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
Mar 03–06, 2025 | INEC Killarney Convention Centre, Killarney, KY, Ireland
Scientific Organizers:
Fyodor D. Urnov, Claire Booth and Laralynne Przybyla
Available Formats:
In Person
On Demand
Available Formats: = In Person = On Demand
Monday, March 3, 2025
Fundraising
Booking Function
Merchandise Options
Registration Options
Registration
4:00–8:00 PM
Welcome Mixer
6:00–8:00 PM
Tuesday, March 4, 2025
Breakfast
7:00–8:00 AM
Welcome and Keynote Address
8:00–9:00 AM
Laura Sepp-Lorenzino, Intellia Therapeutics
The LNP-mRNA Platform for in vivo CRISPR Therapies
The LNP-mRNA Platform for in vivo CRISPR Therapies
From Genomes to Targets I: Uncovering New Biology for Target Identification
9:00–11:15 AM
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
Short Talk(s) Chosen from Abstracts
Coffee Break
9:30–9:50 AM
Poster Setup
11:15–1:00 PM
On Own for Lunch
11:15–5:00 PM
Poster Viewing
1:00–10:00 PM
Symposia Spotlight: Late-breaking research presentations selected from abstract submissions
2:30–4:30 PM
Short Talk(s) Chosen from Abstracts
Coffee Available
4:30–5:00 PM
From Genomes to Targets II: Technologies to Enable New Therapeutic Areas
5:00–7:00 PM
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 Cells in vivo
Nonviral CRISPR Editing of Hematopoietic Cells in vivo
Short Talk(s) Chosen from Abstracts
Social Hour with Lite Bites
7:00–8:00 PM
Posters
7:30–10:00 PM
Wednesday, March 5, 2025
Breakfast
7:00–8:00 AM
From Target to IND I: Preclinical Studies for ex vivo-Manufactured CRISPR Therapies
8:00–11:00 AM
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
Short Talk(s) Chosen from Abstracts
Coffee Break
9:00–9:20 AM
Poster Setup
11:00–1:00 PM
On Own for Lunch
11:00–5:00 PM
Career Roundtable
1:00–2:30 PM
Poster Viewing
1:00–10:00 PM
Health Equity Forum
3:00–4:30 PM
Coffee Available
4:30–5:00 PM
From Target to IND II: Preclinical Studies for in vivo Therapies
5:00–7:00 PM
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
Talk Title to be Announced
Talk Title to be Announced
Manda Arbab, Harvard University and Broad Institute
Precision Genome Editing for Treatment of Neurodegenerative Diseases
Precision Genome Editing for Treatment of Neurodegenerative Diseases
Short Talk(s) Chosen from Abstracts
Social Hour with Lite Bites
7:00–8:00 PM
Posters
7:30–10:00 PM
Thursday, March 6, 2025
Breakfast
7:00–8:00 AM
Genome Engineering in the Clinic I: Advances in Cancer Therapy
8:00–11:00 AM
Tirtha Chakraborty, Vor Biopharma
Genome Engineered Hematopoietic Transplants for the Treatment of AML
Genome Engineered Hematopoietic Transplants for the Treatment of AML
Chiara Bonini, Vita-Salute San Raffaele University
CRISPR-Enabled Cancer Immunotherapies
CRISPR-Enabled Cancer Immunotherapies
Julianne Bruno, Editas
Talk Title to be Announced
Talk Title to be Announced
Speaker to be Announced
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
11:00–5:00 PM
Panel Discussion: Harmonizing the Regulatory Landscape
2:30–4:30 PM
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
Coffee Available
4:30–5:00 PM
Genome Engineering in the Clinic II: Advances in Therapies for Genetic Disease
5:00–6:45 PM
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
Short Talk(s) Chosen from Abstracts
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
6:45–7:00 PM
Social Hour with Lite Bites
7:00–8:00 PM
Cash Bar
8:00–11:00 PM
Entertainment
8:00–11:00 PM
Friday, March 7, 2025
Departure
12:00–11:59 PM
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