May 12, 2021
Programming at the Annual Meeting will begin at 9 a.m. ET (6 a.m. PT, 7 a.m. MT, 8 a.m. CT)
All times are listed in Eastern Time (ET). Schedule is subject to change.
Convert the Annual Meeting to Your Time Zone
9-10 a.m.
Chat Lounge Networking
10-11:45 a.m.
Special Symposium
- Jerry Mendell Award for Translational Science Symposium
Supported by Dr. Suku and Ann Nagendran
Symposia
Building Your Elevator Pitch (Organized by the Communications Committee)
Chair: Edith Pfister, Ph.D.
- 10:00 AM - 10:26 AM
5 Steps for a Polished Grant Submission
Kelly Turner, Ph.D., Baylor College of Medicine
- 10:26 AM - 10:52 AM
Advice for Early-Stage Cell Therapy Professionals
Rayne Rouce, M.D., Baylor College of Medicine
- 10:52 AM - 11:18 AM
Communicating Your Research to Investors
Manisha Pai, Vertex Pharmaceuticals
- 11:18 AM - 11:45 AM
Communicating Complex Science With a Public Audience
Roxanne Khamsi, Freelance Journalist
Cutting Edge Gene and Cell Therapy Research in Europe (Organized by ESGCT)
Co-Chairs: Juan Bueren, Ph.D. and Alberto Auricchio, M.D.
- 10:00 AM - 10:26 AM
AAV Capsid Engineering for In Vivo Gene Therapy
Hildegard Büning, Ph.D., Hannover Medical School
- 10:26 AM - 10:52 AM
Dressing Viruses in Tumors’ Clothing: Cloning-Free Platforms to Trigger Tumor-Specific Immune Response
Vincenzo Cerullo, Ph.D., University of Helsinki
- 10:52 AM - 11:18 AM
LV-Mediated Gene Therapy of Pyruvate Kinase Deficiency
Jose-Carlos Segovia, Ph.D., Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER)
- 11:18 AM - 11:45 AM
Liver-Directed Gene Therapy Clinical Trial for Mucopolysaccharidosis VI
Nicola Brunetti-Pierri, M.D., Telethon Institute of Genetics and Medicine
Entering and Thriving in Industry: Guidance for Academic, Clinical, and Industry Professionals (Organized by the Bio-Industry Committee)
Co-Chairs: Bartholomew Tortella, M.D. and Steven Howe, Ph.D.
- 10:00 AM - 10:15 AM
Preparing For a Career In Industry: What New Entrants Need to Know
Nathaniel Berendson, GlaxoSmithKline (GSK)
- 10:15 AM - 10:30 AM
When to Spin Out: One Academic Perspective
Matthew Porteus, M.D., Ph.D., Stanford University School of Medicine
- 10:30 AM - 10:45 AM
Attracting, Developing, and Retaining the Best Young Academic Science Talent: A Guide for Those in Industry
Kate Barclay, Ph.D., UK BioIndustry Association
- 10:45 AM - 11:00 AM
Life in The Industry Matrix: Journeying Through the Industry Professional Environment
Freda Lewis-Hall, M.D., Pfizer (retired)
- 11:00 AM - 11:45 AM
Panel Discussion
Immunological Barriers to Gene Therapy: Are They Surmountable? (Organized by the Immune Responses to Gene & Cell Therapy Committee)
Co-Chairs: Maria Castro, Ph.D. and Roberto Calcedo, Ph.D.
- 10:00 AM - 10:35 AM
Complement Activation Following AAV Gene Delivery
Pascal Deschatelets, Ph.D., Apellis Pharmaceuticals
- 10:35 AM - 11:10 AM
Cytokine Storm in Response to Gene and Cell Transfer
Stephan Grupp, M.D., Ph.D., University of Pennsylvania Perelman School of Medicine
- 11:10 AM - 11:45 AM
CARs and Armoured CARs: Improving CAR T Cell Therapy for Cancer
Renier Brentjens, M.D., Ph.D., Memorial Sloan Kettering Cancer Center
Novel Viral Gene Transfer Vectors and Applications (Organized by the Viral Gene Transfer Vectors Committee)
Co-Chairs: Masato Yamamoto, M.D., Ph.D. and Phillip Tai, Ph.D.
