#ASGCT17 - 2020 Annual Meeting · Sangamo Therapeutics 814 Spark Therapeutics 702 STEMCELL...

FINAL PROGRAM • POCKET GUIDE • WASHINGTON, DC • 2017 1

AMERICAN SOCIETY OF GENE & CELL THERAPY

20TH ANNUAL MEETINGFINAL PROGRAM POCKET GUIDE

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TABLE OF CONTENTSMobile App Instructions ........................................................................................ 2

A Welcome from Cindy Dunbar, MD .................................................................. 3

Annual Meeting Exhibitors .................................................................................... 4

Exhibit Hall Floor Plan ............................................................................................ 5

Annual Meeting Supporters ................................................................................. 6

Hotel Floor Plans .................................................................................................... 8

Program Schedule .............................................................................................. 11

ASGCT AWARD RECIPIENTSCongratulations to the follow individuals for receiving an ASGCT Award!

SONIA SKARLATOS PUBLIC SERVICE AWARD

George Stamatoyannopoulos, MD, Dr. SciUniversity of Washington

Seattle, WA

OUTSTANDING ACHIEVEMENT AWARD

Maria-Grazia Roncarolo, MD Stanford University School of Medicine

Stanford, CA

OUTSTANDING NEW INVESTIGATOR AWARDS

James N. Kochenderfer, MDNational Cancer Institute

Bethesda, MD

Lili Yang, PhD University of California Los Angeles

Los Angeles, CA

2 FINAL PROGRAM • POCKET GUIDE • WASHINGTON, DC • 2017

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Dear Colleagues,On behalf of the American Society of Gene & Cell Therapy (ASGCT), it is my pleasure to welcome you to the ASGCT 20th Annual Meeting. I am honored to join you and thousands of our colleagues from around the globe who share in our Soci-ety’s mission of advancing knowledge, awareness, and edu-cation to advance the discovery and clinical application of gene and cell therapies for alleviation of human disease.

This scientific and educational program strongly reflects the rapid advance-ments that have occurred in our field over the past year. Our invited faculty includes more than 100 of the most distinguished investigators in our field, pre-senting cutting edge research in Scientific Symposia, or overviews of import-ant concepts and technologies in Education Sessions, throughout the four day meeting. In addition, join us in welcoming Dr. Eric Olson, who will present the George Stamatoyannopoulos Lecture on Thursday following the presenta-tion of the ASGCT Public Service Award to Dr. Stamatoyannopoulos, and Dr. Robert Langer, who will present the Presidential Lecture on Friday afternoon. From the nearly 800 abstracts submitted to the meeting, the Program Commit-tee has chosen four groundbreaking scientific and clinical advances across the breadth of our field to be presented in the Presidential abstract session Fri-day afternoon, and six practice-changing and exciting clinical abstracts to be presented in the Clinical Spotlight Session Saturday morning.To decompress partway through the meeting, I am delighted to invite you to join the ASGCT leadership and me for the 20th Anniversary Reception in the Great Hall of the Library of Congress. We will celebrate the 20th Anniversary of our Society with drinks, passed appetizers, and a commemorative cham-pagne toast in a stunning setting. Live music will be provided by member John Tisdale, our featured speaker Eric Olson, and their band. Tickets are available at the registration counter.Be sure to visit our Exhibit Hall to find out about the products and services of-fered by almost 70 partner companies, via conversations at their booth and more formal presentations in the Tools & Technologies Forum. Explore new op-portunities at the Employment Center. Finally, I would like to thank everyone involved in the planning and execution of our Annual Meeting, especially our wonderful ASGCT staff, along with the Program Committee, Scientific and Education Committees, Abstract review-ers, and other volunteer leaders. Their sustained support, dedication and hard work throughout the year now comes to fruition in what is sure to be a spec-tacular meeting. Lastly, thank you for attending our Society’s Annual Meeting. I sincerely hope you enjoy the science, technology, networking, and camaraderie that we have built together for the last 20 years!Best regards,

Cindy Dunbar, MD President, ASGCT

A WELCOME FROM CINDY DUNBAR, MD


EXHIBITOR BOOTH NUMBER

ABL, Inc. 610Advanced Cell Diagnostics, Inc. 207AGTC 501Akron Biotech 811Aldevron 502Alere Technologies AS (formerly Axis-Shield)

413

Alfa Wassermann Separation Technologies

604

ALS Automated Lab Solutions GmbH 102American Society of Gene & Cell Therapy

817

Applied StemCell, Inc. 205ArcticZymes 715Be The Match BioTherapies 105BioSpherix Medical 704Brammer Bio, LLC 505Cell Press 809CEVEC Pharmaceuticals GmbH 806ChemoMetec, Inc. 311Children’s Hospital of Philadelphia 401City of Hope 301Cobra Biologics 613Fresenius Kabi USA, LLC 611FUJIFILM Diosynth Biotechnologies 208GeneWerk GmbH 410Genezen Laboratories, Inc. 813GenoSafe 714GenScript USA Inc. 510GSK 202 &

204Imanis Life Sciences, LLC 504Intellicyt, A Sartorius Company 103Juno Therapeutics 803 &

805KBI Biopharma, Inc. 203Kite Pharma, Inc. 808LEROY Biotech 614Logos Biosystems 503Lonza 709

ANNUAL MEETING EXHIBITORS

The American Society of Gene & Cell Therapy would like to acknowledge and thank the following exhibitors for their participation at the ASGCT 20th

Annual Meeting. ASGCT greatly appreciates their support and encourages all attendees to make time each day to visit each of these exhibitors in Exhibit Hall A.

EXHIBITOR BOOTH NUMBER

Lovelace Biomedical 305MAK-SYSTEM Corp 309 &

408Malvern Instruments 405Mary Ann Liebert, Inc. 403MaxCyte 302Michelson Prize & Grants 711MilliporeSigma 615Miltenyi Biotec Inc. 402 &

404MPI Research 602National Gene Vector Biorepository (NGVB)

303

Nature Technology Corp. 308New England Biolabs 201Norgen Biotek Corp. 712NOVASEP 815Oxford BioMedica 810Pall Life Sciences 713Paragon Bioservices, Inc. 513 &

515PeproTech, Inc. 700Phoenix Bio USA Corporation 512Polyplus-transfection 304Powell Gene Therapy Center 310Precision NanoSystems Inc. 107Puresyn, Inc. 511

Sangamo Therapeutics 814Spark Therapeutics 702STEMCELL Technologies Inc. 605Terumo BCT 703Thermo Fisher Scientific 209TriLink BioTechnologies 101Vigene Biosciences 411Virovek Incorporation 710VIVEBIOTECH S.L. 812Waisman Biomanufacturing 601Wilson Wolf 603WuXi AppTec 104


EXHIBIT HALL FLOOR PLAN

ASGCT 20TH ANNUAL MEETINGMay 10-13, 2017 • Marriott Wardman Park HotelExhibit Hall A & B South


ANNUAL MEETING SUPPORTERS

PartnerMaxCyte Lunch Symposium, Registration Bags

Spark Therapeutics Conference WiFi

University of Massachusetts Medical School20th Anniversary Reception, Minority Travel Grants

ContributorAdverumLanyards, Excellence in Research Award, Travel GrantsApplied Genetic Technologies CorporationPens, Excellence in Research Awards, Travel Grants, Registration Kiosksbluebird bioCommercialization Workshop, Excellence in Research Award, Travel Grants, General Meeting Support

Brammer Bio, LLCUSB Drives, Excellence in Research Awards

EditasMobile App

Juno TherapeuticsConference Grant Support

Miltenyi Biotec Inc.Evening Symposium

National Institutes of HealthConference Grant SupportFunding for this conference was made possible (in part) by Grant Number 1R13TR002018-01 from the National Center for Advancing Translational Sciences NCATS).

NovartisHotel Key Cards

Oxford BioMedicaPresidential Symposium

PfizerConference Grant Support

Seattle Children’s Escalator Clings, Hand-Out

Terumo BCTLunch Symposium


ANNUAL MEETING SUPPORTERS, CONTINUED

PatronACGT Mentoring Event

AldevronMedia Partner

Alliance for Regenerative Medicine Commercialization Workshop Cocktail Reception

Alpha-1 FoundationConference Grant Support

Genezen Laboratories, Inc.Coffee Mugs

GenVec Pens

Growing Gene and Cell Therapy (GGACT) CooperativeClinical Trials Training Course

March of Dimes Conference Grant Support

Mary Ann Liebert, Inc.Excellence in Research Award

MDPI – Genes JournalOutstanding New Investigator Award

Molecular PharmaceuticsExcellence in Research Award

O.D.260 IncTravel Grants

PCT (Caladrius)Delegate Notepad

Plasmid FactoryExcellence in Research Award

REGENXBIOHand-Out

Sangamo Therapeutics, Inc. Commercialization Workshop Cocktail Reception

UniQureHand-Outs, Travel Grants


HOTEL FLOOR PLAN

• Employment Center (Hall A & B South)

• Exhibit Hall (Hall A & B South)

• Poster Hall (Hall A & B South)

• Session Rooms - Lincoln Rooms (Education Sessions, Oral Abstract Sessions, Scientific Symposia)

EXHIBITION LEVEL


HOTEL FLOOR PLAN

EXHIBITION LEVEL

HOTEL FLOOR PLAN

LOBBY LEVEL

• Plenary Lecture Hall - Salons 1-3• Session Rooms (Education Sessions, Oral Abstract Sessions,

Scientific Symposia)• Committee Meetings (Park Tower Rooms)• Registration - Convention Registration Desk• Speaker Ready - Convention Registration Desk


HOTEL FLOOR PLAN

• Committee Meetings• Industry Symposia - Thurgood

Marshall Northeast• Session Rooms (Education Sessions,

Oral Abstract Sessions, Scientific Symposia)

• Trainee Lounge - Harding Room

MEZZANINE LEVEL


PROGRAM SCHEDULE • WEDNESDAY MAY 10W

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Trainee Lounge7:00 am – 5:30 pm Room: Harding The Trainee Lounge is reserved for Students and Trainees as a designated spot to network, grab a refreshing snack and beverage and meet other students and trainees. Members and Non-members are welcome!

