The American Society of Gene and Cell Therapy’s (ASGCT) Annual Meeting is the premier event for professionals in gene and cell therapy to learn from the latest scientific research, stay up to date on new technologies, and make career-advancing connections with peers. Many members in the SCGE consortium attend and present at the event. Learn more about SCGE investigator involvement below:

Tuesday May 7th

LNP development and screening

James Dahlman

LNP development and screening

James Dahlman, Georgia Tech & Emory School of Medicine

9:25am | Ballroom 1

Neurologic Diseases I - Oral Abstract Session

Steve Gray

Neurologic Diseases I Oral Abstract Session – Co-chair 

Steve Gray, University of Texas Southwestern Medical Center

1:30pm | Ballroom 4

New Technologies for Gene Targeting and Gene Correction - Oral Abstract Session

David Segal
New Technologies for Gene Targeting and Gene Correction Oral Abstract Session – Co-chair 

David Segal, University of California Davis

1:30pm | Ballroom 1

Wednesday May 8th

Prospects for Prenatal Gene and Cell Therapy - Scientific Symposium

William Peranteau

Prospects for Prenatal Gene and Cell Therapy (Scientific Symposium) – Co-chair 

William Peranteau, Children’s Hospital of Philadelphia

8:00am | Room 307-308

Prenatal therapies – how far we’ve come and the challenges that remain

Alice Tarantal
Prenatal therapies – how far we’ve come and the challenges that remain

Alice Tarantal, University of California Davis

8:00am | Room 307-308

764: Engineered Ribonucleoprotiens (eRNPs) for Non-Viral In Vivo Delivery of Gene Editors to Treat Ophthalmic Disorders

Adam Silverman

764: Engineered Ribonucleoprotiens (eRNPs) for Non-Viral In Vivo Delivery of Gene Editors to Treat Ophthalmic Disorders

Poster Session

Adam Silverman, Spotlight Therapeutics (SCGE PI: Kris Saha)

12:00pm – 7:00pm | Exhibit Hall

679: Systematic Off-Target Analyses of Base-Editing Therapeutic Leads Show Reduced Off-Target Editing, Reduced Bystander Editing, and Increased On-Target Editing by Hybrid Guide RNAs

Xiao Wang

679: Systematic Off-Target Analyses of Base-Editing Therapeutic Leads Show Reduced Off-Target Editing, Reduced Bystander Editing, and Increased On-Target Editing by Hybrid Guide RNAs

Poster Session

Xiao Wang, University of Pennsylvania (SCGE PI: Peranteau, Musunuru)

12:00pm – 7:00pm | Exhibit Hall

120: Second Generation AAV Capsids Reprogrammed to Bind Human Transferrin Receptor are Targeted to the Brain and De-targeted from the Liver in Human TRFC Knock-in Mice

Ken Chan

120: Second Generation AAV Capsids Reprogrammed to Bind Human Transferrin Receptor are Targeted to the Brain and De-targeted from the Liver in Human TRFC Knock-in Mice

Ken Chan, The Broad Institute (SCGE PI: Ben Deverman)

4:45pm | Room 309-310

Thursday May 9th

2084: Hemophilia gene therapy follow up, factor 8 and 9, dose-response, efficacy, durability

Lindsey George

2084: Hemophilia gene therapy follow up, factor 8 and 9, dose-response, efficacy, durability

Lindsey George, Children’s Hospital of Philadelphia (SCGE PIs Peranteau and Musunuru)

8:00am | Room 307-308

2078: How Confident Are You? Expanded Results From The First NIST Genome Editing Consortium Interlab Study

Samantha Maragh

2078: How Confident Are You? Expanded Results From The First NIST Genome Editing Consortium Interlab Study

Samantha Maragh, NIST (SCGE PI Shengdar Tsai)

8:10am | Ballroom 2

Keynote

Beverly Davidson

Keynote

Beverly Davidson, Children’s Hospital of Philadelphia

10:25am | Hall A&B

1394: The Somatic Cell Genome Editing Consortium Toolkit

Mindy Dwinell

1394: The Somatic Cell Genome Editing Consortium Toolkit

Poster Session

Mindy Dwinell, Medical College of Wisconsin

12:00pm-7:00pm | Exhibit Hall

991: Human CA4-Targeting AAV Capsids Efficiently Transduce the CNS in Humanized Mice