- 10:00 AM - 10:26 AM
Novel Poxvirus Vectors
Takafumi Nakamura, Ph.D., Tottori University
- 10:26 AM - 10:52 AM
Induced Pluripotent Stem Cells Using Single-Cycle Measles Virus Vector
Patricia Devaux, Ph.D., Mayo Clinic
- 10:52 AM - 11:18 AM
HSV Delivery of Genetic Circuits
Ron Weiss, Ph.D., Massachusetts Institute of Technology
- 11:18 AM - 11:45 AM
In Vivo Hematopoietic Stem Cell Gene Therapy With Hdad5/35++ Adenovirus Vectors
Andre Lieber, M.D. Ph.D., University of Washington
Regulatory Lessons Learned From COVID-19: Anomaly to Precedent (Organized by the Regulatory Affairs Committee)
Co-Chairs: Kit Shaw, Ph.D. and S. Kaye Spratt, Ph.D.
- 10:00 AM - 10:15 AM
EMA Experience on How Emergency Measures Will Inform Flexibility for Gene Therapies
Emer Cooke, European Medicines Agency
- 10:15 AM - 10:30 AM
Clinical Trial Efficacy Assessments During the Covid-19 Pandemic
Wilson Bryan, M.D., Food and Drug Administration
- 10:30 AM - 10:45 AM
Creating a Remote Data-Collection Paradigm in a Rare Pediatric Disease: A Case Study
Genevieve Laforet, M.D., Ph.D., Aspa Therapeutics
- 10:45 AM - 11:00 AM
Incorporating Regulatory Guidance to Provide Continuity for Ongoing Clinical Trials
Jonathan Cotliar, M.D., Science37
- 11:00 AM -11:45 AM
Panel Discussion
Toxicities and Limitations of Gene Therapy (Organized by the Gene & Cell Therapy of Genetic and Metabolic Diseases Committee)
Co-Chairs: Paris Margaritis, D.Phil. and Moanaro Biswas, Ph.D.
- 10:00 AM - 10:26 AM
Mtm Patient Deaths
Carsten Bonnemann, M.D., National Institutes of Health, NINDS
- 10:26 AM - 10:52 AM
The Challenges of DMD Gene Therapy Clinical Trials
Michael Binks, M.D., Pfizer Worldwide Research, Development and Medical
- 10:52 AM - 11:18 AM
Adeno-Associated Virus-Related Toxicities in Nonhuman Primates
Juliette Hordeaux, D.V.M., Ph.D., Gene Therapy Program, University of Pennsylvania
- 11:18 AM - 11:45 AM
Hemophilia - Addressing Durability and Variability of Gene Therapy
Glenn Pierce, M.D., Ph.D., Third Rock Ventures
Translational Gene and Cell Therapy Studies in Cardiovascular Medicine (Organized by Cardiovascular Gene & Cell Therapy Committee)
Co-Chairs: Sangeetha Vadakke-Madathil, Ph.D. and Margaret Sleeper, V.M.D.
- 10:00 AM - 10:26 AM
Gene Therapy With Cyclin-A2 in Pigs
Hina Chaudhry, M.D., Mount Sinai Hospital
- 10:26 AM - 10:52 AM
Gene Therapy of Myosin Binding Protein C in Hypertrophic Cardiomyopathy
Julian Stelzer, Ph.D., Case Western Reserve University
- 10:52 AM - 11:18 AM
Cardiac Regenerative Strategies
Eldad Tzahor, Ph.D., Weizmann Institute of Science
- 11:18 AM - 11:45 AM
Cardiac Phosphoinositide 3-Kinase (P110Α) as a Therapeutic Target for Diabetic Cardiomyopathy - An AAV-Mediated Gene Therapy Approach
Rebecca Ritchie, Ph.D., Monash University
10:45 a.m.-12:15 p.m.