Education Session 1008:00 am - 9:45 amRoom: Marriott Salon 1Getting Started in Genome EditingCO-CHAIRS: Toni Cathomen, PhD and Thomas Wechsler, PhDSPEAKERS

8:00 am – 8:35 am Jacob Corn, PhD. UC Berkeley, Berkeley, CA, Innovative Genomics Institute, Berkeley, CAHigh Efficiency Genome Editing with CRISPR-Cas

8:35 am – 9:10 am Mitchell J. Weiss, MD, PhD. St. Jude Children’s Research Hospital, Memphis, TNMoving Editing Toward the Clinic

9:10 am – 9:45 am Laurie Zoloth, PhD. Northwestern University, Evanston, ILSecond Draft: Ethical and Theological Issues in Human Gene Editing

Education Session 1018:00 am - 9:45 amRoom: Delaware ABIntroducing Therapeutic ExosomesCO-CHAIRS: Eloise Hudry, PhD and Bakhos A. Tannous, PhDSPEAKERS

8:00 am – 8:35 am Samir A. El Andaloussi, PhD. Karolinska Institute, Huddinge, SwedenPreparation and Characterization of Extracellular Vesicles for Therapeutic Applications



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017 8:35 am – 9:10 am

Joshua N. Leonard, PhD. Northwestern University, Evanston, ILLoading of Extracellular Vesicles with Nucleic Acids

9:10 am – 9:45 am Alissa Weaver, MD, PhD. Vanderbilt University School of Medicine, Nashville, TNTrafficking of Extracellular Vesicles: Biogenesis, Release, Uptake and Fate

Education Session 1028:00 am - 9:45 amRoom: Lincoln 5,6Expanding Indications and Varieties of Engineered T CellsCO-CHAIRS: Avery D. Posey Jr., PhD and Blythe D. Sather, PhDSPEAKERS

8:00 am – 8:35 am Rebecca Gardner, MD. Seattle Children’s Hosptial, Seattle, WA, University of Washington, Seattle, WACAR-T Cells for Blood Cancers

8:35 am – 9:10 am Christine Brown, PhD. City of Hope National Medical Center, Duarte, CACAR T-Cells for Brain Tumors

9:10 am – 9:45 am James L. Riley, PhD. The University of Pennsylvania, Philadelphia, PACAR-T Cell Approaches as part of an HIV Cure Strategy

Scientific Symposium 1038:00 am - 9:45 amRoom: Lincoln 2,3,4The Impact of Bioinformatics Capabilities on Product Characterization - Organized by the Bio Industry Liaison CommitteeCO-CHAIRS: Mark L. Bonyhadi, PhD and Robert E. Sobol, MDSPEAKERS

8:00 am – 8:35 am Garry Nolan, PhD. Stanford University School of Medicine, Stanford, CACyTof for Diagnosis and Monitoring of Treatment of Hematologic Malignancies

8:35 am – 9:10 am Rong Fan, PhD. Yale University, New Haven, CTSingle Cell 42-Plex Cytokine Analysis to Monitor Immune Therapies

9:10 am – 9:45 am Frederic D. Bushman, PhD. University of Pennsylvania Perelman School of Medicine, Philadelphia, PAInsertion Site Analysis as a Component of Gene Therapy Product Characterization



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Scientific Symposium 1048:00 am - 9:45 amRoom: Virginia ABCTargeting Non-coding RNA in Cardiovascular Disease - Organized by the Cardiovascular Gene & Cell Therapy CommitteeCO-CHAIRS: Charles R. Bridges, MD, ScD and Michael A. Laflamme, MD, PhDSPEAKERS

8:00 am – 8:35 am Andrew H. Baker, PhD. University of Glasgow, Glasgow, United Kingdom, University of Edinburgh, Edinburgh, United KingdomRole of Non-coding RNAs in Vascular Pathology and Therapy

8:35 am – 9:10 am Thomas Thum, MD, PhD. Hannover Medical School, Hannover, GermanyTreating Heart Failure via Non-coding RNAs

9:10 am – 9:45 am Mark W. Feinberg, MD. Brigham and Women’s Hospital/ Harvard Medical School, Boston, MATargeting MicroRNAs in Atherosclerosis

Scientific Symposium 1058:00 am - 9:45 amRoom: Maryland ABCPhysical Vector Delivery for Immunotherapy - Organized by the Physical Delivery, Therapeutics & Vector Development CommitteeCO-CHAIRS: Laurent Humeau, PhD and Ernst Wagner, PhDSPEAKERS

8:00 am – 8:35 am Flordeliza S. Villanueva, MD. University of Pittsburgh, Pittsburgh, PATherapeutic Applications & Mechanisms of Sonoporation

8:35 am – 9:10 am Cornelia L. Trimble, MD. Johns Hopkins University, Baltimore, MDThe Electroporated Human Papilloma Virus DNA Vaccine Trial

9:10 am – 9:45 am Kevin Hollevoet, PhD. KU Leuven – Leuven University, Leuven, BelgiumDNA-based Antibody Therapy via In Vivo Electrotransfer

Coffee Break9:45 am - 10:15 am



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017 Oral Abstract Session 110

10:15 am - 12:15 pmRoom: Marriott Salon 1AAV Vector BiologyCO-CHAIRS: Casey A. Maguire, PhD and R. Jude Samulski, PhDPRESENTERS

10:15 AM 1: Wild-Type and Recombinant AAV Integration in Human Cardiomyocytes: Focus on Mitochondrial GenomeJessika Ceiler, German Cancer Research Center and National Center for Tumor Diseases, Heidelberg, Germany

10:30 AM 2: Novel AAV Vector Reservoirs: Peripheral Blood Cells and Hematopoietic ProgenitorsIrene Gil-Farina, German Cancer Research Center and National Center for Tumor Diseases, Heidelberg, Germany

10:45 AM 3: Role of Truncated Recombinant AAV Genomes in Tumor FormationPatrick Westmoreland, The Ohio State College of Medicine, Columbus, OH

11:00 AM 4: Assaying Patterns of rAAV Integration in Humanized Mice: Implications for rAAV GenotoxicityStephanie Smith, National Institutes of Health, Bethesda, MD

11:15 AM 5: The Multi-Serotype Receptor AAVR Interacts with AAV2 and AAV5 via Separate DomainsJianming Qiu, University of Kansas Medical Center, Kansas City, KS

11:30 AM 6: A Single Amino Acid in AAV Capsids Regulate the Requirement of the Assembly Activating Protein (AAP) for AssemblySwapna Kollu, Oregon Health & Science University, Portland, OR

11:45 AM 7: Evolutionary Interrogation of AAP-Dependency on AAV Assembly Highlights Mechanism and Structural DeterminantsAnna Maurer, Harvard Medical School, Boston, MA

12:00 PM 8: Vector Dose-Dependent Delayed CD8+ T Cell-Mediated Clearance of AAV Encoded Antigen in LiverSandeep Kumar, University of Florida, Gainesville, FL



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Oral Abstract Session 11110:15 am - 12:15 pmRoom: Virginia ABCOncolytic Viruses for the Treatment of CancerCO-CHAIRS: David L. Bartlett, MD, Paola Grandi, PhD and David Kirn, MDPRESENTERS

10:15 AM 9: Oncolytic Measles Viruses as Therapeutic Vectors for Tumor-Directed BiTE ExpressionTobias Speck, National Center for Tumor Diseases, Heidelberg, Germany

10:30 AM 10: Immune System as a Determinant for Response to Oncolytic Measles VirotherapyCheyne Kurokawa, Mayo Clinic, Rochester, MN

10:45 AM 11: Type 1 IFN Response Is a Major Determinant of Oncolytic Measles Virus Activity in a Model of Sequential Stromal Cell TransformationSarah Aref, University College London (UCL), Cancer Institute, London, United Kingdom

11:00 AM 12: Combinatorial Treatment of “Armed” Oncolytic Adenovirus Expressing Checkpoint Inhibitor and Cytokine with Chimeric Antigen Receptor T-Cells Leads to Superior Anti-Tumor Effects in Head and Neck CancerAmanda Rosewell Shaw, Baylor College of Medicine, Houston, TX

11:15 AM 13: Targeting Historically Recalcitrant Colorectal Cancer Cells: Novel Oncolytic Chimeric Poxvirus Is Imageable and More Potent Than PredecessorsSusanne Warner, City of Hope, Beckman Research Institute, Duarte, CA

11:30 AM 14: Development of the Novel miRNA Engineered Oncolytic Virotherapy for Clinical TrialYang Jia, The University of Tokyo, Tokyo, Japan

11:45 AM 15: Immune Stimulation and Immune Checkpoint Nhibition Provided by a Single Agent: An Oncolytic HSV Expressing scFv Antibody Against PD-1Carmela Passaro, Brigham and Women’s Hospital, Boston, MA

12:00 PM 16: Phase 2 Trial Evaluating Biodistribution and Shedding of Talimogene Laherparepvec (T-VEC) in Patients (Pts) with Unresectable Stages IIIB/IV MelanomaRobert Andtbacka, Huntsman Cancer Institute, Salt Lake City, UT



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10:15 am - 12:15 pmRoom: Lincoln 2,3,4Genome Editing: Transcriptional Regulation and SpecificityCO-CHAIRS: Prashant Mali, PhD and Pratiksha ThakorePRESENTERS

10:15 AM 17: Activity and Specificity of Engineered CRISPR-Cpf1 Nucleases in Human CellsBenjamin Kleinstiver, Massachusetts General Hospital, Charlestown, MA

10:30 AM 18: Targeted Multiplex Regulation of Endogenous Human Genes Using Drug-Inducible CRISPR-Cpf1-Based Transcription FactorsYuGyoung Tak, Massachusetts General Hospital, Charlestown, MA

10:45 AM 19: Improvement of CRISPR Activity and Specificity via Proximal Binding of Multiple CRISPR/Cas Systems (proxy-CRISPR)Gregory Davis, MilliporeSigma, St. Louis, MO

11:00 AM 20: High-Throuput Screening Selection of microRNAs Enhancing Cas9-Mediated Homologous RecombinationLorena Zentilin, ICGEB, Trieste, Italy

11:15 AM 21: dCas9 Epigenome Editing Suggests Histone Methylation Does Not Always Precede Target Gene RepressionHenriette O’Geen, University of California, Davis, Davis, CA

11:30 AM 22: Easy and Efficient Gene Editing of Mammalian Embryos Using rAAV Vectors - From Mouse to MonkeyDan Wang, University of Massachusetts Medical School, Worcester, MA

11:45 AM 23: New Zinc Finger Nuclease Architectures for Highly Efficient Genome Engineering in Primary Cells at Large Scale with No Detectable Off-Target EffectsEdward Rebar, Sangamo Therapeutics, Richmond, CA

12:00 PM 24: Sustained Tau Reduction via Zinc Finger Protein Transcription Factors as a Potential Next-Generation Therapy for Alzheimer’s Disease and Other TauopathiesBryan Zeitler, Sangamo Therapeutics, Inc., Richmond, CA