Nuria R. Botticello-Romero

Nuria R. Botticello-Romero, The Broad Institute (SCGE PI Ben Deverman)

12:00-7:00pm | Exhibit Hall

169: In Vivo Correction of Metabolic Pathogenic Variants via Base Editing and Prime Editing: Toward the Equitable Treatment of Hepatic Inborn Errors of Metabolism

Xiao Wang

Xiao Wang, Children’s Hospital of Philadelphia (SCGE PIs Peranteau and Musunuru)

3:00pm | Room 307-308

Targeted Gene Insertion - Oral Abstract Session

Shengdar Tsai

Targeted Gene Insertion, Oral Abstract Session – Co-chair

Shengdar Tsai, St. Jude Children’s Research Hospital

3:45pm | Ballroom 3

CAR T-cell Therapies - Oral Abstract Session

Charles Gersbach

CAR T-cell Therapies, Oral Abstract Session – Co-chair

Charles Gersbach, Duke University

3:45pm | Room 307-308

220: Engineered Virus-Like Particles for Transient Delivery of Prime Editor Ribonucleoprotein Complexes in Vivo

Meirui An

Meirui An, The Broad Institute (SCGE PI David Liu)

4:00pm | Room 314-317

217: An AAV Capsid Programmed to Bind Human and Macaque Receptors Mediates Enhanced Gene Delivery to the CNS and Skeletal Muscle In Vivo

Jason Wu

Jason Wu, The Broad Institute (SCGE PI Ben Deverman)

5:00pm | Room 309-310

Friday May 10th

2120: Precision genome editing for treatment of genetic neurological diseases

Mandana Arbab

2120: Precision genome editing for treatment of genetic neurological diseases

Mandana Arbab, Boston Children’s Hospital, Harvard Medical School

8:26am | Ballroom 2

DNA Polymerase Editors (DPEs)

Erik Sontheimer

DNA Polymerase Editors (DPEs)

Erik Sontheimer, UMass Chan Medical School

8:52am | Ballroom 2

237: Potency Assay Enabling Both Ex Vivo and In Vivo Genome Editing Therapeutcis for Sickle Cell Disease

Utku Goreke

Utku Goreke, University of California, Berkeley (SCGE PIs Giannikopoulos, Gurkan, Nguyen)

9:00am | Room 339-342

1635: CRISPR Vision Program: Nonviral Genome Editing to Treat Congenital Blindness

Kris Saha

1635: CRISPR Vision Program: Nonviral Genome Editing to Treat Congenital Blindness

Poster Session

Kris Saha, University of Wisconsin-Madison

12:00pm-7:00pm | Exhibit Hall

1551: Engineering Human CNS Receptor-Targeted Capsids for Improved Antibody Evasion

Simon Pacouret
1551: Engineering Human CNS Receptor-Targeted Capsids for Improved Antibody Evasion

Poster Session

Simon Pacouret, The Broad Institute (SCGE PI Ben Deverman)

12:00pm-7:00pm | Exhibit Hall

1553: Benchmarking Natural and Synthetic AAV Capsids for Neutralizing Antibody Evasion

William Donahoe

1553: Benchmarking Natural and Synthetic AAV Capsids for Neutralizing Antibody Evasion

Poster Session

William Donahoe, The Broad Institute (SCGE PI Ben Deverman)

12:00pm-7:00pm | Exhibit Hall

Saturday May 11th

Unending Experiments: Ethical Challenges and Responsibilities for Gene Therapy’s Road Ahead (Scientific Symposium)

Kris Saha

Unending Experiments: Ethical Challenges and Responsibilities for Gene Therapy’s Road Ahead, Scientific Symposium – Co-chair 