Exhibit Hall Open
Exhibitor Showcase
Unchained Labs
- 10:45 AM - 11:30 AM
Get Hassle-Free AAV & LNP Sample Prep and Characterization From Unchained Labs’ Gene Therapy Squad
Kevin Lance, Ph.D., Unchained Labs
Wyatt Technology
- 10:45 AM - 11:30 AM
Use DLS, SEC-MALS and FFF-MALS towards Well Characterized Gene Vectors
Michelle Chen, Ph.D.; Bob Collins; and Eric Seymour, Wyatt Technology
Bio‐Rad Laboratories
-
11:30 AM - 12:15 PM
Implementing Droplet Digital PCR in Clinical Trial Testing
Mark Wissel, Ph.D., Eurofins Viracor BioPharma Services and Tara Ellison, Ph.D., Bio‐Rad Laboratories
Halo Labs
- 11:30 AM - 12:15 PM
Introducing the Aura CL for Cell and Gene Therapy Aggregation, Particle Analysis and ID
Bernardo Cordovez, Ph.D., Halo Labs
12:15-2 p.m.
Plenary Session
George Stamatoyannopoulos Memorial Lecture and Presentation of the Excellence in Research Awards—Sponsored by REGENXBIO
Michel Sadelain, M.D., Ph.D.
Director of the Center for Cell Engineering; Stephen and Barbara Friedman Chair at Memorial Sloan-Kettering Cancer Center
2-3:30 p.m.
Exhibit Hall Open
Chat Lounge Networking
Industry Sponsored Symposia
Cytiva - Platform processing for AAV production
- 2:00 PM - 3:30 PM
Mats Lundgren, Ph.D., Cytiva
Miltenyi Biotec - Latest Advances in Cell and Gene Therapy
- 2:00 PM - 2:05 PM
Introduction
Matthew Porteus, M.D., Ph.D., Stanford University School of Medicine
- 2:05 PM - 2:30 PM
Development of WU-NK-101, an Off-the-Shelf Memory NK Cell Therapy for the Treatment of AML
Kenneth Chrobak, Ph.D., Wugen
- 2:30 PM - 2:55 PM
In Vivo and In-Vitro Characterization of MART1 Specific T Cells Generated Using the AIM Technology and Prodigy System
Ruipeng Wang, NexImmune
- 2:55 PM - 3:20 PM
Manufacturing Genome Edited Hematopoietic Stem Cells: From Now to the Future
Matthew Porteus, M.D., Ph.D., Stanford University School of Medicine
Vertex Pharmaceuticals Inc. - Restoring and Replacing: Strategies for Potential New Treatment Approaches
- 2:00 PM - 2:05 PM
Welcome and Introduction
Bastiano Sanna, Ph.D., Vertex Pharmaceuticals Inc.
- 2:05 PM - 2:25 PM
Reviewing the Latest Advances in Genetic and Cell Therapy Technologies
Bastiano Sanna, Ph.D., Vertex Pharmaceuticals Inc.
- 2:25 PM - 2:45 PM
Restoring at the Molecular Level: CRISPR/Cas9- Mediated Gene Editing Technology
Eric N. Olson, Ph.D., UT Southwestern Medical Center
- 2:45 PM - 3:05 PM
Replacement at the Cellular Level: Transplantable Stem Cell-Derived Technology
Douglas A. Melton, Ph.D., Harvard University
- 3:05 PM - 3:30 PM
Panel Discussion
Voyager Therapeutics - Advancing AAV Gene Therapy for CNS Disease
- 2:00 PM - 2:05 PM
Welcome and Introduction
Omar Khwaja, M.D., Ph.D., Voyager Therapeutics
- 2:05 PM - 2:15 PM
Voyager Therapeutics’ Commitment to Advancing AAV Gene Therapy for CNS Disease
Andre Turenne, Voyager Therapeutics
- 2:15 PM - 2:35 PM
Building a Better AAV Capsid for CNS Gene Therapies
David Schaffer, Ph.D., University of California, Berkeley
- 2:35 PM - 2:55 PM
Optimizing the Transgene for AAV CNS Gene Therapies
Guangping Gao, Ph.D., University of Massachusetts Medical School
- 2:55 PM - 3:15 PM
Evolving Approaches to Direct Delivery of AAV CNS Gene Therapies
Mark Richardson, M.D., Ph.D., Massachusetts General Hospital
- 3:15 PM - 3:30 PM
Q&A – Moderated by Omar Khwaja, M.D., Ph.D.