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Oral Abstract Session 11310:15 am - 12:15 pmRoom: Lincoln 5,6Genome Editing and Integration Analysis in Metabolic and Endocrine DisordersCO-CHAIRS: Randy J. Chandler, PhD, MB and Pasqualina Colella, PhDPRESENTERS

10:15 AM 25: Nuclease-Mediated Allelic Exchange as a Therapeutic Strategy to Repair Recessive Compound Heterozygous Mutations: Proof-of-Concept in a New Mouse Model of TyrosinemiaDan Wang, University of Massachusetts Medical School, Worcester, MA

10:30 AM 26: Rescue of Mice with Methylmalonic Acidemia from Immediate Neonatal Lethality Using an Albumin Targeted, Promoterless Adeno-Associated Viral Integrating VectorRandy Chandler, National Institutes of Health, Bethesda, MD

10:45 AM 27: Liver-Based Expression of the Human alpha-Galactosidase A Gene in a Murine Fabry Model Results in Continuous High, Therapeutic Levels of Enzyme Activity and Effective Substrate ReductionThomas Wechsler, Sangamo Therapeutics, Richmond, CA

11:00 AM 28: Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic MiceShen Shen, Editas Medicine, Cambridge, MA

11:15 AM 29: Somatic Genome Editing with AAV Vectors Generates and Corrects a Metabolic DiseaseWilliam Lagor, Baylor College of Medicine, Houston, TX

11:30 AM 30: ZFN-Mediated In Vivo Genome Editing Results in Phenotypic Correction in MPS I and MPS II Mouse ModelsRussell DeKelver, Sangamo Therapeutics, Richmond, CA

11:45 AM 31: Molecular Characterization of Hematopoietic System Reconstitution in 7 Metachromatic Leukodystrophy Patients Following Hematopoietic Stem Cell Gene TherapyAndrea Calabria, San Raffaele Telethon Institute for Gene Therapy, Milan, Italy

12:00 PM 32: Effect of AAV Gene Therapy on Hepatic Tumor Induced by Deficiency of Glucose-6-PhosphataseHye Ri Kang, Duke University, Durham, NC



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10:15 am - 12:15 pmRoom: Maryland ABCGene Therapies for Musculoskeletal DiseasesCO-CHAIRS: Carsten Bonnemann, MD and Jerry R. Mendell, MDPRESENTERS

10:15 AM 33: Systemic Gene Editing for Muscular Dystrophy Using AAV-CRISPR/Cas9Niclas Bengtsson, University of Washington, Seattle, WA

10:30 AM 34: Dystrophin Restoration in a Humanized Mouse Model of Duchenne Muscular Dystrophy by Gene Editing with S. aureus Cas9Jacqueline Robinson-Hamm, Duke University, Durham, NC

10:45 AM 35: Efficient In Vivo Correction of a Splicing Defect Using an HDR-Independent MechanismEvgueni Ivakine, HSC, Toronto, ON, Canada

11:00 AM 36: Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Functional Improvement in Dysf/- Mouse Model of LGMD2BDanielle Griffin, The Research Institute at Nationwide Childrens Hospital, Columbus, OH

11:15 AM 37: Systemic Injections of Peptide-Conjugated Morpholinos Improve Cardiac Symptoms of a Dog Model of Duchenne Muscular DystrophyRika Maruyama, University of Alberta, Edmonton, AB, Canada

11:30 AM 38: Evaluation of Re-Administration of a Recombinant Adeno-Associated Vector Expression Acid Alpha-Glucosidase (rAAV9-DES-hGAA) in Pompe Disease: Preclinical to Clinical PlanningManuela Corti, University of Florida, Gainesville, FL

11:45 AM 39: Prolonged Exon 2 Skipping and Robust Dystrophin Expression 1 Year Post Single Neonatal Injection of an AAV9.U7snRNA Vector in the Dup2 MouseNicolas Wein, Nationwide Children’s Hospital, Columbus, OH

12:00 PM 40: Toxicity & Biodistribution Study Demonstrates Safety of AAV-Mediated Human Beta-Sarcoglycan Gene TherapyEric Pozsgai, Nationwide Children’s Hospital, Columbus, OH



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Oral Abstract Session 11510:15 am - 12:15 pmRoom: Delaware ABSynthetic/Molecular Conjugates and Physical Methods of Gene Delivery ICO-CHAIRS: Yi Chen, PhD and Dexi Liu, PhDPRESENTERS

10:15 AM 41: Ultrasound Mediated Gene Delivery of High Expressing Factor VIII Variant Plasmids Combined with Immunomodulation Generated Long Term Therapeutic Levels of Factor VIII in Hemophilia A MiceCarol Miao, Seattle Children’s Research Institute, Seattle, WA

10:30 AM 42: Plasmid Delivery to the Skin Using Increased Temperature and Gene ElectrotransferRichard Heller, Old Dominion University, Norfolk, VA

10:45 AM 43: A High-Throughput Screening Platform to Identify Nanoparticle Formulations for Transfection of Primary and Post-Mitotic Differentiated CellsDavid Wilson, Johns Hopkins University, Baltimore, MD

11:00 AM 44: A Systematic Evaluation of Factors Affecting Extracellular Vesicle Uptake and Functional Cargo DeliveryDevin Stranford, Northwestern University, Evanston, IL

11:15 AM 45: Mucus-Penetrating Non-Viral Gene Delivery Platform for Obstructive Lung DiseasesNamho Kim, Johns Hopkins University School of Medicine, Baltimore, MD

11:30 AM 46: New Recombinant Fusion Gene for Treatment of Obesity and Metabolic DisordersMingming Gao, University of Georgia, Athens, GA

11:45 AM 47: Clostridium as “Trojan Horse” Vectors for Cancer TreatmentMaria Zygouropoulou, University Nottingham, Nottingham, United Kingdom

12:00 PM 48: Vector-Free Genome Editing of Primary Immune Cells for Cell TherapyDevin Bridgen, SQZ Biotech, Watertown, MA



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017 Industry Symposium 120

12:15 pm - 1:45 pmRoom: Thurgood Marshall NortheastThe Multiple Benefits of Using a Functionally Closed, Hollow-Fiber Bioreactor for Culturing T-Cells - Sponsored by Terumo BCT

SPEAKERSPatrick Hanley, PhD. Children’s National Health System, Washington, DCExploring the Boundaries of T-cell Production -- The Impact of Source Material, Seeding Density, and Media TypeJim Beltzer, PhD. Terumo BCT, USA, Lakewood, COExploring the Boundaries of T-cell Production -- The Impact of Source Material, Seeding Density, and Media Type

Break 12:15 pm - 1:45 pmLunch Break (On Own - Not Provided)

Education Session 1301:45 pm - 3:30 pmRoom: Maryland ABCUnderstanding Immune System Obstacles to Gene TherapiesCO-CHAIRS: Austin J. Barrett, MD and Andrew M. Scharenberg, MDSPEAKERS

1:45 pm – 2:20 pm Cor H. J. Lamers, PhD. Erasmus University Medical Center, Rotterdam, NetherlandsPreventing and Coping with Immune Responses against CAR-T Cells

2:20 pm – 2:55 pm Anne Galy, PhD. Genethon, Evry, France, Inserm, Evry, FranceThe Impact of Transgenes and Target Tissues on AAV Vector Immunogenicity

2:55 pm – 3:30 pm David Russell, MD, PhD. University of Washington, Seattle, WAAvoiding Rejection of Allogeneic Cell Therapies



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Education Session 1311:45 pm - 3:30 pmRoom: Virginia ABCPluripotent Regenerative Cell TherapiesCO-CHAIRS: Linzhao Cheng, PhD and Hans-Peter Kiem, MD, PhDSPEAKERS

1:45 pm – 2:20 pm Jizhong Zou, PhD. NHLBI, Bethesda, MDEngineering iPSC for Regenerative Medicine Applications

2:20 pm – 2:55 pm Kah-Whye Peng, PhD. Mayo Clinic, Rochester, MNWhere Are My Cells? Noninvasive Imaging of Regenerative Medicines

2:55 pm – 3:30 pm Kapil Bharti, PhD. National Eye Institute, Bethesda, MDDeveloping Autologous iPS Cell Therapy for Macular Degeneration

Education Session 1321:45 pm - 3:30 pmRoom: Delaware ABRNA TherapeuticsCO-CHAIRS: Richard P. Harbottle, PhD and Stephen L. Hart, PhDSPEAKERS

1:45 pm – 2:20 pm Drew Weissman, PhD. University of Pennsylvania, Philadelphia, PAIntroduction to Synthetic RNA Therapeutics

2:20 pm – 2:55 pm Luis A. Brito, PhD. Moderna Therapeutics Inc., Cambridge, MATranslating mRNA from Benchside to Bedside

2:55 pm – 3:30 pm Chantal Pichon, PhD. Center for Molecular Biophysics- CNRS, Orleans, FranceDesign and Delivery of Messenger RNA-based Vaccines



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017 Scientific Symposium 133

1:45 pm - 3:30 pmRoom: Lincoln 2,3,4Upgrading the Efficacy and Safety of CARs - Organized by the Cancer Gene & Cell Therapy CommitteeCHAIR: Richard Morgan, PhDSPEAKERS

1:45 pm – 2:20 pm Michel Sadelain, MD, PhD. Memorial Sloan Kettering Cancer Center, New York, NYIncreasing the Horsepower of CARs with CRISPR/Cas9

2:20 pm – 2:55 pm Evanthia Galanis, MD, DSc. Mayo Clinic, Rochester, MNCombination Viral and Immunotherapies for Glioblastoma

2:55 pm – 3:30 pm Sattva S. Neelapu, MD. The University of Texas MD Anderson Cancer Center, Houston, TXPredicting and Managing CAR-T Toxicities

Scientific Symposium 1341:45 pm - 3:30 pmRoom: Marriott Salon 1Getting Cures to Patients - Organized by the Ethics CommitteeCO-CHAIRS: Theodore Friedmann, MD and Timothy D. Hunt, JDSPEAKERS

1:45 pm – 2:20 pm Margaret Anderson FasterCures, Washington, DCFaster Cures: Accelerating New Treatments Forward to Patients

2:20 pm – 2:55 pm Martin Mense, PhD. Cystic Fibrosis Foundation Therapeutics, Lexington, MABench to Bedside Acceleration of Novel Therapies - The Cystic Fibrosis Foundation Approach

2:55 pm – 3:30 pm Lyric Jorgenson, PhD. Office of Science Policy, NIH, Bethesda, MDAchieving Lift-off with the Cancer Moonshot