Kris Saha, University of Wisconsin-Madison

8:00am | Room 309-310

Base Editing and Prime Editing II, Oral Abstract Session

Scot Wolfe

Base Editing and Prime Editing II, Oral Abstract Session – Co-chair 

Scot Wolfe, UMass Chan Medical School

10:15am | Ballroom 3

ASGCT presentations where SCGE researchers contributed but are not presenting

SCGE Researcher Abstracts
Aravind Asokan 221: New Biological Insights into Extracellular Vesicle Associated AAV Help Improve Vector Yield and Provides Rational for Intraluminal AAV Packing
Gang Bao 160: Delivery of Large Gene Circuits In Vivo Using an Engineered Baculovirus Vector for Multifactorial Control of Therapeutic Gene Expression
194: In Vivo Expansion of Gene-Targeted Hepatocytes through Transient Inhibition of an Essential Gene
445: Engineering Gene Therapy Vector Properties in the Baculovirus
712: Highly Efficient Gene-Editing of S. aureus Cas9 in Patient Hematopoietic Stem and Progenitor Cells for Treating Sickle Cell Disease
Daniel Carlson 517: Swine Reporter Models for Somatic Cell Genome Editing
Karl J Clark 681: Unconstrained Mitochondrial DNA Editing with Evolved TALE Proteins
Beverly Davidson 50: AAV-Ep+ in Preclinical Studies for Late Infantile Neuronal Ceroid Lipofuscinosis
85: Cochlear Gene Therapy Delivery Innovations, AAV Capsid Variants and Cell Type-Specific Regulatory Elements to Facilitate CSF-Mediated Administration
273: AAV-Based Anti-RAN Antibody Therapy for C9orf72 ALS FTD
311: AAV Capsid Selection at Spatial and Single-Cell Resolution in Non-Human Primate Retina
335: miRNA Site-Blocking Oligonucleotides as a Novel Therapeutic Strategy for STXBP1 Encephalopathy
453: CRISPR Screen for rAAV Production Implicates Genes Associated with Infection
971: Novel AAV Variants for Huntington’s Disease Gene Therapy
1002: Gene Editing Strategies to Treat Spinocerebellar Ataxia Type 1
1130: Ependyma-Derived ApoE2 Protects Against Alzheimer’s Disease Phenotypes in the Presence of ApoE4
1216: miXon, a System for Tunable Control of miRNA Expression by Drug-Induced Splicing
1582: Enhancing Airway Transduction Using AAV Equipped with Advanced Barcoding Methods for CF Gene Therapy
1615: Rescuing STXBP1 Haploinsufficiency in Patient Derived iPSCs Using AAV Mediated Gene Replacement
Ben Deverman 250: Addressing the Translational Potential of Neurotropic AAVs Using Functionally Mature Human Stem Cell-Derived Blood-Brain Barrier Organoids
Stephen Carl Ekker 681: Unconstrained Mitochondrial DNA Editing with Evolved TALE Proteins
Guangping Gao 20: Curable Hyperactive Behaviors and Serum Biomarkers upon Gene Replacement Therapy in Succinic Semialdehyde Dehydrogenase Deficiency in Mice
 24: AAV9 Delivered Artificial microRNAs Effectively Improves GFAP Pathology and Motor Impairment in an Alexander Disease Rat Model
32: Promoter Influences Acute Liver Toxicity and Long-Term Hepatic Genotoxicity in rAAV SMA Gene Therapy in Mice
36: Developing an AAV-Based Gene Replacement Therapy for Mitochondrial Alanyl-tRNA Synthetase 2 (AARS2) Leukodystrophy
37: Extracellular Vesicles as the Underlying Mechanism for Cross-Correction with rAAV-Based Gene Therapy for GM3 Synthase Deficiency
61: Producing High-Purity rAAV Vectors by Recombination-Dependent Minicircle Dual Transfection
67: Novel AAV8 and AAVrh39 ITRs and ITR-Proximal Regions Significantly Enhance Transgene Expression through Enhancer/Promoter-Like Activities
179: AAV-Mediated RhoA Knockdown as a Potential Gene Therapy for Glaucoma
215: Identification of Natural Human-Derived AAV2 Variants with Muscle Tropic Properties in Non-Human Primate Screens
252: Transgene Protein Evolution as a Novel Strategy for Next-Gen Gene Therapy in Canavan Disease
303: Optimized Artificial miRNAs Delivered by rAAV9 Dramatically Improve Survival, Respiratory and Motor Functions of SOD1G93A-ALS Mice
347: Predicting Truncation Events in AAV Vector Genome Designs Using Deep Learning
348: Characterization of AAV Vectors in Tissues Transduced with Conventional and Novel ITRs Show Differences in ITR Recombination, Episome Structures, and Transgene Expression
 
Charles Gersbach 185: Discovering Novel Transcriptional Regulators of T Cell Exhaustion for Epigenetic Reversal of T Cell Dysfunction
186: T-Cell Epigenetic Reprogramming with TGIF2LX-Overexpression Enhances Adoptive Cell Therapy
Anastasia Khvorova 336: Modulation of Somatic Repeat Expansion with Small Interfering RNAs
337: Therapeutic Oligonucleotides Induce Acute Toxicity in CNS, Preventable by the Addition of Divalent Cations to Formulation
1223: Divalent siRNA Therapy for Huntington’s Disease Demonstrates Robust Safety and Efficacy in Pre-Clinical Assessment in an Ovine Model
 
