May 14, 2021
10-11:45 a.m.
Scientific Symposia
Newborn Screening: Innovative Policies and Technologies to Eliminate the Diagnostic Odyssey (Government Relations Committee)
Co-Chairs: Diane Berry, Ph.D. and Philip Reilly, M.D., J.D.
- 10:00 AM - 10:15 AM
Newborn Screening: An Inside Look at the Landscape, Challenges, and Opportunities for Strengthening Newborn Health
Max Bronstein, MGB Consulting
- 10:15 AM - 10:30 AM
The Impact of Newborn Screening Policies for Genetic Diseases on Children and Families
Elisa Seeger, Aiden Jack Seeger Foundation
- 10:30 AM - 10:45 AM
How Will Gene and Cell Therapies Transform Newborn Screening?
Don Bailey, Ph.D., RTI International
- 10:45 AM - 11:00 AM
Screenplus: A Multi-Disorder, Multi-Tiered, Consented Pilot Newborn Screening Program
Melissa Wasserstein, M.D., Montefiore Medical Center
- 11:00 AM - 11:15 AM
Advances in Diagnostic Testing Technology: Implications for NBS
Ryan Taft, Ph.D., Illumina
- 11:15 AM - 11:45 AM
Panel Discussion
Safety and Efficacy of Bodywide Therapy for Musculo-Skeletal Diseases (Musculo-Skeletal Gene and Cell Therapy Committee)
Co-Chairs: Jyoti Jaiswal, Ph.D. and Christina Pacak, Ph.D.
- 10:00 AM - 10:26 AM
Overview of Data From Clinical Trials of AAV Gene Therapy for DMD
Perry Shieh, M.D., Ph.D., University of California, Los Angeles
- 10:26 AM - 10:52 AM
Safety/Efficacy of Systemic Genome Editing for DMD
Christopher Nelson, Ph.D., University of Arkansas
- 10:52 AM - 11:18 AM
Optimization Studies to Enable a Phase 1 Clinical Trial of iPSC-Derived Myogenic Progenitors for DMD
Rita Perlingeiro, Ph.D., University of Minnesota
- 11:18 AM - 11:45 AM
rAAV Controlled Gene Delivery to Heal Cartilage Defects Combined With Biomaterials in Large Animals
Magali Cucchiarini, Ph.D., Saarland University Medical Center
Vaccine Nanotechnology for Rapid Response Applications (Nanoagents and Synthetic Formulations Committee)
Co-Chairs: Jordan Green, Ph.D. and Julie Champion, Ph.D.
- 10:00 AM - 10:26 AM
Polyplex Based DNA Nanoparticles as Vaccines
Darrell Irvine, Ph.D., Massachusetts Institute of Technology
- 10:26 AM - 10:52 AM
Identifying Vital Immunogenic Epitopes in Pathogens as Therapeutic Targets
Guarav Gaiha, M.D., Ragon Institute of MGH
- 10:52 AM - 11:18 AM
Replicating Viral RNA as Gene Expression Platform
Jesse Erasmus, Ph.D., University of Washington
- 11:18 AM - 11:45 AM
Development of RNA Vaccines
Drew Weissman, Ph.D., University of Pennsylvania
New Advances in Physical Gene Delivery and Nucleic Acid Vectorology (Physical Delivery, Therapeutics, and Vector Development Committee)
Co-Chairs: Loree Heller, Ph.D. and Carol Miao, Ph.D.
- 10:00 AM - 10:26 AM
Electroporation to Deliver DNA Coronavirus Vaccine
Kate Broderick, Ph.D., Inovio Pharmaceuticals, Inc.