Panelists: David Schaffer, Ph.D.; Guangping Gao, Ph.D.; Mark Richardson, M.D., Ph.D.
3:30-5:15 p.m.
Plenary Session
Outstanding New Investigator Symposium—Sponsored by Burroughs Wellcome Fund
- 3:30 PM - 3:56 PM
Mechanisms Underlying CAR T Cell Function
Marcela Maus, M.D., Ph.D., Massachusetts General Hospital
- 3:56 PM - 4:22 PM
Enhancing Genome Editing Technologies Using Protein Engineering
Benjamin Kleinstiver, Ph.D., Massachusetts General Hospital
- 4:22 PM - 4:48 PM
Using the Blood to Treat the Brain: Engineering the Hematopoietic System to Treat Non-Hematological Diseases
Natalia Gomez-Ospina, M.D., Ph.D., Stanford
- 4:48 PM - 5:15 PM
Gene Therapy Approaches to β-hemoglobinopathies
Annarita Miccio, Ph.D., Institut Imagine
5:15-6:45 p.m.
Exhibit Hall Open
Networking Roulette
Tools and Tech Forum II
- 5:15 PM - 5:30 PM
Emerging Technologies: Optimizing Mammalian Cell Culture Cultivation and Analysis
Holly Hattaway, PHC Corporation of North America
- 5:30 PM - 5:45 PM
Streamlined Detection and Characterization of CRISPR Editing Using the rhAmpSeqTM CRISPR Analysis System
Gavin Kurgan, Ph.D., Integrated DNA Technologies (IDT)
- 5:45 PM - 6:00 PM
Fibro Chromatography in Downstream AAV Processing
Peter Guterstam, Ph.D., Cytiva
- 6:00 PM - 6:15 PM
Seeing is Believing - 3D Visualization of VectorMediated Expression in Whole Animals
Hemi Dimant, Ph.D., Invicro, A Konica Minolta Company
- 6:15 PM - 6:30 PM
Quality Matters - Advanced AAV Vector Manufacturing for Reliable Preclinical Results
Christian Thirion, SIRION Biotech
- 6:30 PM - 6:45 PM
The Impact of Closed Systems on Cell and Gene Therapy Scalability
Jayanthi Grebin, CPC
- 6:45 PM - 7:00 PM
Automated Parallel Chromatography to Accelerate Downstream Process Development in Gene Therapy
Jana Langhoff, Tecan; Tim Schroeder, Repligen
Industry Sponsored Symposia
908 Devices - Mass SpectrometryBased Process Analytical Technologies for Cell Therapies
Pall Corporation
5:30-7:15 p.m.
Oral Abstract Session II
AAV Biology, Engineering, Immunology and Animal Modeling
Co-Chairs: Allison Bradbury, Ph.D. and Miguel Sena-Esteves, Ph.D.
