Talk: In Vivo Editing of Lung Stem Cells for Durable Gene Correction in Mice
 
  Tori Yamamoto, Gladstone Institutes
Short Talk: In Vivo CRISPRa Screens Uncover Positive and Negative Regulators of T Cell Accumulation in the Immunocompetent Tumor Microenvironment
 
7:00–8:00 PM Social Hour with Lite Bites INEC Foyer
7:30–10:00 PM Poster Session 1 INEC Foyer
Wednesday, March 5, 2025
7:00–8:00 AM Breakfast Individual Hotel
8:00–11:00 AM From Target to IND I: Preclinical Studies for ex vivo-Manufactured CRISPR Therapies INEC Main Auditorium
  Annarita Miccio, Imagine Institute and Inserm
Editing Approaches to beta-Hemoglobinopathies and their Impact on Hematopoietic Stem Cells
 
  * Fyodor D. Urnov, University of California, Berkeley
Engineering CRISPR Therapeutics for N=1 Genetic Disease
 
  Toni Cathomen, University Medical Center Freiburg
HLH or Other Immune Deficiency Indications
 
  Kunal Rai, The University of Texas MD Anderson Cancer Center
Short Talk: Precision Epigenome Engineering to Improve T Cell Therapy
 
  Christian McRoberts Amador, Duke University
Short Talk: Epigenetic Reprogramming of Tumor-Infiltrating Lymphocytes for Improved Anti-Tumor Killing
 
  Zaneta Matuszek, Broad Institute of Harvard and MIT
Short Talk: Base Editing Of Trinucleotide Repeats Mitigates Repeat Expansions In TNR Disease Models
 
9:00–9:20 AM Coffee Break INEC Foyer
9:20–9:25 AM Award Recipient Acknowledgement INEC Main Auditorium
11:00–1:00 PM Poster Setup INEC Foyer
11:00–5:00 PM On Own for Lunch
1:00–10:00 PM Poster Viewing INEC Foyer
2:30–4:15 PM Regulatory Innovation and Harmonization to Expand Access to Precision Gene Therapies (Panel Discussion) INEC Main Auditorium
  * Claire Booth, University College London
 
  Jennifer E. Adair, Fred Hutchinson Cancer Research Center
How to Enable Participation of the Most Neglected and Why this will give your Technology an Edge
 
  Fyodor D. Urnov, University of California, Berkeley
The Tractable Nonclinical, CMC, and Regulatory Challenges of Engineering a Bespoke Gene Editor On Demand for a Newborn with Two Months to Live
 
  * Daniel O'Connor, The Association of the British Pharmaceutical Industry (ABPI)
Panel Moderator
 
  Ana Hidalgo-Simon, Leiden University
Panelist
 
  Patrick Celis, Committee for Advanced Therapies (CAT) at European Medicines Agency
Panelist
 
  Julian Beach, Medicines and Healthcare products Regulatory Agency
Panelist
 
4:30–5:00 PM Coffee Available INEC Foyer
5:00–7:00 PM From Target to IND II: Preclinical Studies for in vivo Therapies INEC Main Auditorium
  * Pietro Genovese, Boston Children's Hospital
Epitope Editing for an Immunotherapy “Stealth” Hematopoiesis
 
  Cecilia Cotta-Ramusino, Tessera Therapeutics
Writing DNA with RNA: Genome Engineering by Target-Primed Reverse Transcription
 
  Sarah Grandinette, University of Pennsylvania
Developing Bespoke Gene Editing Treatments for Patients With Carbamoyl Phosphate Synthetase 1 (CPS1) Deficiency in Real Time: Successes and Challenges
 
  Krishanu Saha, University of Wisconsin-Madison
Short Talk: A Nonviral CRISPR Platform to Edit Pathogenic Mutant Alleles in The Retina
 
7:00–8:00 PM Social Hour with Lite Bites INEC Foyer
7:30–10:00 PM Poster Session 2 INEC Foyer
Thursday, March 6, 2025
7:00–8:00 AM Breakfast Individual Hotel
8:00–10:30 AM Genome Engineering in the Clinic I: Advances in Cancer Therapy INEC Main Auditorium
  * Tirtha Chakraborty, Vor Biopharma
Genome Engineered Hematopoietic Transplants for the Treatment of AML
 
  Waseem Qasim, University in London
Advanced Immunotherapy Through Clinical Genome Editing
 
  Olubunmi O Afonja, Editas Medicine
Targeted HSC In Vivo Editing Strategies Leveraging Ex Vivo Experience
 
  Luca Biasco, nChroma Bio
Efficient and Liver-Detargeted In Vivo Multiplex Gene Editing of Human HSPCs
 
9:00–9:20 AM Coffee Break INEC Foyer
11:00–5:00 PM On Own for Lunch
3:30–4:30 PM Career Roundtable INEC Foyer
  Laralynne Przybyla, Arena BioWorks
PRO Chair Disease Mechanisms and Models
 
  Helen Bateup, University of California, Berkeley
Associate Professor
 
  Jason D Fernandes, Scribe Therapeutics
Principal Scientist
 
  Alex Eccleston, Springer Nature
Senior Editor
 
4:30–5:00 PM Coffee Available INEC Foyer
5:00–7:00 PM Genome Engineering in the Clinic II: Advances in Therapies for Genetic Disease INEC Main Auditorium
  * Claire Booth, University College London
From Gene Therapy to Genome Editing for Inborn Errors of Immunity
 
  Kiran Musunuru, University of Pennsylvania
Towards a Platform for In Vivo Corrective Editing of Ultra-Rare and N-of-1 Metabolic Disorders
 
  Amit Vikram Khera, Verve Therapeutics
Single-Course in Vivo Gene Editing Treatments for Cardiovascular Disease
 
  Mohammed Asmal, Prime Medicine
Short Talk: First in Human Trial of Prime Editing to Treat p47-phox Chronic Granulomatous Disease
 
  Timothy Mullen, Be Biopharma
Short Talk: A Precision Gene Engineered B Cell Medicine Producing Sustained Levels of Active Factor IX for Hemophilia B Therapy
 
7:00–7:15 PM Meeting Wrap-Up: Outcomes and Future Directions (Organizers) INEC Main Auditorium
7:15–8:00 PM Social Hour with Lite Bites INEC Foyer
8:00–9:00 PM Cash Bar INEC Foyer
8:00–9:00 PM Trivia INEC Foyer
Friday, March 7, 2025
12:00–11:59 PM Departure