- 10:26 AM - 10:52 AM
RNA-Guide DNA Insertion With CRISPR-Associated Transposases
Feng Zhang, Ph.D., Massachusetts Institute of Technology
- 10:52 AM - 11:18 AM
mRNA as Therapeutics to Treat Rare Genetic Disorders
Paolo Martini, Ph.D., Moderna
- 11:18 AM - 11:45 AM
Ultrasound-Targeted Microbubble Destruction to Facilitate Gene Delivery
Mark Borden, Ph.D., University of Colorado
Hot Topics and Remaining Challenges in RNAi and Oligonucleotide Therapy for 2021 (Oligonucleotide and RNAi Therapeutics Committee)
Chair: Paloma Giangrande, Ph.D.
- 10:00 AM - 10:26 AM
CNS Toxicity in Higher Mammals Related to AAVMediated RNAi Gene Therapy
Beverly Davidson, Ph.D., Children’s Hospital of Philadelphia
- 10:26 AM - 10:52 AM
Splice Correction and Reduction of Toxic DMPK RNA In Vitro and In Vivo Utilizing Novel Antibody Targeted Antisense Oligonucleotides
Romesh Subramanian, Ph.D., Dyne Therapeutics
- 10:52 AM - 11:18 AM
Discussion of N-Lorem Foundation’s Mission and Progress to Create Individual Antisense Therapies for Patients in The United States With Ultra-Rare Diseases Caused by Genetic Mutations That Affect Approximately N=1-10 Patients in the World
Stanley Crooke, M.D. Ph.D., n-Lorem Foundation
- 11:18 AM - 11:45 AM
Opportunities and Challenges in Development of ASOs for Treatment of Neuromuscular Disease
Toby Ferguson, M.D., Ph.D., Biogen
Race to Respiratory Therapies for COVID-19 (Respiratory and GI Tract Gene and Cell Therapy Committee)
Chair: Amy Ryan, Ph.D.
- 10:00 AM - 10:35 AM
Animal Models for SARS-CoV-2 Research Tracy Webb, D.V.M., Ph.D., Colorado State University and Danielle Adney, Ph.D., National Institutes of Health, NIAID
- 10:35 AM - 11:10 AM
AAVCOVID: An AAV-Based, Single Dose, Rt Stable Covid-19 Vaccine Candidate
Luk Vandenberghe, Ph.D., Grousbeck Gene Therapy Center, Mass Eye and Ear, Harvard & The Broad Institute of Harvard and MIT
- 11:10 AM - 11:45 AM
Airway Vaccine Approach SARS-Cov-2 Using RAAV
James Wilson, M.D., Ph.D., University of Pennsylvania
RNA Therapies for Neurologic and Ophthalmic Disorders (Neurologic and Ophthalmic Gene and Cell Therapy Committee)
Co-Chairs: Kourous Rezaei, M.D. and Jason Shepherd, Ph.D.
- 10:00 AM - 10:26 AM
Splicing Modulation Therapy for Inherited Retinal Diseases
Rob Collin, Ph.D., Radboud University Medical Centre
- 10:26 AM - 10:52 AM
The Use of ODNs for Cellular Therapy of Neurodegenerative Disorders
Holly Kordasiewicz, Ph.D., Ionis Pharmaceuticals
- 10:52 AM - 11:18 AM
From Impossible to Possible: A Personal Journey From Diagnosis to Drug Development for Angelman Syndrome
Allyson Berent, D.V.M., Foundation for Angelman Syndrome Therapeutics
- 11:18 AM - 11:45 AM
Shifting the Paradigm of Gene Therapy for Neuromuscular Diseases
Pavlina Konstantinova, Ph.D., Vectory
Clinical Trials Spotlight Symposium
Chair: Terence R. Flotte, M.D.
- 10:00 AM - 10:15 AM
194: Systemic AAV Delivery Activates the Classical Complement Pathway Leading to Thrombotic Microangiopathy
Stephanie Salabarria, BH.Sc., University of Florida
- 10:15 AM - 10:30 AM
195: Activity of Banked (Off-the-Shelf) CD30. CAR-Modified Epstein-Barr Virus-Specific T Cells in Patients with CD30-Positive Lymphoma
David Quach, Ph.D.
