Mar 03–06, 2025 | INEC Killarney Convention Centre, Killarney, KY, Ireland
Scientific Organizers:
Fyodor D. Urnov, Claire Booth, and Laralynne Przybyla

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Scientific Organizers: Fyodor D. Urnov, Claire Booth, and Laralynne Przybyla
Fyodor D. Urnov
Claire Booth
Laralynne Przybyla
***Meeting program subject to change.
Available Formats: = In Person = On DemandMonday, March 3, 2025
Fundraising
Booking Function
Merchandise Options
Registration Options
Tuesday, March 4, 2025
Megabase-scale Loss of Heterozygosity Provoked by CRISPR-Cas9 DNA Double-strand Breaks
Towards CRISPR Neurotherapetics via Human Brain Organoid Functional Genomics
Dissecting Mechanisms of Human Disease via ex vivo Next-Generation Functional Genomics
Decoding and Programming the Regulatory Genome
Short Talk: Arrayed CRISPR Screening in Intestinal Epithelial Organoids for Target Identification in Inflammatory Bowel Disease
Short Talk: Probe-Based Single-Cell Screening of Synthetic Protein and Genetic Perturbations for Cell Engineering
Session Chair
Session Chair
Deep Learning Prediction and Customization of CRISPR-Cas PAMs
CRISPR/hfCas12Max DNA-Editing Therapy Restores Muscle Function in Duchenne Muscular Dystrophy: From Bench to Bedside
CHANGE-Seq-R Massively Parallel Biochemical Assay Paired with Machine Learning to Define Genetic Features Impacting Genome-Wide Activity of CRISPR Editors
Engineering of Next Generation Epigenetic Editors for Enhanced Durable Repression in Non-Human Primates
Functional Restoration of Immune Defects in STAT1 Gain-of-Function Immunodeficiency Following Gene Editing
Highly Efficient In Vivo Editing of Neurons Via Peptide-Mediated Delivery of CRISPR Enzymes
High-Throughput Approaches to Uncover Synergistic Drug Combinations: Automating CRISPR and Beyond
The Genetic Landscape of Human DNA Repair: Implications for CRISPR Therapies
Nonviral CRISPR Editing of Hematopoietic Stem Cells
Short Talk: In Vivo Editing of Lung Stem Cells for Durable Gene Correction in Mice
Short Talk: In Vivo CRISPRa Screens Uncover Positive and Negative Regulators of T Cell Accumulation in the Immunocompetent Tumor Microenvironment
Wednesday, March 5, 2025
Editing Approaches to beta-Hemoglobinopathies and their Impact on Hematopoietic Stem Cells
Engineering CRISPR Therapeutics for N=1 Genetic Disease
HLH or Other Immune Deficiency Indications
Short Talk: Precision Epigenome Engineering to Improve T Cell Therapy
Short Talk: Epigenetic Reprogramming of Tumor-Infiltrating Lymphocytes for Improved Anti-Tumor Killing
Short Talk: Base Editing Of Trinucleotide Repeats Mitigates Repeat Expansions In TNR Disease Models
How to Enable Participation of the Most Neglected and Why this will give your Technology an Edge
The Tractable Nonclinical, CMC, and Regulatory Challenges of Engineering a Bespoke Gene Editor On Demand for a Newborn with Two Months to Live
Panel Moderator
Panelist
Panelist
Panelist
Epitope Editing for an Immunotherapy “Stealth” Hematopoiesis
Writing DNA with RNA: Genome Engineering by Target-Primed Reverse Transcription
Developing Bespoke Gene Editing Treatments for Patients With Carbamoyl Phosphate Synthetase 1 (CPS1) Deficiency in Real Time: Successes and Challenges
Short Talk: A Nonviral CRISPR Platform to Edit Pathogenic Mutant Alleles in The Retina
Thursday, March 6, 2025
Genome Engineered Hematopoietic Transplants for the Treatment of AML
Advanced Immunotherapy Through Clinical Genome Editing
Targeted HSC In Vivo Editing Strategies Leveraging Ex Vivo Experience
Efficient and Liver-Detargeted In Vivo Multiplex Gene Editing of Human HSPCs
PRO Chair Disease Mechanisms and Models
Associate Professor
Principal Scientist
Senior Editor
From Gene Therapy to Genome Editing for Inborn Errors of Immunity
Towards a Platform for In Vivo Corrective Editing of Ultra-Rare and N-of-1 Metabolic Disorders
Single-Course in Vivo Gene Editing Treatments for Cardiovascular Disease
Short Talk: First in Human Trial of Prime Editing to Treat p47-phox Chronic Granulomatous Disease
Short Talk: A Precision Gene Engineered B Cell Medicine Producing Sustained Levels of Active Factor IX for Hemophilia B Therapy
Friday, March 7, 2025
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