Meeting at a Glance

May 16-20, 2023  |  Los Angeles, CA

The American Society of Gene and Cell Therapy’s (ASGCT) Annual Meeting is the premier event for gene and cell therapy professionals. It’s the best place for people in the field to learn from the latest scientific research and stay up to date on new technologies. Join SCGE researches for a week of workshops, poster sessions, oral abstracts, and scientific symposiums.

Visit us at Booth #863 

  • Meet with NIH Program representatives
  • Demo the SCGE Toolkit
  • See the achievements of Phase 1 
  • Learn about the focus of Phase 2
  • Hear out about the NIH TARGETED Challenge

SCGE Scientific Symposium

Friday, May 19th  |  10:15 AM – 12:00 PM  |  West Hall B

This symposium will highlight some of the most exciting science and successful facets of program design of the SCGE program. This session will demonstrate the facets of this program that are quintessential to all Common Fund programs: it’s transformative, catalytic, synergistic, cross-cutting, and unique features. The proposed speakers were selected for their unparalleled expertise, deep engagement with the SCGE program opportunities, and the broad relevance of their anticipated presentation to the ASGCT membership.

Session Details

SpeakerSymposium TopicTime
Felicia Qashu, PhD, NIH/ODSCGE Initiatives Introduction10:15 AM
Charles A. Gersbach, PhD, Duke UniversityTechnologies to Facilitate Translation of Somatic Cell Editing10:30 AM
Ross Wilson, PhD, University of California BerkeleyDelivery Initiative Highlights10:45 AM
Shunqing Liang, PhD, University of Massachusetts Medical SchoolDevelop combinatorial non-viral and viral CRISPR delivery for lung diseases11:00 AM
Cathleen Lutz, PhD, The Jackson Laboratory Animal Initiative Highlights11:15 AM
Bruce R. Conklin, MD, Gladstone Institute of Cardiovascular MedicineBiological Initiative Highlight11:30 AM
Marrah Lachowicz-Scroggins, PhD, NIH/NHLBIPhase 2 of the NIH's Somatic Cell Genome Editing Program11:45 AM

SCGE Member Abstracts

Wednesday, May 17th

PresenterAbstract TitleCategorySession TitleTimeLocation
Kathy SnowDevelopment and Characterization of New Reporter Mouse Models for In Vivo Gene EditingPoster SessionWednesday Poster Session12:00 PMPoster #504
Karthikeyan PonnienselvanReducing the Inherent Auto-Inhibitory Interaction within the pegRNA Enhances Prime Editing EfficiencyPoster Session Wednesday Poster Session12:00 PMPoster #523
Samantha MaraghOutcomes from the First NIST Genome Editing Consortium Interlab StudyPoster Session Wednesday Poster Session12:00 PMPoster #533
Nicole PaulkAAV Immuno-Gene Therapy Delivers Vectorized Cytokines to Effectively Treat High-Grade GliomasOral Abstract SessionPreclinical and Proof-of-Concept In Vivo Studies I3:45 PMRoom 408 AB
Cicera LazzarottoCHANGE-seq-BE Enables Sensitive and Unbiased Genome-Wide Profiling of Adenine Base Editors In VitroOral Abstract SessionGenome Editing Therapies & Safety I4:15 PMRoom 515 AB
Alice TarantalMonitoring Fetal and Infant Somatic Cell Genome Editing in Rhesus Monkeys with Total-Body PETOral Abstract SessionAAV Vectors: Clinical and Non-Human Primate Studies4:30 PMRoom 411
Alice TarantalAAV Serotype Tropism and Editing in Young Rhesus MonkeysOral Abstract SessionAAV Vectors: Clinical and Non-Human Primate Studies4:45 PMRoom 411
Wan DuShuttle Peptides-Mediated Local RNP Delivery of Editing Complex in the Sensory Organs of the Inner Ear and Retina In Vivo in Adult MiceOral Abstract SessionSynthetic/Molecular Conjugates and Physical Methods for Delivery of Gene Therapeutics I4:45 PMPetree Hall C

Thursday, May 18th

PresenterAbstract TitleCategorySession TitleTimeLocation
Jinghua LiurAAV-Delivered Hepatocyte-Specific Expression of miR-375 Protects Against the Acetaminophen-Induced Acute Liver Failure in MicePoster SessionThursday Poster Session12:00 PMPoster #915
Alexandra Piotrowski-DaspitGene Editing in the Lung Using Polymeric VehiclesPoster SessionThursday Poster Session12:00 PMPoster #977
Krishanu SahaSingle-Cell Transcriptional Analysis of Human In Vitro Platforms to Study Genome Editing Effects in Retinal TissuesPoster SessionThursday Poster Session12:00 PMPoster #1016
Santiago R. CastilloUnconstrained Mitochondrial DNA Base Editing Enables Precise Disease ModelingPoster SessionThursday Poster Session12:00 PMPoster #1061
Jarryd CampbellCRISPR-Activated Biomedical Reporter Pigs for In Vivo Genome Editing Pre-Clinical StudiesPoster SessionThursday Poster Session12:00 PMPoster #1084
Krishanu SahaControlling CRISPR-Cas9 Genome Editing in Human Cells Using a Small-Molecule Inducible Degradation StrategyPoster SessionThursday Poster Session12:00 PMPoster #1210
Sophia LiuNatural AAV2 Variants Demonstrate Stronger Neurotropism, Penetrance of the Blood-Brain Barrier, and Differential Transduction of Brain Regions Compared to AAV9Poster SessionThursday Poster Session12:00 PMPoster #1247
Yichao LiLarge-Scale GUIDE-seq-2 Profiling Reveals Effects of Human Genetic Variation on Cellular Off-Target Genome Editing ActivityOral Abstract SessionGenome Editing Therapies & Safety II2:45 PMConcourse Hall 152 & 153
Madeleine SittonModeling Gene Editing Outcomes in Microphysiological Human Tissue System Models of Duchenne Muscular DystrophyOral Abstract SessionGenome Editing Therapies & Safety II3:00 PMConcourse Hall 152 & 153
Katarina KulhankovaShuttle Peptide Delivers Base Editor RNPs to Rhesus Monkey Airway Epithelial Cells In VivoOral Abstract SessionGene Targeting and Gene Correction: New Technologies3:45 PMRoom 515 AB
Agnieszka CzechowiczLentiviral-Mediated Gene Therapy for Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical TrialsOral Abstract SessionHematologic and Immunologic Diseases4:30 PMRoom 501 AB
Jennifer HamiltonCell Type-Programmable CRISPR-Cas9 Delivery for Human T Cell EngineeringOral Abstract SessionGene Targeting and Gene Correction: New Technologies4:45 PMRoom 515 AB
Victor Van LaarDevelopment of a Monoparticle-Based CRISPR Gene Editing and Intraparenchymal Delivery System for Neurological ApplicationsOral Abstract SessionSynthetic/Molecular Conjugates and Physical Methods for Delivery of Gene Therapeutics II5:15 PMPetree Hall C