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Scientific Symposium 1351:45 pm - 3:30 pmRoom: Lincoln 5,6New Frontiers in Gene and Cell Therapies for Metabolic Diseases, Aging and Tissue Regeneration - Organized by the Gene & Cell Therapy of Genetic and Metabolic Diseases CommitteeCO-CHAIRS: Dawn Blessing and Federico Mingozzi, PhDSPEAKERS

1:45 pm – 2:20 pm Maria A. Blasco, PhD. Spanish National Cancer Research Center - CNIO, Madrid, SpainGene Therapy for Telomere Loss Disorders

2:20 pm – 2:55 pm Fatima Bosch, PhD. Universitat Autonoma de Barcelona, Bellaterra - Barcelona, SpainManipulating Metabolism with Gene Therapy

2:55 pm – 3:30 pm Mauro Giacca, MD, PhD. International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, ItalyStimulating the Endogenous Regeneration Potential of the Heart

Coffee Break3:30 pm - 3:45 pm

Oral Abstract Session 1403:45 pm - 5:30 pmRoom: Marriott Salon 1Immunotherapy Clinical Trials for CancerCO-CHAIRS: Conrad Russell Y. Cruz, MD, PhD and Daniel J. Powell, PhDPRESENTERS

3:45 PM 49: EBV-Specific T Cells Outside of the HSCT SettingRayne Rouce, Baylor College of Medicine, Houston, TX

4:00 PM 50: Long-Term Follow-Up Data from 126 Patients with Recurrent High Grade Glioma from Three Phase 1 Trials of Toca 511 and Toca FC: Update and Justification for a Phase 2/3 TrialDoug Jolly, Tocagen Inc., San Diego, CA

4:15 PM 51: Induction of APOBEC Expression During Multiple Types of Immuno, Gene and Viral Therapies Enhances Tumor Cell EscapeChristopher Driscoll, Mayo Clinic, Rochester, MN

4:30 PM 52: Engineering T Cells and B Cells for Immunotherapy Using V(D)J RecombinationDaniel Nataf, Tel Aviv University, Tel Aviv, Israel



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017 4:45 PM 53: In Vivo Gene Transfer and Hepatocyte-Restricted

Transgene Expression in a Murine Model of On-Target Off-Tumor Cytotoxicity by CAR T Cell TherapyMauro Castellarin, University of Pennsylvania, Philadelphia, PA

5:00 PM 54: Disease Burden and Transplant on Long-Term Survival After CD19 CAR T Cells in Adults with Relapsed Acute Lymphoblastic LeukemiaJae Park, Memorial Sloan Kettering Cancer Center, New York, NY

5:15 PM 55: CARs in LeukemiaJun Xu, University of Pennsylvania, Philadelphia, PA

Oral Abstract Session 1413:45 pm - 5:30 pmRoom: Delaware ABCardiovascular and Pulmonary Gene and Cell TherapiesCO-CHAIRS: W. Todd Cade, PT, PhD and Jennifer L. Gori, PhDPRESENTERS

3:45 PM 56: hsa-miR-665 Prevents Cardiomyocyte Hypertrophy and Preserve Normal Cardiac Function After Pressure OverloadMauro Giacca, ICGEB, Trieste, Italy

4:00 PM 57: CRISPR/Cas9-Mediated Introduction of the Sodium-Iodide Symporter Gene Enables Non-Invasive In Vivo Tracking of Rhesus iPSC-Derived CellsJohn Ostrominski, National Institutes of Health (NIH), Bethesda, MD

4:15 PM 58: Systemic Delivery of AAVB1-GAA Gene Therapy for Respiratory Pathology in Pompe DiseaseMai ElMallah, University of Massachusetts Medical School, Worcester, MA

4:30 PM 59: Cell-Based Therapy of Airway Infections Utilizing iPSC-Derived Macrophages Produced in BioreactorsMania Ackermann, Hannover Medical School (MHH), Hannover, Germany

4:45 PM 60: Pre-Clinical Development of AAV-Mediated TAZ Gene Delivery to Treat Barth SyndromeSilveli Suzuki-Hatano, University of Florida, Gainesville, FL

5:00 PM 61: Proof-of-Concept for Non-Nuclease-Mediated Genome Editing to Treat a-1 Antitrypsin DeficiencyFlorie Borel, University of Massachusetts Medical School, Worcester, MA

5:15 PM 62: Development of NSC-Mediated Enzyme/Prodrug Therapy for Small Cell Lung CancerMeher Masihi, City of Hope National Medical Center, Duarte, CA



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Oral Abstract Session 1423:45 pm - 5:30 pmRoom: Lincoln 2,3,4Gene and Cell Therapies for Hematologic and Immunologic Diseases ICO-CHAIRS: Bobby Gaspar, MD, PhD and Andrew Wilber, PhDPRESENTERS

3:45 PM 63: Efficacy and Safety of Liver-Directed Lentiviral Gene Therapy in Hemophilia B Dogs and Non-Human PrimatesAlessio Cantore, San Raffaele Telethon Institute for Gene Therapy, Milan, Italy

4:00 PM 64: CD20 Receptor Targeted Lentiviral Gene Transfer of IGG-Fusion Protein into B Cells to Induce Tolerance in Hemophilia B MiceMoanaro Biswas, University of Florida, Gainesville, FL

4:15 PM 65: T Cell Gene Therapy Corrects Humoral and Cytotoxic Defects in X-Linked Lymphoproliferative Disease (XLP)Claire Booth, UCL GOSH Institute of Child Health, London, United Kingdom

4:30 PM 66: Expansion of Human T Regulatory Cells by Lentiviral Vector Mediated Expression of STAT5B or BACH2 Transcription FactorsDaniela Cesana, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy

4:45 PM 67: Development of Gene Editing Strategies Aimed at Inducing Fetal Hemoglobin for the Treatment of Hemoglobinopathies Using the Nonhuman Primate ModelOlivier Humbert, Fred Hutchinson Cancer Research Center, Seattle, WA

5:00 PM 68: Cytoreductive Conditioning Intensity Predicts Clonal Diversity in ADA-SCID Retroviral Gene Therapy PatientsAaron Cooper, University of California, Los Angeles, Los Angeles, CA

5:15 PM 69: A Diversity of Human Hematopoietic Differentiation Programs Identified Through In Vivo Tracking of Hematopoiesis in Wiskott-Aldrich Syndrome PatientsEmmanuelle Six, Imagine Institute, Paris, France



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3:45 pm - 5:30 pmRoom: Maryland ABCOligonucleotide TherapeuticsCO-CHAIRS: Marcin Kortylewski, PhD and Paul Valdmanis, PhDPRESENTERS

3:45 PM 70: Mechanism and In Vivo Activity of a Small Activating RNA Targeting CEBPA, a Novel Therapeutic in Clinical Trials for Liver DiseaseJon Voutila, MiNA Therapeutics Ltd, London, United Kingdom

4:00 PM 71: A 3’ tRNA Derived Small RNA (tsRNA) Affects Translation in Rapidly Dividing Cells and a Target for Hepatocellular CarcinomaHakkyun Kim, Stanford Univ, Stanford, CA

4:15 PM 72: TLR9-Targeted Systemic Delivery of CpG-STAT3 Antisense Oligonucleotides Induces Regression of Bone-Localized Prostate Tumors in MiceDayson Moreira, Beckman Research Institute at City of Hope, Duarte, CA

4:30 PM 73: Treatment of Sepsis by Neutralization of Extracellular Histones with Nucleic Acid AptamersKevin Urak, University of Iowa, Iowa City, IA

4:45 PM 74: Modulation of Pro-Inflammatory IL-6 Trans Signalling Axis by Splice Switching Antisense Oligonucleotides as a Therapeutic Modality in InflammationDhanu Gupta, Karolinska Instituet, Stockholm, Sweden

5:00 PM 75: Artificial miRNAs Reduce Human Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington’s DiseaseEdith Pfister, UMass Medical School, Worcester, MA

5:15 PM 76: Small Hairpin RNAs Delivered in Human Cortical Spheroids Compete with Endogenous microRNAsPaul Valdmanis, Stanford University, Stanford, CA

Oral Abstract Session 1443:45 pm - 5:30 pmRoom: Lincoln 5,6RNA Virus VectorsCO-CHAIRS: Axel Schambach, MD, PhD and Bruce Torbett, PhD, MSPHPRESENTERS

3:45 PM 77: Lentiviral Vectors Escape Innate Sensing but Trigger p53 in Human Hematopoietic Stem and Progenitor CellsFrancesco Piras, SR-TIGET, IRCCS San Raffaele Scientific Institute, Milan, Italy



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4:00 PM 78: LTR1 Vectors Enhance Safety in Gene Therapy and Can Be Exploited for Rapid, Transient Gene DeliveryJohn Counsell, University College London, London, United Kingdom

4:15 PM 79: Generation of Immune Stealth Lentiviral Vectors by Producer Cell Genome EditingMichela Milani, San Raffaele-Telethon Institute for Gene Therapy, Milan, Italy

4:30 PM 80: BaEV-LVs Efficiently Transduce HSCs-Derived Human Progenitor T Cells, Acceleratingt Cell Reconstitution In Vivo and Allowed Correction of X-SCID Progenitor T CellsEls Verhoeyen, INSERM /CIRI/C3M, Lyon, France

4:45 PM 81: Multidimensional Vector Interaction Capture to Study the Effects of SIN.LVs and Chromatin Insulators on the Cellular GenomeMonica Volpin, San Raffaele Telethon Institute for Gene Therapy, Milan, Italy

5:00 PM 82: Library Screening for Novel Chromatin Insulator Elements for Use in Gene TherapySheng Zhou, St Jude Children’s Research Hospital, Memphis, TN

5:15 PM 83: Gammaretroviral and Lentiviral Vector Based Insertional Mutagenesis Screens to Identify Prostate Cancer Therapeutic TargetsJonah Hocum, Washington State University, Spokane, WA

Oral Abstract Session 1453:45 pm - 5:30 pmRoom: Virginia ABCEngineering and Manufacturing Vectors and Cells ICO-CHAIRS: Larry A. Couture, PhD and Rimas Orentas, PhDPRESENTERS

3:45 PM 84: Novel Manufacturing of Gene Corrected Autologous Blood Stem Cells for Gene Therapy of Fanconi Anemia Complementation Group AJennifer Adair, Fred Hutch, Seattle, WA

4:00 PM 85: Building a Better T-Cell: T Cell Process Development by Multifactorial Design of Experiments Approach and High Throughput Automated Functional AssaysLan Cao, Unum Therapeutics Inc, Cambridge, MA

4:15 PM 86: Sleeping Beauty (SB) Transposition Integration Events Mediated by SB Transposase from mRNA and CAR from SB DNA for T-Cell TherapySourindra Maiti, M.D. Anderson Cancer Center, Houston, TX



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Regulation of GDNF Expression In VivoLuis Quintino, Lund University, Lund, Sweden

4:45 PM 88: S/MAR Based Nano-Vectors: Novel Tools for the Genetic Modification of Patient-Derived Cancer CellsMatthias Bozza, DKFZ Heidelberg, Heidelberg, Germany

5:00 PM 89: RNA Aptamers Selected Through Blind-SELEX Inhibit Pancreatic Cancer Cell Metastasis and Invasion by Regulating Epithelial Mesenchymal Transition(EMT)Sorah Yoon, Beckman Research Institute of City of Hope, Duarte, CA

5:15 PM 90: Bioengineering Gene Therapy Vectors for Hemophilia A Through Ancestral Sequence ReconstructionChristopher Doering, Emory University School of Medicine, Atlanta, GA

Exhibit Hall Welcome Reception & Poster Session 15:30 pm - 7:30 pmRoom: Exhibit Hall A & B South

Reconnection and Mentoring Event 7:30 pm – 9:30 pm Room: Marriott Salon 1 Sponsored by ACGT

Industry Symposium 1507:30 pm – 9:30 pmRoom: Thurgood Marshall NortheastSponsored by Miltenyi Biotec Inc.