- 10:30 AM - 10:45 AM
196: Updated Results From HGB-206 LentiGlobin for Sickle Cell Disease Gene Therapy Study: Group C Data and Group A AML Case Investigation
John Tisdale, M.D., National Institutes of Health
- 10:45 AM - 11:00 AM
197: ADVM-022 Intravitreal Gene Therapy for Neovascular AM.D. - Results From the Phase 1 OPTIC Study
Szilárd Kiss, M.D., Weill Cornell Medicine
- 11:00 AM - 11:15 AM
198: Natural Killer T Cells Expressing a GD2- CAR and IL-15 Are Safe and Can Induce Complete Remission in Children with Relapsed Neuroblastoma - A First-in-Human, Phase 1 Trial
Andras Heczey, M.D., Baylor College of Medicine
- 11:15 AM - 11:30 AM
199: Long Term Follow Up for the Development of Subsequent Malignancies in Patients Treated With Genetically Modified Immune Effectors
David Steffin, M.D., Baylor College of Medicine
- 11:30 AM - 11:45 AM
200: Updated Results of Transpher A, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-102 Gene Therapy for Sanfilippo Syndrome Type A (Mucopolysaccharidosis IIIA)
Kevin Flanigan, M.D., Nationwide Children’s Hospital
10:45 a.m.-12:15 p.m.
Exhibitor Showcase
Single Cell Multiomics to Accelerate Cancer Immune Cell Therapy Research
- 10:45 AM - 11:15 AM
Multi-modal and Multi-tissue Profiling of Glioblastoma Patients Treated with CAR T Cell Therapy
Nicholas Banovich, Ph.D., Translational Genomics Research Institute (TGen)
- 11:15 AM - 11:30 AM
Single Cell and Spatial Multiomics to Accelerate Cancer Immune Cell Therapy Research
Abbey Cutchin, 10x Genomics
Trends and Challenges in Cell & Gene Therapy
- Ly Nguyen-Jatkoe, Ph.D., Informa Pharma Intelligence
Exhibit Hall Open
12:15-2 p.m. Oral Abstract Session V
Base Editing and Gene Editing Approaches
Co-Chairs: Giulia Pavani, Ph.D. and Shengdar Tsai, Ph.D.
- 12:15 PM - 12:30 PM
201: Intracellular Rnase Activity Limits the Efficiency of mRNA-Based Gene Editing in Nonhuman Primate Hematopoietic Stem and Progenitor Cells
Christopher Peterson, Ph.D., Fred Hutchinson Cancer Research Center
- 12:30 PM - 12:45 PM
205: Cleavage-Free dCas9 Knock-In GeneEditing Tool Leveraging RNA-Guided Targeting of Recombineering Proteins
Le Cong, Ph.D., Stanford University
- 12:45 PM - 1:00 PM
203: Novel CRISPR-Associated Transposase Systems for Targeted DNA Integration
Daniela Goltsman, Ph.D., Metagenomi
- 1:00 PM - 1:15 PM
204: CRISPR-Cas9 Cytidine and Adenosine Base Editing of Splice-Sites Mediates HighlyEfficient Disruption of Proteins in Primary and Immortalized Cells
Mitchell Kluesner, University of Minnesota
- 1:15 PM - 1:30 PM
202: Enhancing the RNA Base-Editing Activity, Functionality and Specificity of the ADAR2 Deaminase Domain
Dhruva Katrekar, Ph.D., University of California - San Diego
- 1:30 PM - 1:45 PM
206: Novel CRISPR Associated Gene Editing Systems Discovered in Metagenomic Samples Enable Efficient and Specific Genome Engineering for Cell Therapy Development
Gregory Cost, Ph.D., Metagenomi
- 1:45 PM - 2:00 PM
207: Base Editing Approaches for the Treatment of β-hemoglobinopathies through Disruption of the Erythroid-Specific BCL11A Enhancer to Reactivate Fetal Hb
Panagiotis Antoniou, Institut Imagine
Cancer Immunotherapy
Co-Chairs: Jan Joseph Melenhorst, Ph.D. and Sarwish Rafiq, Ph.D.
