Precision Genome Engineering: Translating the Human Genome to the Clinic
Mar 03–06, 2025 | INEC Killarney Convention Centre, Killarney, KY, Ireland
Scientific Organizers:
Fyodor D. Urnov, Claire Booth, and Laralynne Przybyla
In Person
On Demand
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Mar 03–06, 2025 | INEC Killarney Convention Centre, Killarney, KY, Ireland
Scientific Organizers: Fyodor D. Urnov, Claire Booth, and Laralynne Przybyla
Fyodor D. Urnov
Claire Booth
Laralynne Przybyla
Available Formats:
Supported by:
In Person
On Demand
***Meeting program subject to change.
Available Formats: = In Person = On DemandMonday, March 3, 2025
Fundraising
Booking Function
Merchandise Options
Registration Options
Registration
4:00–8:00 PM
INEC Foyer
Welcome Mixer and Dinner
6:00–8:00 PM
INEC Foyer
Tuesday, March 4, 2025
Breakfast
7:00–8:00 AM
Individual Hotel
Welcome and Keynote Address
8:00–9:00 AM
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
Megabase-scale Loss of Heterozygosity Provoked by CRISPR-Cas9 DNA Double-strand Breaks
From Genomes to Targets I: Uncovering New Biology for Target Identification
9:00–11:30 AM
INEC Main Auditorium
Helen Bateup, University of California, Berkeley
Towards CRISPR Neurotherapetics via Human Brain Organoid Functional Genomics
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
Dissecting Mechanisms of Human Disease via ex vivo Next-Generation Functional Genomics
Charles Gersbach, Duke University
Decoding and Programming the Regulatory Genome
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
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
Short Talk: Probe-Based Single-Cell Screening of Synthetic Protein and Genetic Perturbations for Cell Engineering
Coffee Break
9:30–9:50 AM
INEC Foyer
Poster Setup
11:30–1:00 PM
INEC Foyer
On Own for Lunch
11:30–5:00 PM
Poster Viewing
1:00–10:00 PM
INEC Foyer
Symposia Spotlight: Late-breaking research presentations selected from abstract submissions
3:00–4:30 PM
INEC Main Auditorium
* Jennifer E. Adair, Fred Hutchinson Cancer Research Center
Session Chair
Session Chair
* Jason Swinderman, UCSF
Session Chair
Session Chair
Peter Cameron, Profluent Bio
Deep Learning Prediction and Customization of CRISPR-Cas PAMs
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
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
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
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
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
Highly Efficient In Vivo Editing of Neurons Via Peptide-Mediated Delivery of CRISPR Enzymes
Coffee Available
4:30–5:00 PM
INEC Foyer
From Genomes to Targets II: Technologies to Enable New Therapeutic Areas
5:00–7:00 PM
INEC Main Auditorium
* Emma Chory, Duke University
High-Throughput Approaches to Uncover Synergistic Drug Combinations: Automating CRISPR and Beyond
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
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
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
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
Short Talk: In Vivo CRISPRa Screens Uncover Positive and Negative Regulators of T Cell Accumulation in the Immunocompetent Tumor Microenvironment
Social Hour with Lite Bites
7:00–8:00 PM
INEC Foyer
Poster Session 1
7:30–10:00 PM
INEC Foyer
Wednesday, March 5, 2025
Breakfast
7:00–8:00 AM
Individual Hotel
From Target to IND I: Preclinical Studies for ex vivo-Manufactured CRISPR Therapies
8:00–11:00 AM
INEC Main Auditorium
Annarita Miccio, Imagine Institute and Inserm
Editing Approaches to beta-Hemoglobinopathies and their Impact on Hematopoietic Stem Cells
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
Engineering CRISPR Therapeutics for N=1 Genetic Disease
Toni Cathomen, University Medical Center Freiburg