- 5:30 PM - 5:45 PM
64: A Multi-Mechanistic Anti-Angiogenic AAV Gene Therapy Product Candidate, 4D-150, for the Treatment of Wet Age-Related Macular Degeneration (wAM.D.) and Diabetic Macular Edema (DME): Intravitreal Biodistribution, Transgene Expression, Safety and Efficacy in Non-Human Primates
Peter Francis, M.D., Ph.D., 4D Molecular Therapeutics
- 5:45 PM - 6:00 PM
65: Evolving Synthetic AAV Variants for Genome Editing in Immune Cell Populations
Jonathan Ark, Molecular Genetics and Microbiology, Duke University
- 6:00 PM - 6:15 PM
66: Real Time Blood Brain Barrier Disruption In A Multi-Species Model
Ana Rita Batista, Ph.D., University of Massachusetts Medical School
- 6:15 PM - 6:30 PM
67: AAV2:2.Retro-Mediated Delivery of Mutant Huntingtin Throughout Cortico-Basal Ganglia Circuitry Leads to the Progressive Development of Motor and Cognitive Decline, Along With Microstructural Changes in White and Gray Matter, in a Novel Rhesus Macaque Model of Huntington’s Disease
Alison Weiss, Ph.D., Oregon National Primate Research Center/OHSU
- 6:30 PM - 6:45 PM
68: Investigating Mechanisms of Variability of AAV5-hFVIII-SQ Expression in Mice
Bridget Yates, BioMarin Pharmaceutical Inc.
- 6:45 PM - 7:00 PM
69: Thermoresponsive Polymer-AAV Nanoparticle Vectors Improved Transgene Expression on Immunized Murine Model
Kai Wang, Ph.D., The University of North Carolina at Chapel Hill
- 7:00 PM - 7:15 PM
70: AAV Vector Dose Dependent Redundant and Non-Redundant Roles of TLR9 and IL1R Signaling in CD8 T Cell Activation Upon Muscle Gene Transfer
Ning Li, Ph.D., IU School of Medicine
CAR Modified Cellular Therapies
Co-Chairs: Maria-Grazia Roncarolo, M.D. and Pietro Genovese, Ph.D.
- 5:30 PM - 5:45 PM
71: Pre-Selected CAR_T_N/SCM Outperform CARTBULK In Driving Tumor Eradication In The Absence Of Severe CRS And ICANS
Silvia Arcangeli, Ph.D., San Raffaele Hospital
- 5:45 PM - 6:00 PM
72: CD5 CAR T-Cells Avoid Self-Elimination by Continuously Degrading CD5 Protein
Royce Ma, Baylor College of Medicine
- 6:00 PM - 6:15 PM
77: Precise Targeting of AML With First-inClass OR / NOT Logic-Gated Gene Circuits in CAR-NK Cells
Brian Garrison, Ph.D., Senti Biosciences
- 6:15 PM - 6:30 PM
74: Investigating the Therapeutic Efficacy of Disruption of Cell Intrinsic Checkpoint Regulator CTLA-4 in Chimeric Antigen Receptor T cells
Sangya Agarwal, University of Pennsylvania Perelman School of Medicine
- 6:30 PM - 6:45 PM
75: Non-Human Primate Derived CD20 CAR T Cells Elicit a Bystander Effect on CD8 but Not CD4 CAR-T Cells
Ulrike Gerdemann, M.D., Dana Farber Cancer Institute; Boston Children’s Hospital
- 6:45 PM - 7:00 PM
76: Enhanced Generation of T-Cell Derived Naïve Pluripotent Cells as a Renewable Cell Source for the Mass Manufacture of Off-the-Shelf CAR T Cell Therapies
Yi-Shin Lai, Ph.D., Fate Therapeutics
Gene Therapies for Hemoglobinopathies
Co-Chairs: John Chapin, M.D. and Pankaj Mandal, Ph.D.