- 12:15 PM - 12:30 PM
208: Clinical Application of CRISPR Edited Tumor Infiltrating Lymphocytes in Gastrointestinal Cancer
Beau Webber, Ph.D., University of Minnesota
- 12:30 PM - 12:45 PM
209: Single-Cell Sequencing Approach for the Discovery of Mutant IDH1 Reactive T Cell Receptors From a Glioma Vaccine Trial and an MHC-Humanized Mouse Model
Khwab Sanghvi, German Cancer Research Center
- 12:45 PM - 1:00 PM
210: Epigenome Editing Enables PD1 Silencing in Primary T Cells
Maria Silvia Roman Azcona, University of Freiburg
- 1:00 PM - 1:15 PM
211: mRNA Delivery of an Evolved Bispecific Single Domain Antibody to Synergize the Immune Checkpoint Blockade Therapy for Liver Malignancies
Rihe Liu, Ph.D., University of North Carolina - Chapel Hill
- 1:15 PM - 1:30 PM
212: Exhausted, Tumor-Specific T Cells Can Be Exploited to Generate a Library of T-Cell Receptors for Adoptive T-Cell Therapy in Blood Malignancies
Francesco Manfredi, M.D., San Raffaele University
- 1:30 PM - 1:45 PM
213: Changes in the Tumor Microenvironment in Patients with Glioblastoma Multiforme Treated with IFN-a Immune Cell & Gene Therapy (TEM-GBM_001 Study)
Bernhard Gentner, M.D., San Raffaele Telethon Institute for Gene Therapy
- 1:45 PM - 2:00 PM
214: Small Molecule-Regulated Gene Circuit for Controlling Cytokine Expression in Cell Therapies
Michelle Hung, Ph.D., Senti Bio
Gene Therapy for Lysosomal Storage Disorders
Co-Chairs: Lina Colella, Ph.D. and Pasquale Piccolo, Ph.D.
- 12:15 PM - 12:30 PM
215: RGX-121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II: Interim Analysis of the First in Human Study
Roberto Giugliani, M.D., Medical Genetics Service, HCPA
- 12:30 PM - 12:45 PM
216: AVR-RD-01, an Investigational Lentiviral Gene Therapy for Fabry Disease: Clinical Data Trends from Phase 1 and Phase 2 Studies up to 3.5 Years
Mark Thomas, M.D., Royal Perth Hospital
- 12:45 PM - 1:00 PM
217: Updated Results of Transpher B, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-101 Gene Therapy for Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB)
Maria de Castro, M.D., Hospital Clínico Universitario de Santiago de Compostela
- 1:00 PM - 1:15 PM
218: Safety and Efficacy of LiverDirected Gene Therapy in Patients with Mucopolysaccharidosis Type VI
Nicola Brunetti-Pierri, M.D., Telethon Institute of Genetics and Medicine
- 1:15 PM - 1:30 PM
219: Ex Vivo Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type IH (Hurler Syndrome): An Interim Analysis with a Median Follow Up of 19 Months
Bernhard Gentner, M.D., San Raffaele Telethon Institute for Gene Therapy
- 1:30 PM - 1:45 PM
220: A Targeted AAV Gene Therapy Product Candidate, 4D-310, for the Treatment of Fabry Disease: Intravenous Biodistribution, Transgene Expression and Safety in Non-Human Primates
Kevin Whittlesey, Ph.D., 4D Molecular Therapeutics
- 1:45 PM - 2:00 PM
221: Efficacy of Intracerebroventricular AdenoAssociated Virus Encoding Iduronidase with Dorsal Root Ganglia-Detargeting Sequences in a Mouse Model of Mucopolysaccharidosis Type I
Juliette Hordeaux, D.V.M., Ph.D., University of Pennsylvania
Lentiviral Vector Manufacturing
Co-Chairs: Magalie Penaud-Budloo, Ph.D. and Matthias Hebben, Ph.D.