Friday, May 19th

PresenterAbstract TitleCategorySession TitleTimeLocation
Benjamin DevermanEngineering AAVs to Target and Cross the Brain VasculatureScientific SymposiumNew Approaches in Cell and Gene Therapy for Cardiovascular Disease9:15 AMRoom 411
Marrah Lachowicz-Scroggins and Felicia QashuIntroduction to SCGE ConsortiumScientific SymposiumSomatic Cell Genome Editing (SCGE) Program10:15 AMWest Hall B
Charles GersbachTechnologies to Facilitate Translation of Somatic Cell EditingScientific SymposiumSomatic Cell Genome Editing (SCGE) Program10:30 AMWest Hall B
Ross WilsonDelivery Initiative Highlights (SCGE Consortium)Scientific SymposiumSomatic Cell Genome Editing (SCGE) Program10:45 AMWest Hall B
Shunqing LiangDevelop combinatorial non-viral and viral CRISPR delivery for lung diseases (SCGE Consortium)Scientific SymposiumSomatic Cell Genome Editing (SCGE) Program10:45 AMWest Hall B
Cathleen LutzAnimal Initiative HighlightScientific SymposiumSomatic Cell Genome Editing (SCGE) Program11:15 AMWest Hall B
Bruce ConklinBiological Initiative HighlightScientific SymposiumSomatic Cell Genome Editing (SCGE) Program11:30 AMWest Hall B
Marrah Lachowicz-ScrogginsPhase 2 of the NIH's Somatic Cell Genome Editing ProgramScientific SymposiumSomatic Cell Genome Editing (SCGE) Program11:45 AMWest Hall B
Chris WalkeyA Multi-Dimensional Survey of AAV Tropism in MicePoster SessionFriday Poster Session12:00 PMPoster #1409
John KellyDevelopment of a Highly Efficient Modular and Multiplexed CRISPR Editing System for Multimodal Tracking of CAR-T CellsPoster SessionFriday Poster Session12:00 PMPoster #1502
Scot WolfeEnhancing CRISPR Genome Editing by Extensive Chemical Modification and Self-Delivery of Guide RNAsPoster SessionFriday Poster Session12:00 PMPoster #1507
Alice TarantalTranslational Programs in Gene Therapy and Somatic Cell Genome Editing: Nonhuman Primate Research OpportunitiesPoster SessionFriday Poster Session12:00 PMPoster #1545
Bikash PattnaikValidating Reliability of Genome Editing Using Ion Channels as Drug TargetPoster SessionFriday Poster Session12:00 PMPoster #1619
Kirstan GimseTranscriptomic Profiling of the Mouse Hippocampus After Intracerebral Injection of Cas9 Nanocapsule Genome EditorsPoster SessionFriday Poster Session12:00 PMPoster #1653
Madalena CastroPeptide-Mediated Delivery of CRISPR Ribonucleoprotein (RNP) into Primary Human CD34+ Hematopoietic Stem CellsPoster SessionFriday Poster Session12:00 PMPoster #1687
Cathleen LutzOptimizing Base Editing Strategies in a Mouse Model of Zellweger SyndromeOral Abstract SessionAAV Vectors - Preclinical and Proof-of-Concept In vivo Studies I4:30 PMRoom 408 AB
Varun KattaOptimization and Characterization of Genome Editing of Human Hematopoietic Stem Cells to Induce Fetal Hemoglobin towards SAGES-1 Clinical TrialOral Abstract SessionGene Targeting and Gene Correction: Hemoglobin, Muscle, and Eye5:00 PMRoom 515 AB

Saturday, May 20th

PresenterAbstract TitleCategorySession TitleTimeLocation
Joseph Muldoon Efficient and Minimally Perturbative CAR-T Cell Engineering Using Peptide-Enabled CRISPR RNP DeliveryOral Abstract SessionCell Therapy Product Manufacturing8:00 AMPetree Hall D
Alan RosalesEvolving Nephrotropic AAV Variants Using Ex Vivo NHP Kidney Perfusion and Human Kidney OrganoidsOral Abstract SessionAAV Development for Eye, Muscle, Kidney and CNS9:15 AMConcourse Hall 150 & 151
Alexandra Piotrowski-DaspitPolymeric nanoparticle strategies to restore CFTR functionScientific SymposiumStrategies to Deliver Genetic Therapies for Cystic Fibrosis11:15 AMRoom 502 AB