- 5:30 PM - 5:45 PM
78: Early Results From a Phase 1/2 Study of ARU-1801 Gene Therapy for Sickle Cell Disease (SCD): Safety and Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant
Punam Malik, M.D., Cincinnati Children’s Hospital Medical Center
- 5:45 PM - 6:00 PM
79: Immune Reconstitution in Transfusion Dependent Beta-Thalassemia Patients Treated With Hematopoietic Stem Cell Gene Therapy
Samantha Scaramuzza, Ph.D., San Raffaele Telethon Institute for Gene Therapy
- 6:00 PM - 6:15 PM
80: Multiplex Base Editing of Hematopoietic Stem and Progenitor Cells to Enrich Therapeutic Cells Post Engraftment
Olivier Humbert, Ph.D., Fred Hutchinson Cancer Research Center
- 6:15 PM - 6:30 PM
81: In Vivo HSC Gene Therapy for Hemoglobinopathies: A Proof of Concept Evaluation in Rhesus
Macaques Chang Li, Ph.D., University of Washington
- 6:30 PM - 6:45 PM
82: Hematopoietic Reconstitution and Lineage Commitment in HSC Gene Therapy Patients Are Influenced by the Disease Background
Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene Therapy
- 6:45 PM - 7:00 PM
83: Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric Patients
José Luis López Lorenzo, M.D., Hospital Universitario Fundación Jiménez Díaz; Instituto de Investigación Sanitaria Fundación Jiménez Díaz
- 7:00 PM - 7:15 PM
83: Base Editing of the -200 Region of the γ-Globin Promoters Leads to Fetal Hb Reactivation and Rescues the Sickle Cell Disease Phenotype in Primary Patient Cells
Panagiotis Antoniou, Institut Imagine
Immune Responses to AAV Vectors
Co-Chairs: Ying Kai Chan, Ph.D. and Manish Muhuri, Ph.D.
- 5:30 PM - 5:45 PM
85: Declining FVIII Activity Following Hepatic AAV Gene Transfer Because of Translational Shutdown Linked to an Immune Response
John Butterfield, University of Florida
- 5:45 PM - 6:00 PM
86: Requirements for Cross-Presenting Dendritic Cells and CpG Motifs in CD8+ T Cell Response to AAV Gene Transfer
Thais Bertolini, Ph.D., Indiana University School of Medicine
- 6:00 PM - 6:15 PM
87: Defining and Overcoming Preexisting T-Cell Adaptive Immunity to SaCas9 CRISPR-Cas Genome Editors
Andrea Lee, St. Jude Children’s Research Hospital
- 6:15 PM - 6:30 PM
88: Clinical Outcomes in Patients With and Without Pre-Existing Neutralizing Antibodies to the Vector: 6 Month Data From the Phase 3 HOPE-B Gene Therapy Trial of Etranacogene Dezaparvovec
Michael Recht, M.D., Ph.D., The Hemophilia Center at Oregon Health and Science University
- 6:30 PM - 6:45 PM
91: Novel miRNA-Binding Sites That Recruit miR652 and miR-223 in AAV Vector Designs Boost Transgene Levels and Synergistically Suppress Cell-Mediated Immunity
Manish Muhuri, Ph.D., University of Massachusetts Medical School
- 6:45 PM - 7:00 PM
90: IL-1a and IL-1b Are Essential for Inflammasome Independent CD8+ T Cell Responses to Hepatic AAV Gene Transfer
Sandeep Kumar, Ph.D., Indiana University
Novel Factors in AAV Transduction and AAV Genomes
Co-Chairs: David Markusic, Ph.D. and Amanda Dudek, Ph.D.
- 5:30 PM - 5:45 PM
92: Chemical Mediated Recruitment of Epigenetic Modifiers Regulate Adeno-Associated Virus Episomal Transgene Expression
Jessica Umana, University of North Carolina - Chapel Hill
- 5:45 PM - 6:00 PM
93: The Human Silencing Hub (HUSH Complex) is a Potent Regulator of AAV Transgene Silencing
Anshuman Das, Ph.D., Duke University
- 6:00 PM - 6:15 PM
94: Effects of Sexual Dimorphism and Genetic Background on AAV Tissue Transduction in Mice Following Intravenous Administration of a Diverse Capsid Pool
Elad Firnberg, Ph.D., REGENXBIO, Inc.