HLH or Other Immune Deficiency Indications
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
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
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
Short Talk: Base Editing Of Trinucleotide Repeats Mitigates Repeat Expansions In TNR Disease Models
Coffee Break
9:00–9:20 AM
INEC Foyer
Award Recipient Acknowledgement
9:20–9:25 AM
INEC Main Auditorium
Poster Setup
11:00–1:00 PM
INEC Foyer
On Own for Lunch
11:00–5:00 PM
Poster Viewing
1:00–10:00 PM
INEC Foyer
Regulatory Innovation and Harmonization to Expand Access to Precision Gene Therapies (Panel Discussion)
2:30–4:15 PM
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
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
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
Panel Moderator
Ana Hidalgo-Simon, Leiden University
Panelist
Panelist
Patrick Celis, Committee for Advanced Therapies (CAT) at European Medicines Agency
Panelist
Panelist
Julian Beach, Medicines and Healthcare products Regulatory Agency
Panelist
Panelist
Coffee Available
4:30–5:00 PM
INEC Foyer
From Target to IND II: Preclinical Studies for in vivo Therapies
5:00–7:00 PM
INEC Main Auditorium
* Pietro Genovese, Boston Children's Hospital
Epitope Editing for an Immunotherapy “Stealth” Hematopoiesis
Epitope Editing for an Immunotherapy “Stealth” Hematopoiesis
Cecilia Cotta-Ramusino, Tessera Therapeutics
Writing DNA with RNA: Genome Engineering by Target-Primed Reverse Transcription
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
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
Short Talk: A Nonviral CRISPR Platform to Edit Pathogenic Mutant Alleles in The Retina
Social Hour with Lite Bites
7:00–8:00 PM
INEC Foyer
Poster Session 2
7:30–10:00 PM
INEC Foyer
Thursday, March 6, 2025
Breakfast
7:00–8:00 AM
Individual Hotel
Genome Engineering in the Clinic I: Advances in Cancer Therapy
8:00–10:30 AM
INEC Main Auditorium
* Tirtha Chakraborty, Vor Biopharma
Genome Engineered Hematopoietic Transplants for the Treatment of AML
Genome Engineered Hematopoietic Transplants for the Treatment of AML
Waseem Qasim, University in London
Advanced Immunotherapy Through Clinical Genome Editing
Advanced Immunotherapy Through Clinical Genome Editing
Olubunmi O Afonja, Editas Medicine
Targeted HSC In Vivo Editing Strategies Leveraging Ex Vivo Experience
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
Efficient and Liver-Detargeted In Vivo Multiplex Gene Editing of Human HSPCs
Coffee Break
9:00–9:20 AM
INEC Foyer
On Own for Lunch
11:00–5:00 PM
Career Roundtable
3:30–4:30 PM
INEC Foyer
Laralynne Przybyla, Arena BioWorks
PRO Chair Disease Mechanisms and Models
PRO Chair Disease Mechanisms and Models
Helen Bateup, University of California, Berkeley
Associate Professor
Associate Professor
Jason D Fernandes, Scribe Therapeutics
Principal Scientist
Principal Scientist
Alex Eccleston, Springer Nature
Senior Editor
Senior Editor
Coffee Available
4:30–5:00 PM
INEC Foyer
Genome Engineering in the Clinic II: Advances in Therapies for Genetic Disease
5:00–7:00 PM
INEC Main Auditorium
* Claire Booth, University College London
From Gene Therapy to Genome Editing for Inborn Errors of Immunity
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
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
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
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
Short Talk: A Precision Gene Engineered B Cell Medicine Producing Sustained Levels of Active Factor IX for Hemophilia B Therapy
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
7:00–7:15 PM
INEC Main Auditorium
Social Hour with Lite Bites
7:15–8:00 PM
INEC Foyer
Cash Bar
8:00–9:00 PM
INEC Foyer
Trivia
8:00–9:00 PM
INEC Foyer
Friday, March 7, 2025
Departure
12:00–11:59 PM
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