PROGRAM SCHEDULE • THURSDAY, MAY 11THURSDA

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Education Session 2008:00 am - 9:45 amRoom: Lincoln 5,6The ABCs of AAVCO-CHAIRS: Aravind Asokan, PhD and Dirk Grimm, PhDSPEAKERS

8:00 am – 8:35 am Guangping Gao, PhD. University of Massachusetts Medical School, Worcester, MAThe Library of AAV Serotypes: Choosing the Right One for your Application

8:35 am – 9:10 am Matthew L. Hirsch, PhD. University of North Carolina at Chapel Hill, Chapel Hill, NDModifying the AAV Genome for Gene Therapy Applications

9:10 am – 9:45 am Federico Mingozzi, PhD. University Pierre and Marie Curie and Genethon, Evry, FranceCellular Immunity to AAV and What to Do About It

Education Session 2018:00 am - 9:45 amRoom: Virginia ABCUpdates on Gene Therapies for Non-Malignant Diseases of the Immune SystemCO-CHAIRS: Bobby Gaspar, MD, PhD and Elizabeth Kang, MDSPEAKERS

8:00 am – 8:35 am Bruce L. Levine, PhD. University of Pennsylvania, Philadelphia, PAEngineering Synthetic Immunity and Permanent HIV Resistance

8:35 am – 9:10 am Suk See De Ravin, MD, PhD. Laboratory of Host Defenses, NIAID, National Institutes of Health, Bethesda, MDGene Therapies for Immunodeficiency Disorders


PROGRAM SCHEDULE • THURSDAY, MAY 11TH

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9:10 am – 9:45 am Luca Biasco, PhD. Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Boston, MAClonal Tracking of Human Hematopoiesis Through Integration Sites Analysis

Education Session 2028:00 am - 9:45 amRoom: Delaware ABOncolytics from the Bench to BedsideCO-CHAIRS: Daniel J. Powell, PhD and Masato Yamamoto, MD, PhDSPEAKERS

8:00 am – 8:35 am John C. Bell, PhD. Ottawa Hospital Research Institute, Ottawa, ON, CanadaThe Science Behind the Use of Oncolytics for Cancer

8:35 am – 9:10 am Noriyuki Kasahara, MD, PhD. University of Miami, Miami, FLPathways for Translational Development of Oncolytic Viruses

9:10 am – 9:45 am Stephen J. Russell, MD, PhD. Mayo Clinic, Rochester, MNOncolytics in the Clinic

Scientific Symposium 2038:00 am - 9:45 amRoom: Marriott Salon 1Exosomes Offer New Therapeutic DimensionsCO-CHAIRS: Xandra O. Breakefield, PhD and Amine MelianiSPEAKERS

8:00 am – 8:35 am Anastasia Khvorova, PhD. University of Massachusetts Medical School, Worcester, MAExosomes as Therapeutic RNA Delivery Vehicles

8:35 am – 9:10 am Casey A. Maguire, PhD. Massachusetts General Hospital, Boston, MAExosome-associated AAV Vectors for Gene Delivery and Therapy of Inner Ear Disorders

9:10 am – 9:45 am Jan Lotvall, MD, PhD. Codiak BioSciences Inc., Cambridge, MARNAi Targeting MYC in Cancer



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Scientific Symposium 2048:00 am - 9:45 amRoom: Lincoln 2,3,4Therapeutic Editing of the Human Genome and Epigenome - Organized by the Genome Editing CommitteeCO-CHAIRS: Michael C. Holmes, PhD and J. Keith Joung, MD, PhDSPEAKERS

8:00 am – 8:35 am Shengdar Q. Tsai, PhD. St. Jude Children’s Research Hospital, Memphis, TNDefining Genome-wide Off-target Effects of CRISPR-Cas Nucleases

8:35 am – 9:10 am Angelo Lombardo, PhD. San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Milan, ItalyTargeted Epigenome Editing for Therapeutic and Basic Applications

9:10 am – 9:45 am Jennifer L. Gori, PhD. Editas Medicine, Cambridge, MACRISPR/Cas9-mediated Editing of Hematopoietic Stem Cells for the Treatment of β-hemoglobinopathies

Scientific Symposium 2058:00 am - 9:45 amRoom: Maryland ABCImmune Models to Predict Toxicity and Efficacy of Gene Therapies - Organized by the Immune Responses to Gene & Cell Therapy CommitteeCO-CHAIRS: Antonia Follenzi, MD, PhD and Pawel J. Muranski, MDSPEAKERS

8:00 am – 8:35 am Paola Grandi, PhD. University of Pittsburgh School of Medicine, Pittsburgh, PADeveloping Oncolytic Therapies for Brain Tumors

8:35 am – 9:10 am Leslie S. Kean, MD, PhD. Seattle Children’s Hospital, Seattle, WAModeling Immunotherapies in Non-Human Primates

9:10 am – 9:45 am James M. Wilson, MD, PhD. University of Pennsylvania, Philadelphia, PAAAV-Associated Hepatotoxicity



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Exhibit Hall Coffee Social - with Oral Poster Session9:45 am - 10:30 amRoom: Exhibit Hall A & B SouthOral Poster SessionCHAIR: Luk H. Vandenberghe, PhDSPEAKERS

9:45 am – 10:00 am Nicole K. Paulk, PhD. Stanford University, Stanford, CArAAV Is Extensively and Differentially Post-Translationally Modified in Human versus Insect Cell Line Production Methods

10:00 am – 10:15 am Veronique Bolduc, PhD. National Institute of Neurological Disorders and Stroke/NIH, Bethesda, MDA Dominant-Negative COL6A1 Pseudoexon Insertion Is Skippable Using Splice-Modulating Oligonucleotides

10:15 am – 10:30 am Kahori Shimizu, PhD. Osaka Ohtani University, Osaka, JapanAdenovirus Vector-Induced Hepatotoxicity During the Early Phase of Adenoviral Treatment Is Attributed to Inflammatory Cytokine-Induced Leaky Expression of Adenovirus Genes

Plenary Session 21010:30 am - 11:45 amRoom: Marriott Salon 1,2,3George Stamatoyannopoulos Lecture & Presentation of the Excellence in Research Awards & Sonia Skarlatos Public Service Award PresentationCHAIR: Cynthia Dunbar, MDSPEAKER

Eric Olson, PhD. UT Southwestern Medical Center at Dallas, Dallas, TXCorrection of Duchenne Muscular Dystrophy by Genomic Editing

Break 11:45 am - 1:15 pmLunch Break (On Own - Not Provided)



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Plenary Session 2301:15 pm - 2:25 pmRoom: Marriott Salon 1,2,3Outstanding New Investigator SymposiumCHAIR: Beverly L. Davidson, PhDSPEAKERS

1:15 pm – 1:50 pm James N. Kochenderfer, MD. National Cancer Institute, Bethesda, MDChimeric Antigen Receptor T-cell Therapies for Hematologic Malignancies

1:50 pm – 2:25 pm Lili Yang, PhD. University of California, Los Angeles, Los Angeles, CAStem Cell-Engineered T Cell Immunotherapy for Cancer

Plenary Session 2312:25 pm - 3:30 pmRoom: Marriott Salon 1,2,3Special Regulatory SessionCHAIR: Cynthia Dunbar, MDSPEAKER

2:25 pm – 3:00 pmBarbara J. Evans, PhD, JD, LL.M. University of Houston Law Center, Houston, TXRegulatory Alternatives for Human Gene Editing

3:00 pm – 3:30 pmWilson W. Bryan, MD. Food and Drug Administration, Rockville, MD21st Century Cures Act

Exhibit Hall Coffee Social3:00 pm - 3:45 pmRoom: Exhibit Hall A & B South

Oral Abstract Session 2403:45 pm - 5:15 pmRoom: Lincoln 2,3,4Targeted Gene and Cell Therapies for CancerCO-CHAIRS: Chantal Pichon, PhD and Katy Rezvani, MD, PhDPRESENTERS

3:45 PM 273: Delivery Cellular Platform Enables Targeted Brain Delivery to GlioblastomaHeba Samaha, Baylor College of Medicine, Houston, TX



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4:00 PM 274: Intrathecal AAV9.trastuzumab for Prophylaxis and Treatment of HER2+ Breast Cancer Brain MetastasesWilliam Rothwell, University of Pennsylvania, Philadelphia, PA

4:15 PM 275: Olfactory Ensheathing Cells for Glioblastoma Gene TherapyLitia Carvalho, MGH, Charlestown, MA

4:30 PM 276: CD7-Edited T Cells Expressing a CD7-Specific CAR for the Therapy of T-Cell MalignanciesDiogo Silva, Baylor College of Medicine, Texas Children’s Hospital, Houston, TX

4:45 PM 277: Neural Stem Cell Mediated Oncolytic Virotherapy for Ovarian CancerJennifer Batalla, City of Hope, Duarte, CA

5:00 PM 278: Efficacy of a Novel Enzyme/Prodrug Combination for Clostridia Directed Enzyme Prodrug TherapyAlexandra Mowday, University of Auckland, Auckland, New Zealand

Oral Abstract Session 2413:45 pm - 5:15 pmRoom: Lincoln 5,6Immune Cell TherapiesCO-CHAIRS: Donald B. Kohn, MD and Carolyn Lutzko, PhDPRESENTERS