- 6:15 PM - 6:30 PM
95: High Throughput Screening of Diverse MiniPromoter Libraries Within AAV via Expression Linked Promoter Selection (ELiPS)
Kazuomori Lewis, University of California, Berkeley
- 6:30 PM - 6:45 PM
96: GMEB2 is a Conserved Cellular AAV Restriction Factor That Inhibits Transduction of Human Stem Cells
Amanda Dudek, Ph.D., Stanford University
- 6:45 PM - 7:00 PM
97: Rationally Designed Inverted Terminal Repeats Improve AAV Vector Production
Liujiang Song, Ph.D., University of North Carolina, Chapel Hill
- 7:00 PM - 7:15 PM
98: Characterization of AAV Inverted Terminal Repeats by Atomic Force Microscopy
Marianne Laugel, University of Nantes
Preclinical Gene Therapy for Neurologic Disorders II
Co-Chairs: Gwladys Gernoux, Ph.D. and Juliette Hordeaux, D.V.M., Ph.D.
- 5:30 PM - 5:45 PM
99: A Novel Exon Specific U1 snRNA Therapeutic Strategy to Prevent Retinal Degeneration in Familial Dysautonomia
Anil Chekuri, Ph.D., Massachusetts General Hospital Research Institute; Harvard Medical School
- 5:45 PM - 6:00 PM
105: Efficacy of a Vectorized Anti-Tau Antibody Using Systemic Dosing of a Blood Brain Barrier Penetrant AAV Capsid in Mouse Models of Tauopathies
Wencheng Liu, Ph.D., Voyager Therapeutics
- 6:00 PM - 6:15 PM
101: Evolution of Modified AAV Vectors in Rhesus Macaque Cochlea
Paul Ranum, Ph.D., The Children’s Hospital of Philadelphia
- 6:15 PM - 6:30 PM
102: A Novel Retinal Gene Therapy Strategy for Batten Disease and Beyond
Maura Schwartz, The Research Institute at Nationwide Children’s Hospital
- 6:30 PM - 6:45 PM
103: Reprogramming to Recover Youthful Epigenetic Information and Restore Vision
Yuancheng Lu, Ph.D., Harvard Medical School
- 6:45 PM - 7:00 PM
104: Efficacious, Safe, and Stable Inhibition of Corneal Neovascularization With rAAV-KH902 in a Mouse Model of Corneal Alkali Injury
Wenqi Su, M.D., University of Massachusetts Medical School
Synthetic/Molecular Conjugates and Physical Methods for Delivery
Co-Chairs: Angela Pannier, Ph.D. and Kenya Kamimura, M.D., Ph.D.
- 5:30 PM - 5:45 PM
106: Combinatorial Modified mRNA Induces Cardiovascular Regeneration Post Muscle Ischemic Injury
Keerat Kaur, Ph.D., Icahn School of Medicine, Mount Sinai
- 5:45 PM - 6:00 PM
107: Sustained Episomal Transgene Expression In Vivo Driven by Non-Viral DNA Delivery to Rodent Liver
Stoil Dimitrov, M.D., Ph.D., Moderna
- 6:00 PM - 6:15 PM
108: Optimization of Transcutaneous Ultrasound Mediated Gene Delivery Into Large Animals
Megan Manson, Seattle Children’s Research Institute
- 6:15 PM - 6:30 PM
109: Assembling Several mRNA Strands for Facilitating mRNA Delivery With and Without Using Carriers
Satoshi Uchida, M.D., Ph.D., Kyoto Prefectural University of Medicine; Kawasaki Institute of Industrial Promotion
- 6:30 PM - 6:45 PM
110: Development of Hydrodynamics-Based Gene Therapy for Liver Cancer
Kenya Kamimura, M.D., Ph.D., Niigata University
- 6:45 PM - 7:00 PM
111: Hematopoietic Stem and Progenitor Cells-Targeted Polymeric Nanoparticles for In Vivo Gene Therapy
Rkia El kharrag, Ph.D., Fred Hutchinson Cancer Research Center
- 7:00 PM - 7:15 PM
112: Delivery of CRISPR/Cas9 for Recovering the Expression of the Endogenous FVIII in Hemophilia A Mice
Chun-Yu Chen, Ph.D., Seattle Children’s Hospital Research Institute
Targeted Gene and Cell Therapy for Cancer
Co-Chairs: Michael Milone, M.D., Ph.D. and Hernando Lopez-Bertoni, Ph.D.