- 12:15 PM - 12:30 PM
222: Stabilization of Lentiviral Vector Genomic RNA during Production Using a Novel RNA Chaperone Leads to Increased Yields
Jordan Wright, Ph.D., Oxford BioMedica
- 12:30 PM - 12:45 PM
223: Knockout of Entry Receptors in Virus Producer Cells for Improved Titer and Quality BaEVRless-Pseudotyped Retroviral Particles
Denise Klatt, Ph.D., Dana Farber Cancer Institute
- 12:45 PM - 1:00 PM
224: Development of pEMBR™- An Improved Adenovirus Helper Plasmid for AAV Production
David Dismuke, Ph.D., Forge Biologics
- 1:00 PM - 1:15 PM
225: Genetically Engineering Packaging Cells to Enhance Titer and Infectivity of Lentiviral Vectors
Jiaying Han, University of California - Los Angeles
- 1:15 PM - 1:30 PM
227: Processing of Lentiviral Vectors Pseudotypes Using Anion Exchange and Affinity Chromatography
Yuefei Huang, Ph.D., U.S. Food and Drug Administration
- 1:30 PM - 1:45 PM
228: Separation of Empty Capsids from Full Capsids for AAV Gene Therapy Using a Flow through and Step Elution Approach
Danielle Ladwig, Voyager Therapeutics
Metabolic and Muscle Diseases, Tissue and Immunological Engineering
Co-Chairs: Douglas Martin, Ph.D. and Isabelle Richard, Ph.D.
- 12:15 PM - 12:30 PM
229: Immunogenicity of An AAV-based, Room-Temperature Stable, Single Dose COVID-19 Vaccine in Mouse and NHP
Nerea Zabaleta, Ph.D., Grousbeck Gene Therapy Center
- 12:30 PM - 12:45 PM
230: Systemic AAV9 Gene Therapy Rescues Propionic Acidemia (PA) Mice From Neonatal Lethality and Provides Sustained Therapeutic Benefits
Lina Li, M.D., Ph.D., National Human Genome Research Institute
- 12:45 PM - 1:00 PM
231: A Randomized, Double-Blind, PlaceboControlled, Gene-Delivery Clinical Trial of rAAVrh74.MHCK7.micro-dystrophin for Duchenne Muscular Dystrophy
Jerry Mendell, M.D., Nationwide Children’s Hospital
- 1:00 PM - 1:15 PM
232: Unprecedented Low Dose of AAV-Mediated Gene Transfer Corrects the Pathology in a Model for Fukutin-Related-Protein Deficiencies
Isabelle Richard, Ph.D., Genethon
- 1:15 PM - 1:30 PM
233: AAV Mediated Apelin Transduction Facilitates Cell Cycle Entry of cTnT-Positive Cells in the Heart
Andrew Park, AstraZeneca
- 1:30 PM - 1:45 PM
234: Vectorized SARS-CoV-2 Human IgG Expression in Mice and Ovine Animal Models Is Feasible and Well Tolerated
Amira Rghei, University of Guelph
- 1:45 PM - 2:00 PM
235: Intravenous AAV5 Gene Therapy with Human CYP21A1 Corrects Phenotypic Deficiencies of the 21-Hydroxylase Knockout Mouse Model and Demonstrates Durability and Safety in Non-Human Primates and Mice
Rachel Eclov, Ph.D., BridgeBio
New Technologies Advancing Gene Therapy for Neurologic Diseases
Co-Chairs: Dan Wang, Ph.D. and Paul Valdmanis, Ph.D.
