4:00–8:00 PM | Registration | INEC Foyer |
6:00–8:00 PM | Welcome Mixer and Dinner | INEC Foyer |
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 |
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 |
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 |
12:00–11:59 PM | Departure |