- 5:30 PM - 5:45 PM
113: Gene-based Immune Reprogramming Overcomes the Immunosuppressive Microenvironment of Liver Metastases and Enables Protective T Cell Responses
Thomas Kerzel, San Raffaele Telethon Institute for Gene Therapy
- 5:45 PM - 6:00 PM
114: Inducible Tumor-Targeted Interferon-α Gene Therapy Inhibits Glioblastoma Multiforme in Mouse Model Without Adverse Systemic Effects
Filippo Birocchi, San Raffaele Telethon Institute for Gene Therapy
- 6:00 PM - 6:15 PM
115: Design and Demonstration of Potent In Vitro and In Vivo Activity for CART-ddBCMA, a BCMATargeted CAR-T Cell Therapy Incorporating a Non-scFv Binding Domain
Janine Buonato, Ph.D., Arcellx
- 6:15 PM - 6:30 PM
116: A SOX2 Engineered Epigenetic Silencer Factor Represses the Cancer Genetic Program and Eradicate Glioblastoma Development
Vania Broccoli, Ph.D., San Raffaele Scientific Institute
- 6:30 PM - 6:45 PM
117: Oncolytic Adeno-Immunotherapy Expressing IL-12p70 and Immune Checkpoint Blockade PD-L1 Minibody Modulates the Host Immune System to Enable HER2.CAR T-Cells to Cure Pancreatic Tumors
Amanda Rosewell Shaw, Ph.D., Baylor College of Medicine
- 6:45 PM - 7:00 PM
118: UCARTCS1A, an Allogeneic CAR T-Cell Therapy Targeting CS1 in Patients With Relapsed/ Refractory Multiple Myeloma (RRMM): Preliminary Translational Results From a First-in-Human Phase I Trial (MELANI-01)
Krina Patel, M.D., The University of Texas M.D. Anderson Cancer Center
- 7:00 PM - 7:15 PM
119: A Drug-Regulated Anti-CD33 Chimeric Antigen Receptor With Potent Anti-AML Activity and a Reversible On-Off Switch
Jacob Applebaum, M.D., Ph.D., Seattle Children’s Therapeutics; University of Washington
Upstream Process Development for AAV Vector Production
Co-Chairs: Sanford Boye and Laura Adamson-Small, Ph.D.
- 5:30 PM - 5:45 PM
120: Co-Identification and Characterization of Host and Viral Protein Interactomes During AAV Production by Two Different Proximity Labeling Methods
Ji Sun Lee, Ph.D., University of Massachusetts Medical School
- 5:45 PM - 6:00 PM
121: The Effects of ITR Structure and Plasmid Backbone on Plasmid Stability and Yield
Ruofan Wang, Ph.D., Vigene Biosciences
- 6:00 PM - 6:15 PM
122: Increasing Gene Therapy Vector Production Using Viral Sensitizer Molecules
Jean-Simon Diallo, Ph.D., Virica Biotech, Inc.
- 6:15 PM - 6:30 PM
123: Transcriptional Response of HEK293 Cells to Clinical-Scale Recombinant Adeno Associated Virus Production by Transient Transfection
Cheng-Han Chung, Ph.D., Pfizer, Inc.
- 6:30 PM - 6:45 PM
124: Vector Engineering of pRep-Cap and pHelper Enhanced AAV Productivity by Triple Transfection in Suspension HEK293 Cells
Bingnan Gu, Ph.D., Lonza Houston Inc.
- 6:45 PM - 7:00 PM
125: High Titer rAAV Production Upon Upstream Process Development of Stable Helper-Virus Free ELEVECTA® Producer Cells
Juliana Coronel, Ph.D., Cevec Pharmaceuticals GmbH
- 7:00 PM - 7:15 PM
126: Genome-Wide CRISPR Activation Screen Reveals That SKA2 and ITPRIP Increase AAV Manufacturing via Cell Cycle Modulation
Hyuncheol Lee, D.V.M.,