3:45 PM 279: HIV-Specific T Cells from HIV Naive Adult and Cord Donors Target a Range of Novel Viral Epitopes - Implications for a Cure Strategy After Allogeneic Stem Cell TransplantShabnum Patel, The George Washington University, Washington, DC

4:00 PM 280: NKT Cells Expressing a GD2-Specific Chimeric Antigen Receptor with CD28 Endodomain and IL-15 Undergo Dramatic In Vivo Expansion and Mediate Long-Term Tumor Control in a Metastatic Model of NeuroblastomaWei Huang, Baylor College of Medcine, Houston, TX

4:15 PM 281: Development of KITE-585: A Fully Human Anti-BCMA CAR T-Cell Therapy for the Treatment of Multiple MyelomaGregor Adams, Kite Pharma, Santa Monica, CA

4:30 PM 282: Multi-Pathogen-Specific T Cells Effectively Control Invasive Aspergillosis without Inducing AlloreactivityAnastasia Papadopoulou, George Papanicolaou Hospital, Thessaloniki, Greece

4:45 PM 283: Improving CAR T Cell Function by Reversing the Immunosuppressive Tumor Environment of Breast CancerPradip Bajgain, Baylor College of Medicine, Houston, TX



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5:00 PM 284: Immune Modulatory Biomaterials for Cell-Based TherapiesOmid Veiseh, Sigilon, Cambridge, MA

Oral Abstract Session 2423:45 pm - 5:15 pmRoom: Virginia ABCDNA Viruses and Molecular InterventionsCO-CHAIRS: Joseph C. Glorioso III, PhD and Frank Tufaro, PhDPRESENTERS

3:45 PM 285: Potent Antitumor Effect of Tumor Microenvironment-Targeted Oncolytic Adenovirus Against Desmoplastic Pancreatic CancerYan Li, Hanyang University, Seoul, Korea

4:00 PM 286: Incorporating Synthetic Circuits into Baculovirus Vector for Stringent Control of miRNA and Apoptosis-Inducing Gene Expression in Hepatocellular Carcinoma Cells In Vitro and In VivoMei-Wei Lin, National Tsing Hua University, Hsinchu, Taiwan

4:15 PM 287: Retargeted oHSV Vector Development for Breast Cancer TreatmentBonnie Reinhart, University of Pittsburgh, Pittsburgh, PA

4:30 PM 288: Capsid-Modified AdC7 Vector Expressing Pseudomonas aeruginosa OprF for Therapeutic Immunization Against Chronic Pulmonary P. aeruginosa InfectionAnurag Sharma, Weill Cornell Medicine, New York, NY

4:45 PM 289: A Novel Prime-Boost Ebola Virus Vaccine Study Based on Recombinant Chimpanzee Adenovirus VectorXi Yang, Institut Pasteur of Shanghai, Shanghai, China

5:00 PM 290: Highly Efficient Sendai Virus Mediated CRISPR/CAS9 Gene Editing in Hematopoietic Stem CellsBenhur Lee, Icahn School of Medicine at Mount Sinai, New York, NY



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Oral Abstract Session 2433:45 pm - 5:15 pmRoom: Marriott Salon 1Clinical Trials for Neurologic and Neurosensory DisordersCO-CHAIRS: Martin Marsala, MD and Miguel Sena-Esteves, PhDPRESENTERS

3:45 PM 291: AVXS-101 Phase 1 Gene Therapy Clinical Trial in SMA Type 1: Event Free Survival and Achievement of Developmental MilestonesJerry Mendell, Nationwide Children’s Research Hospital, Columbus, OH

4:00 PM 292: A Phase 1/2 Clinical Trial of Systemic Gene Transfer of scAAV9.U1a.hSGSH for MPS IIIA: Safety, Tolerability, and Preliminary Evidence of BiopotencyKevin Flanigan, Nationwide Children’s Hospital, Columbus, OH

4:15 PM 293: Does a One Time Retinal Gene Therapy Last Long: A Question Answered by the BrainManzar Ashtari, University of Pennsylvania, Philadelphia, PA

4:30 PM 294: Postmortem Assessment of Vector Biodistribution in the First-in-Human Intrathecal scAAV9 Gene Therapy Trial for Giant Axonal NeuropathySteven Gray, University of North Carolina at Chapel Hill, Chapel Hill, NC

4:45 PM 295: Gene Therapy for Aromatic L-Amino Acid Decarboxylase Deficiency: 5 Years After AAV2-hAADC TransductionWuh-Liang Hwu, National Taiwan University Hospital, Taipei, Taiwan

5:00 PM 296: Update on the First-in-Human Clinical Study Evaluating Neural Stem Cells in Patients with Parkinson’s DiseaseRussell Kern, International Stem Cell Corporation, Carlsbad, CA

Oral Abstract Session 2443:45 pm - 5:15 pmRoom: Maryland ABCPharmacology, Toxicology and Assay DevelopmentCO-CHAIRS: Valder R. Arruda, MD, PhD and S. Kaye Spratt, PhDPRESENTERS

3:45 PM 297: Pharmacology and Toxicology Studies Conducted for the First-in-Human Clinical Study of Neural Stem Cells in Parkinson’s DiseaseRodolfo Gonzalez, International Stem Cell Corporation, Carlsbad, CA



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4:00 PM 298: Insertion Site Analysis as a Component of Gene Therapy Product CharacterizationFrederic Bushman, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA

4:15 PM 299: Evaluating Safety and Efficacy of the AAV2tYF-PR1.7-CNGA3 Vector in CNGA3-Deficient SheepLisa Keyes, AGTC, Alachua, FL

4:30 PM 300: Nonclinical Safety Evaluation of scAAV8-hRLBP1 (CPK850) for Treatment of RLBP1-Associated Retinitis PigmentosaTimothy MacLachlan, Novartis Institutes for Biomedical Research, Cambridge, MA

4:45 PM 301: Pre-Clinical Development of a Genetically-Modified Human Dermal Fibroblast (FCX-007) for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)Anna Malyala, Fibrocell Science, Exton, PA

5:00 PM 302: Prenylation of Rab6a as a Potency Assay for Choroideremia Gene TherapyMaria Patricio, University of Oxford, Oxford, United Kingdom

Oral Abstract Session 2453:45 pm - 5:15 pmRoom: Delaware ABSynthetic/Molecular Conjugates and Physical Methods of Gene Delivery II, Oligonucleotide Therapeutics IICO-CHAIRS: Paloma H. Giangrande, PhD and Kevin G. Rice, PhDPRESENTERS

3:45 PM 303: Metabolic Stability and Expression of Novel Double Stranded mRNA Polyplexes for Liver Transfection In VivoJacob Poliskey, University of Iowa, Iowa City, IA

4:00 PM 304: Topical and Systemic Administration of Chemically Modified HCFTR mRNA Restores Lung Function in a Mouse Model of Cystic FibrosisA Haque, University of Tübingen, Tubingen, Germany

4:15 PM 305: XT-101 PLGA/Human IL-10v Plasmid Promotes Improved Quality of Life in Companion Dogs with OsteoarthritisRaymond Chavez, Xalud Therapeutics, Inc., Berkeley, CA

4:30 PM 306: Novel Poly(Sarcosine) Click Shielding Agents Improve Circulation of Redox-Sensitive siRNA Lipo-Polyplexes In VivoPhilipp Klein, LMU Munich, Munich, Germany

4:45 PM 307: Mono-Uridylated siRNA Provides Powerful Therapeutic Tool for Colon CancerMinsun Song, Beckman Research Institute of City of Hope, Duarte, CA



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5:00 PM 308: Chemical Functionalization of Double Stranded mRNAsChristopher White, University of Iowa, Iowa City, IA

Exhibit Hall Networking Reception & Poster Session ll (including Tools and Technologies Forum)5:15 pm - 7:15 pmRoom: Exhibit Hall A & B SouthTools and Technologies Forum SPEAKERS

5:30 pm – 5:45 pm Victor Ayala, PhD. ABL, Inc., Rockville, MDDesign, Support and Preclinical Assessment of Cell and Gene Therapy Based Immunotherapies

5:45 pm – 6:00 pm Andy Kokaji, PhD. STEMCELL Technologies, Vancouver, BC, CanadaT Cell Isolation, Activation and Expansion Reagents to Advance Your Cell Therapy Product Development

6:00 pm – 6:15 pm Joshua Bartoe, MS, DVM. MPI Research, Mattawan, MIOcular Gene Therapy Studies - A PRO Perspective

6:15 pm – 6:30 pm Becky Cyr. Michelson Prize & Grants, Los Angeles, CAThe Application of Gene Therapy in the Search for a Nonsurgical Spay/Neuter Methodfor Dogs & Cats

6:30 pm – 6:45 pm Kevin Murray, MBA. BioSpherix, Ltd., Parish, NYTotal Quality Approach to Gene Therapy Manufacturing

6:45 pm – 7:00 pm Jonathan Mehtala, PhD. Malvern Instruments, Westborough, MARapid AAV Titer Determination Using NanoSight: Improvements to the Labelling Protocol and Preliminary Results for Fluorescent Genome Labelling

7:00 pm – 7:15 pm Jonathan Mitchell, PhD. Imanis Life Sciences, Rochester, MNNoninvasive Imaging of AAV Biodistribution in Small and Large Animals Using the NIS Reporter Gene


PROGRAM SCHEDULE • FRIDAY, MAY 12FRIDA

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Scientific Symposium 3008:00 am - 9:45 amRoom: Lincoln 2,3,4Clinical Advancement of Gene Editing-Moving New Science to the Clinic - Organized by the Clinical Trials and Regulatory Affairs CommitteeCO-CHAIRS: Anna R. Kwilas, PhD and Kathleen Meyer, MPH, PhD, DABTSPEAKERS

8:00 am – 8:35 am Philip D. Gregory, DPhil. bluebird bio, Cambridge, MAMoving Gene Editing Platforms into the Clinic: Learning from Experience

8:35 am – 9:10 am Philippe Duchateau, PhD. Cellectis, Paris, FranceDevelopment of Gene Edited Allogeneic CAR T-Cell Therapy

9:10 am – 9:45 am John A. Zaia, MD. Beckman Research Institute of City of Hope, Duarte, CALeading a Gene Editing Trial: The Academic Clinician Perspective

Scientific Symposium 3018:00 am - 9:45 amRoom: Lincoln 5,6HSC Gene Therapy: Lessons Learned and New Outlooks - Organized by the Hematologic and Immunologic Gene and Cell Therapy CommitteeCO-CHAIRS: Pankaj Qasba, PhD and Juan A. Bueren, PhDSPEAKERS