- 12:15 PM - 12:30 PM
236: Combined Transgene and Intron-Derived miRNA Therapy Reverses Motor Phenotypes in SCA1
Mice Ellie Carrell, Ph.D., Raymond G. Perelman Center for Cellular and Molecular Therapeutics
- 12:30 PM - 12:45 PM
237: Combining MALDI-Based Metabolic Imaging and Molecular Analysis for Effective and Informative Assessment of Therapeutic Efficacy in Sub-Anatomical Regions of the CNS After rAAV Gene Therapy
Dominic Gessler, M.D., Ph.D., University of Massachusetts
- 12:45 PM - 1:00 PM
238: Evolution of Modified AAV in Rhesus Macaque Brain
Paul Ranum, Ph.D., The Children’s Hospital of Philadelphia
- 1:00 PM - 1:15 PM
239: Real-Time MR Tracking of AAV Gene Therapy with Enzyme-Activated MR Probes
Toloo Taghian, Ph.D., University of Massachusetts Medical School
- 1:15 PM - 1:30 PM
240: Inclusion of a Degron Reduces Levels of Undesired Inteins after AAV-Mediated Protein Trans-Splicing in the Retina
Patrizia Tornabene, Telethon Institute of Genetics and Medicine
- 1:30 PM - 1:45 PM
241: Drug-Regulated Splicing Switch for Gene Expression Control
Alex Monteys, Ph.D., University of Pennsylvania
- 1:45 PM - 2:00 PM
242: Targeted Gene Therapy with Engineered Systemic AAVs for the Central and Peripheral Nervous Systems Prevents Motor Coordination Phenotypes in a Mouse Model of Friedreich’s Ataxia
Acacia Hori, California Institute of Technology
Oligonucleotide Therapeutics
Co-Chairs: Mark Kay, M.D., Ph.D. and Paloma Giangrande, Ph.D.
- 12:15 PM - 12:30 PM
243: Robust RNA Editing via Recruitment of Endogenous ADARs Using Circular Guide RNAs
Dhruva Katrekar, Ph.D., University of California - San Diego
- 12:30 PM - 12:45 PM
244: In Vivo Delivery of AAV.U7 Induce Efficient Exon Skipping for a Mutational Hotspot of the DM.D. Gene Results in Protein Restoration & Force Improvement in Skeletal Muscles, Heart & Diaphragm
Dhanarajan Rajakumar, Ph.D., Nationwide Children’s Hospital
- 12:45 PM - 1:00 PM
245: A Versatile Platform for ADAR-Mediated RNA Editing In Vivo in Preclinical Models
Prashant Monian, Ph.D., Wave Life Sciences
- 1:00 PM - 1:15 PM
246: Human miRNA mir-675 Inhibits DUX4 Expression And May Be Exploited As A Potential Treatment For Facioscapulohumeral Muscular Dystrophy
Nizar Saad, Ph.D., Nationwide Children’s Hospital
- 1:15 PM - 1:30 PM
247: The FORCETM Platform Achieves Robust Knock Down of Toxic Human Nuclear DMPK RNA and Foci Reduction in DM1 Cells and in Newly Developed hTfR1/DMSXL Mouse Model
Stefano Zanotti, Ph.D., Dyne Therapeutics
- 1:30 PM - 1:45 PM
248: In Vivo Delivery of Suppressor tRNA Overcomes a Pathogenic Nonsense Mutation in Mice
Jiaming Wang, Ph.D., University of Massachusetts Medical School
- 1:45 PM - 2:00 PM
249: The 3´-tsRNAS Are Aminoacylated Further Implicating Their Role in Ribosome Biogenesis During Tissue Homeostasis and Cancer
Mark Kay, M.D., Ph.D., Stanford University School of Medicine
Pharmacology/Toxicology Studies or Assay Development
Co-Chairs: Cristina Baricordi, Ph.D. and Carmen Unzu, Ph.D.