William R Lagor 194: In Vivo Expansion of Gene-Targeted Hepatocytes through Transient Inhibition of an Essential Gene
Kam W Leong 322: Ultrasound-Facilitated Brain Genome Editing
746: Optimization of Lipid Nanoparticles for Gene Editing of the Liver via Intraduodenal Delivery
David Liu 98: The Impact of Base Editing on Rhesus Macaque Hematopoietic Stem Cell Engraftment and In Vivo Clonal Behavior
191: Efficient and Versatile Programmable Large-Gene Integration by Evolved Recombinases and Prime Editing
Cat Lutz 619: Novel AAV Gene Therapy Candidates Show Promise In Vitro and In Vivo for Treatment of PGAP3-CDG Syndrome
1590: AAV-Mediated Gene Augmentation Therapy for NEK1-ALS
1598: Disruption of Aberrant Splicing of STMN2 by Gene Editing with a Type V CRISPR-Cas Enzyme as a Potential Treatment for ALS
Paul McCray 80: Increasing Saline Tonicity Enhances Airway Gene Transfer
1582: Enhancing Airway Transduction Using AAV Equipped with Advanced Barcoding Methods for CF Gene Therapy
Kiran Musunuru 97: In Utero and Postnatal Base Editing for a Metabolic Liver Disease in Mice and Nonhuman Primates
679: Systematic Off-Target Analyses of Base-Editing Therapeutic Leads Show Reduced Off-Target Editing, Reduced Bystander Editing, and Increased On-Target Editing by Hybrid Guide RNAs
William Peranteau 97: In Utero and Postnatal Base Editing for a Metabolic Liver Disease in Mice and Nonhuman Primates
385: Systemic Delivery of LNP mRNA In Utero Permits Editing in a Mouse Model of Spinal Muscular Atrophy (SMA)
679: Systematic Off-Target Analyses of Base-Editing Therapeutic Leads Show Reduced Off-Target Editing, Reduced Bystander Editing, and Increased On-Target Editing by Hybrid Guide RNAs
846: Intrauterine Cellular Therapy Using a Non-Human Primate (NHP) Model
1753: In Utero Systemic LNP mRNA Delivery Targets the Fetal Brain and Permits Diffuse Gene Editing
Kris Saha 338: A Novel High-Throughput Screen to Identify Factors Controlling CRISPR-Mediated Non-Viral Genome Editing
826: In Vivo Pooled Screening Platform for the Discovery of Optimized Chimeric Antigen Receptor (CAR) Design in T cells
829: Virus-Free CRISPR Knock-In of a Chimeric Antigen Receptor into KLRC1 Generates Potent Target-Specific Natural Killer Cells
830: Metabolic Priming of GD2 TRAC-CAR T Cells During Manufacturing Promotes Memory Phenotypes While Enhancing Persistence
1814: Non-Viral Senolytic CRISPR CAR T Cell Products Autonomously Refine into Pure Cell Therapies
Philip Santangelo  824: Sensitizing Solid Tumors to CAR-Mediated Immunity by Lipid Nanoparticle Delivery of Synthetic Antigens
Erik Sontheimer 380: Addressing the dNTP Bottleneck Restricting Prime Editing Activity
Shengdar Tsai 829: Virus-Free CRISPR Knock-In of a Chimeric Antigen Receptor into KLRC1 Generates Potent Target-Specific Natural Killer Cells
Fyodor Urnov 1174: CRISPR Epigenetic Reprogramming of Myokines as Therapy for Alzheimer’s Disease
Jonathan Watts 380: Addressing the dNTP Bottleneck Restricting Prime Editing Activity
729: Antisense Oligonucleotide Targeting FUS-DDIT3 Oncogene for RNase H Degradation
Scot Wolfe 52: From Mouse to Sheep: Developing an AAV Gene Therapy for Sialidosis
380: Addressing the dNTP Bottleneck Restricting Prime Editing Activity
Qiaobing Xu 1273: Bispecific T-Cell Engager Delivered through Tissue Targeting LNP Demonstrated Potent Antitumor Effects on Both Hematological Malignancies and Solid Tumors