8:00 am – 8:35 am Judith Shizuru, PhD, MD. Stanford University School of Medicine, Stanford, CAcKit Targeting for Hematopoietic Stem Cell Transplantation

8:35 am – 9:10 am Tsvee Lapidot, PhD. Weizmann Institute of Science, Rehovot, IsraelNew Pathways Controlling LT-HSC Homing, Engraftment, and Mobilization


PROGRAM SCHEDULE • FRIDAY, MAY 12FR

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9:10 am – 9:45 am Harry L. Malech, MD. NIH/NIAID Laboratory of Host Defenses, Bethesda, MDEffect of Marrow Conditioning and Integrating Vector Choice on HSC Gene Addition/Engraftment and Lineage Marking: Lessons Learned from Human Clinical Trials

Scientific Symposium 3028:00 am - 9:45 amRoom: Maryland ABCProgress Towards Gene Therapy for Muscular Dystrophies - Organized by the Musculo-Skeletal Gene & Cell Therapy CommitteeCO-CHAIRS: Michele P. Calos, PhD and Charles A. Gersbach, PhDSPEAKERS

8:00 am – 8:35 am Richard J. Samulski, PhD. University of North Carolina at Chapel Hill, Chapel Hill, NCImproving AAV Gene Delivery into Muscle

8:35 am – 9:10 am Louise Rodino-Klapac, PhD. Research Institute at Nationwide Children’s Hospital, Columbus, OHPhase I Safety Trial of AAV Dysferlin Dual Vector Delivery for LGMD2B

9:10 am – 9:45 am Kathryn R. Wagner, MD, PhD. The Kennedy Krieger Institute, The Johns Hopkins School of Medicine, Baltimore, MDMorpholino Therapies for FSHD

Scientific Symposium 3038:00 am - 9:45 amRoom: Marriott Salon 1Preclinical and Clinical Development of Oligonucleotide Therapies - Organized by the Oligonucleotide and RNAi Therapeutics CommitteeCO-CHAIRS: Veenu Aishwarya and Nuria Morral, PhDSPEAKERS

8:00 am – 8:35 am Peter M. Glazer, MD, PhD. Yale University School of Medicine, New Haven, CTNanoparticle Delivery of Triplex-forming Peptide Nucleic Acids for Correction of Thalassemia in Mice



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8:35 am – 9:10 am Ryszard Kole, PhD. University of North Carolina at Chapel Hill, Chapel Hill, NCEteplirsen-the First FDA-Approved RNA Splicing Therapeutic

9:10 am – 9:45 am Muthiah Manoharan, PhD. Alnylam Pharmaceuticals, Cambridge, MARecent Advances in GalNAC-siRNA Conjugates as Human Therapeutics

Scientific Symposium 3048:00 am - 9:45 amRoom: Virginia ABCGene Editing for Liver and Respiratory Diseases - Organized by the Respiratory & GI Tract Gene & Cell Therapy CommitteeCO-CHAIRS: Nicola Brunetti-Pierri, MD and Christian Mueller, PhDSPEAKERS

8:00 am – 8:35 am Edward E. Morrisey, PhD. University of Pennsylvania School of Medicine, Philadelphia, PANew Insights into Lung Development

8:35 am – 9:10 am Brian R. Davis, PhD. University of Texas Health Science Center at Houston, Houston, TXGene Editing of Stem Cells from Cystic Fibrosis Patients

9:10 am – 9:45 am Mark A. Kay, MD, PhD. Stanford University School of Medicine, Stanford, CAThe Use of rAAV Vectors for Genome Editing in vivo without the Use of Nucleases

Scientific Symposium 3058:00 am - 9:45 amRoom: Delaware ABDesigning the Next Generation of Viral Vectors - Organized by the Viral Gene Transfer Vectors CommitteeCO-CHAIRS: Haifeng Chen, PhD and Nicole K. Paulk, PhDSPEAKERS

8:00 am – 8:35 am Luigi M. Naldini, MD, PhD. San Raffaele Telethon Institute for Gene Therapy, Milan, ItalyViral Vectors and Gene Editing: Optimizing Delivery and Efficiency in Hematopoietic Cells



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8:35 am – 9:10 am Mavis Agbandje-McKenna, PhD. University of Florida, Gainesville, FLEngineering AAVs to Enhance Performance

9:10 am – 9:45 am Grant McFadden, PhD. Arizona State University, Tempe, AZThe Next Steps for Oncolytic Viral Therapies

Exhibit Hall Coffee Social - with Oral Poster Session9:45 am - 10:30 amRoom: Exhibit Hall A & B SouthOral Poster Session CO-CHAIRS: Jennifer L. Gori, PhD and Luk H. Vandenberghe, PhDSPEAKERS

9:45 am – 10:00 am Cedric R. Uytingco, PhD. University of Florida, Gainesville, FLGene Therapeutic Restoration of Peripheral Olfactory Impairment in BBS1 Knockout Mouse Model of Bardet-Biedl Syndrome

10:00 am – 10:15 am Dimitrios L. Wagner, Charité - University Medicine Berlin, Berlin, GermanyA Single CRISPR-Mediated Knockout as Novel Suicide Switch and Selection Tool for Gene-Modified T Cells

10:15 am – 10:30 am Danning Li, University of Massachusetts Medical School Gene Therapy Center, Worcester, MATissue and Cell Type-Specific rAAV Gene Therapy Reveals a Novel Therapeutic Cell Target and Biomarker for Canavan Disease

Plenary Session 31010:30 am - 11:30 amRoom: Marriott Salon 1,2,3Outstanding Achievement Award LectureCHAIR: Beverly L. Davidson, PhDSPEAKER

Maria-Grazia Roncarolo, MD. Stanford University School of Medicine, Stanford, CACell and Gene Therapy for Genetic Immunodeficiencies: Popping the Bubble

Break 11:30 am - 1:00 pmLunch Break (On Own - Not Provided)



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Industry Symposium 32011:30 am – 1:00 pmRoom: Thurgood Marshall NortheastSponsored by MaxCyte

Presidential Symposium 330 1:00 pm - 3:15 pmRoom: Marriott Salon 1,2,3Presidential Symposium & Presentation of the Top AbstractsCHAIR: Cynthia Dunbar, MDSPEAKER

1:00 pm – 1:55 pm Robert Langer, PhD. Massachusetts Institute of Technology, Cambridge, MANucleic Acid Delivery SystemsPRESENTATION OF TOP ABSTRACTS

1:55 PM Jorge Mansilla-Soto, Memorial Sloan Kettering Cancer Center, New York, NY

497: CRISPR/Cas9-Mediated Targeting of a CAR into the TRAC Locus Enhances CAR T Cell-Mediated Tumor Rejection

2:15 PM Jane Lebkowski, Asterias Biotherapeutics, Portola Valley, CA498: Safety and Efficacy of Human Embryonic Stem Cell Derived Oligodendrocyte Progenitor Cells (AST-OPC1) in Patients with Subacute Cervical Spinal Cord Injury

2:35 PM Hichem Tasfaout, IGBMC, Strasbourg, France 499: Antisense Oligonucleotide-Mediated Dnm2 Knockdown Prevents and Reverts Myotubular Myopathy in Mice

2:55 PM Pratiksha Thakore, Duke University, Durham, NC 500: In Vivo Gene Silencing with S. aureus CRISPR-Cas9

Repressors

Exhibit Hall Coffee Social3:15 pm - 3:45 pmRoom: Exhibit Hall A & B South



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Oral Abstract Session 3403:45 pm - 5:45 pmRoom: Maryland ABCEvolution and Library-Based Engineering of AAV Vector SystemsCO-CHAIRS: Aravind Asokan, PhD and Shen Shen, PhDPRESENTERS

3:45 PM 501: Discovery of a Neurotropic Footprint That Enables AAV Transport Across the Blood-Brain BarrierBlake Albright, University of North Carolina at Chapel Hill, Chapel Hill, NC

4:00 PM 502: Bioinformatic Identification and Large-Scale In Vitro and In Vivo Characterization of Human-Derived Novel AAV2 and AAV2/3 Capsid Variants Reveals Vectors with New Biological FunctionsLi Luo, University of Massachusetts, Medical School, Worcester, MA

4:15 PM 503: Novel AAV Variants Isolated by Directed Evolution in Primate Display Enhanced Retinal Transduction Following Intravitreal InjectionSanford Boye, University of Florida, Gainesville, FL

4:30 PM 504: Structure-Guided Iterative Evolution of Antigenically Advanced AAV Variants for Therapeutic Gene TransferLongping Tse, UNC Chapel Hill, Chapel Hill, NC

4:45 PM 505: Directed Evolution of AAV Vectors Guided by Deep Sequencing Creates Variants That Bypass Structural Barriers in Canine RetinaLeah Byrne, University of Pennsylvania/University of California Berkeley, Berkeley, CA

5:00 PM 506: In Vivo Selection of a Computationally Designed SCHEMA AAV Library Yields a Novel Variant for Infection of Adult Neural Stem Cells in the Subventricular ZoneDavid Ojala, University of California, Berkeley, Berkeley, CA

5:15 PM 507: An Alternative Route of Infection for a Novel AAVBradley Hamilton, University of Iowa, Iowa City, IA

5:30 PM 508: A Barcode-Based In Vivo Screening Method for Creating Novel AAV Serotypes for CNS-Directed Gene TherapyMarcus Davidsson, Lund University, Lund, Sweden



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Oral Abstract Session 3413:45 pm - 5:45 pmRoom: Marriott Salon 1In Vivo Gene EditingCO-CHAIRS: Charles A. Gersbach, PhD and Morgan Maeder, PhDPRESENTERS

3:45 PM 509: In Vivo Genome Editing via Non-Viral Delivery of Zinc Finger Nucleases Results in Supraphysiological Levels of Therapeutic Proteins in Adult MiceAnthony Conway, Sangamo Therapeutics, Richmond, CA

4:00 PM 510: Non-Viral Delivery of Zinc Finger Nucleases Enable Greater Than 90% Protein Knockdown of Multiple Therapeutic Gene Targets In VivoAnthony Conway, Sangamo Therapeutics, Richmond, CA

4:15 PM 511: In Vivo ZFN-Mediated Editing of the Mutant SERPINA1 Gene Results in Spontaneous Liver Repopulation by Gene-Edited Hepatocytes and Greatly Decreased Fibrosis in the PiZ Mouse Model of alpha-1 Antitrypsin Deficiency Liver DiseaseJayanta Roy-Chowdhury, Albert Einstein College of Medicine, Bronx, NY

4:30 PM 512: In Vivo Selection of Engineered Human CD34+ HSPCs Using Targeted Gene IntegrationKevin Haworth, Fred Hutchinson Cancer Research Center, Seattle, WA