- 12:15 PM - 12:30 PM
250: Liquid-Biopsy-Integration-Site-Sequencing Allows Safety Studies and Longitudinal Monitoring of Vector Integration Sites in LV and AAV-Based In-Vivo GT Applications
Daniela Cesana, Ph.D., San Raffaele Telethon Institute for Gene Therapy
- 12:30 PM - 12:45 PM
251: Physiologically Based Pharmacokinetic Modeling for the Biodistribution of AdenoAssociated Virus Serotype 8 after Intravenous Administration in Mice and Non-Human Primates
Kefeng Sun, Ph.D., Shire Human Genetic Therapies
- 12:45 PM - 1:00 PM
252: LC-MS Confirmation of Single Amino Acid Correction by Base Editing
Bo Yan, Ph.D., Beam Therapeutics
- 1:00 PM - 1:15 PM
253: Lack of Germline Transmission in Male Mice Following Administration of AAV5-hFVIII-SQ, an Investigational Gene Therapy for Hemophilia A
Cheng Su, Ph.D., BioMarin Pharmaceutical Inc.
- 1:15 PM - 1:30 PM
254: DNA Sequence Analysis of Recombinant Adeno-Associated Viral Integrations Events Recovered from Hepatocellular Carcinomas in Mice Reveals Enhancer Insertion as the Mechanism of Vector Genotoxicity
Randy Chandler, Ph.D., National Institutes of Health
- 1:30 PM - 1:45 PM
255: Towards a Clinical Trial of Gene Therapy for Retinitis Pigmentosa Associated with Usher Syndrome Type IB
Rita Ferla, Ph.D., Telethon Institute of Genetics and Medicine
- 1:45 PM - 2:00 PM
256: A GLP Safety and Biodistribution Study of AXO-Lenti-PD Manufactured via Two Processes Delivered at a Higher Volume and Flow Rate
Thomas Pack, Ph.D., Sio Gene Therapies
RNA Virus Vectors
Co-Chairs: Brian Bigger, Ph.D. and Andrew Wilber, Ph.D.
- 12:15 PM - 12:30 PM
257: Combinatorial Relief of Multiple Innate Immune Blocks Allows Efficient Gene Engineering of Quiescent Human Hematopoietic Stem Cells
Erika Valeri, San Raffaele Telethon Institute for Gene Therapy
- 12:30 PM - 12:45 PM
258: Administration During Liver Growth Improves the Efficiency of Lentiviral Vector Based In Vivo Gene Therapy in Mice
Francesco Starinieri, San Raffaele Telethon Institute for Gene Therapy
- 12:45 PM - 1:00 PM
259: MicroRNA Detargeting Proves Superior to Genetic Attenuation for Balancing Safety and Efficacy of Oncolytic Mengovirus in Immunodeficient Glioblastoma Mouse Model
Yogesh Suryawanshi, M.D., Ph.D., Mayo Clinic
- 1:00 PM - 1:15 PM
260: CTCF-Based Chromatin Insulators and Enhancers in Lentiviral Vectors Impact Genome Topology and Vector Safety
Monica Volpin, Ph.D., San Raffaele Telethon Institute for Gene Therapy
AAV Vectors - Clinical Studies
Co-Chairs: Steven Gray, Ph.D. and Diana Bharucha-Goebel, M.D.
- 1:15 PM - 1:30 PM
261: AAV8-Mediated Liver-Directed Gene Therapy as a Potential Therapeutic Option in Adults with Glycogen Storage Disease Type Ia (GSDIa): Updated Phase 1/2 Clinical Trial Results
David Rodriguez-Buritica, M.D., University of Texas McGovern Medical School
- 1:30 PM - 1:45 PM
262: Safety, β-Sarcoglycan Expression, and Functional Outcomes from Systemic Gene Transfer of rAAVrh74.MHCK7.SGCB in Limb Girdle Muscular Dystrophy Type 2E/R4
Louise Rodino-Klapac, Ph.D., Sarepta Therapeutics
- 1:45 PM - 2:00 PM
263: IGNITE-DMD Phase I/II Study of SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy
Carl Morris, Ph.D., Solid Biosciences