4:45 PM 513: CRISPR/Cas9-Mediated In Vivo Gene Targeting Corrects OTC Deficiency in the Newborn OTC spfash MiceLili Wang, University of Pennsylvania, Philadelphia, PA

5:00 PM 514: Self-Destructing CRISPR-Constructs for Targeted Genome Editing in the RetinaGuei-Sheung Liu, University of Tasmania, Hobart, Australia

5:15 PM 515: Nanoparticle Delivery of Cas9 Ribonucleoprotein and Donor DNA In VivoKunwoo Lee, GenEdit, Berkeley, CA

5:30 PM 516: CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor VectorsRasmus Bak, Stanford University, Stanford, CA



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Oral Abstract Session 3423:45 pm - 5:45 pmRoom: Lincoln 2,3,4Immunological Aspects of Gene Therapy and VaccinesCO-CHAIRS: Ashley T. Martino, PhD and Luk Vandenberghe, PhDPRESENTERS

3:45 PM 517: Predictors of Response to CD19-Specific CAR T Cell TherapyJan Melenhorst, University of Pennsylvania, Philadelphia, PA

4:00 PM 518: Antigen-Specific Regulatory T Cells Generated by Factor VIII-CAR Retroviruses Transduction Suppress Anti-Factor VIII Immune ResponsesRichard Fu, Seattle Children’s Research Institute, Seattle, WA

4:15 PM 519: A Novel Platform for Immune Tolerance Induction in Hemophilia A MiceSimone Merlin, University of Piemonte Orientale, Novara, Italy

4:30 PM 520: Type I Interferon Signaling Is Essential for Cross-Presentation and Cross-Priming of CD8+ T Cells Against AAV CapsidJamie Shirley, University of Florida, Gainesville, FL

4:45 PM 521: Modulation of AAV Vector Dosing and Avoidance of Capsid Immune Responses via Repeated Co-Administration of Vector with Rapamycin Tolerogenic NanoparticlesAmine Meliani, University Pierre and Marie Curie - Paris 6 and INSERM U974, Paris, France

5:00 PM 522: Review of CSF and Peripheral Immune Responses Following Intrathecal Gene Transfer for Giant Axonal Neuropathy Intrathecal Gene Transfer for Giant Axonal NeuropathyDiana Bharucha-Goebel, NINDS, NIH, Bethesda, MD

5:15 PM 523: In vivo Persistence of Gene-Modified, Antibody-Secreting Hematopoietic CellsAnne-Sophie Kuhlmann, Fred Hutchinson Cancer Research Center, Seattle, WA

5:30 PM 524: Zmapp Antibody Gene Transfer Mediated by Adeno-Associated Virus Vectors Protects Against Ebola Infections in a Murine ModelMarc-Andre Robert, National Research Council Canada, Montreal, QC, Canada



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Oral Abstract Session 3433:45 pm - 5:45 pmRoom: Virginia ABCGene Therapies for Genetic and Metabolic DisordersCO-CHAIRS: Nicole K. Paulk, PhD and Denise Sabatino, PhDPRESENTERS

3:45 PM 525: Manipulation of miR-143 Regulates Transfer of a Lysosomal Enzyme Across the Blood-Brain Barrier via Mannose 6-Phosphate ReceptorYi Lin, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH

4:00 PM 526: A Blood-Brain Barrier-Crossing Peptide and Lentiviral-Mediated Stem Cell Gene Therapy Correct Cognitive, Motor and Skeletal Impairment in a Mouse Model of Mucopolysaccharidosis Type IIBrian Bigger, The University of Manchester, Manchester, United Kingdom

4:15 PM 527: Overcoming Limitations Inherent in Sulfamidase to Improve MPS IIIA Gene TherapyYonghong Chen, Children’s Hospital of Philadelphia, Philadelphia, PA

4:30 PM 528: Liver-Mediated Gene Therapy with an Engineered Secretable GAA Transgene Results in Whole-Body Correction of Pompe DiseaseGiuseppe Ronzitti, Genethon, Evry, France

4:45 PM 529: Correction of Neurological Manifestations of Mucopolysaccharidosis IIIC by a Novel Rationally Designed Neurotropic AAV Gene Therapy VectorClaire O’Leary, University of Manchester, Manchester, United Kingdom

5:00 PM 530: Systemic Messenger RNA (mRNA) Therapy Delivered via Lipid Nanoparticle as a Treatment for MUT Class Methylmalonic Acidemia (MMA)Jessica Schneller, National Institutes of Health, Bethesda, MD

5:15 PM 531: In Vivo Reprogramming of Pancreatic Duct Cells into Monohormonal Insulin Secreting β-Like CellsYuhan Wang, Oregon Health & Science University, Portland, OR

5:30 PM 532: Molecular Therapy of Melanocortin-4-Receptor Obesity by an Autoregulatory BDNF VectorJason Siu, Ohio State University, Columbus, OH



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Oral Abstract Session 3443:45 pm - 5:45 pmRoom: Delaware ABPreclinical Progress Towards Therapies for Neurologic DisordersCO-CHAIRS: Martin Marsala, MD and Miguel Sena-Esteves, PhDPRESENTERS

3:45 PM 533: Life-Long Transgene Expressions of Dopamine-Synthesizing Enzymes in a Primate Model of Parkinson DiseaseYoshihide Sehara, Jichi Medical University, Tochigi, Japan

4:00 PM 534: Second-Generation MECP2 Gene Therapy Extends Survival in a Mouse Model of Rett Syndrome (RTT) without Apparent ToxicitySarah Sinnett, University of North Carolina (UNC) Gene Therapy Center, Chapel Hill, NC

4:15 PM 535: Expanding the CNS Transduction Profile of AAV4 by Structure-Guided EvolutionMiranda Scalabrino, University of North Carolina at Chapel Hill, Chapel Hill, NC

4:30 PM 536: AAV5-miHTT Gene Therapy Demonstrates Broad Vector Distribution and Strong Mutant Huntingtin Lowering in a Huntington’s Disease Minipig ModelMelvin Evers, uniQure, Amsterdam, Netherlands

4:45 PM 537: Intrathecal scAAV9 Gene Therapy Is an Effective Treatment for PPT1-Deficiency in the Preclinical INCL Mouse ModelAlejandra Rozenberg, University of North Carolina at Chapel Hill, Chapel Hill, NC

5:00 PM 538: Silencing SOD1 to Treat Amyotrophic Lateral Sclerosis: Preclinical Studies in Cynomolgus MonkeysFlorie Borel, University of Massachusetts Medical School, Worcester, MA

5:15 PM 539: Long Term Survival of Sheep with Tay-Sachs Disease After Delivery of a Novel Bicistronic AAV Gene Therapy VectorHeather Gray-Edwards, Auburn University, Auburn, AL

5:30 PM 686: Assessment and Optimization of DNA/RNA Barcode Sequencing for Comparing Transduction Capabilities of Numerous Adeno-Associated Virus (AAV) Strains Following Intravitreal InjectionJohn M. Powers, Oregon Health & Science University, Portland, OR



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Oral Abstract Session 3453:45 pm - 5:45 pmRoom: Lincoln 5,6Preclinical Optimization of ImmunotherapiesCO-CHAIRS: Cathering M. Bollard, MD and David M. Spencer, PhDPRESENTERS

3:45 PM 541: Constitutive Signaling from an Engineered IL-7 Receptor Promotes Durable Tumor Elimination by Tumor Redirected T-CellsThomas Shum, Baylor College of Medicine, Houston, TX

4:00 PM 542: Transgenic c-MPL Provides Ligand-Dependent Co-Stimulation and Cytokine Signals to TCR-Engineered T CellsChristopher Nishimura, Baylor College of Medicine, Houston, TX

4:15 PM 543: Long Term Persistence, Location in Secondary Lymphoid Tissues and Phenotype of CD19 CAR-T Cells in MiceLingfeng Liu, Fred Hutchinson Cancer Research Center, Seattle, WA

4:30 PM 544: The Suppressive Solid Tumor Microenvironment Impairs the Activity of CAR-T Cells and Can Be Targeted by Chimeric Activating Receptor-Bearing Natural Killer CellsRobin Parihar, Baylor College of Medicine, Houston, TX

4:45 PM 545: Synergistic CD4 and CD8 T Cell Receptor Platform for Stem Cell Based Therapy for CancerRichard Koya, Roswell Park Cancer Institute, Buffalo, NY

5:00 PM 546: Dual-Switch GoCAR-T Cells: Dual Molecular Switches to Control Activation and Elimination of CAR-T Cells to Target CD123+ Cancer In VivoMyLinh Duong, Bellicum Pharmaceuticals, Houston, TX

5:15 PM 547: Enhancement of PSMA-Directed Car Adoptive T Cells by PD-1/PD-L1 BlockadeInna Serganova, Memorial Sloan Kettering Cancer Center, New York, NY

5:30 PM 548: From the CART Workshop: Selecting an EphA2-CAR for the Immunotherapy of GBMGiedre Krenciute, Baylor College of Medicine, Houston, TX



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Exhibit Hall Networking Reception & Poster Session lll (including Tools and Technologies Forum)5:45 pm - 7:45 pmRoom: Exhibit Hall A & B SouthTools and Technologies Forum SPEAKERS

6:00 pm – 6:15 pm Nikolay Korokhov, PhD. MilliporeSigma, Billerica, MAChallenges in Viral and Gene Therapy Product Testing: AAV as a Case Study

6:15 pm – 6:30 pm Alaina Schlinker, PhD. Fresenius Kabi USA LLC, Lake Zurich, ILThe LOVO Cell Processing System in Cell Therapy Process Development and Manufacturing

6:30 pm – 6:45 pm Jason Slingsby, PhD. Oxford BioMedica (UK) Limited, Oxford, United KingdomOxford BioMedica: Supporting Clients from Transgene to Commercial Supply of Lentiviral Vectors

6:45 pm – 7:00 pm Xianmin Zeng, PhD. Applied StemCell, Inc. Milpitas, CAMaking Multiple Therapeutic Cell Products from a cGMP-compliant iPSC Line

Closing Night Reception8:00 pm - 11:00 pmLibrary of Congress — Ticketed EventSponsored by University of Massachusetts Medical School

Shuttle Bus Transportation ASGCT will provide free shuttle transportation for the reception at the Library of Congress during the 20th Annual Meeting on Friday, May 12, 2017. Shuttle buses will leave from the 24th Street Entrance of the Marriott Wardman Park near the Center Tower Elevators.

Meeting TimesFriday, May 12, 2017 — 7:00 pm Departure (Running continuously until 11:00 pm)


PROGRAM SCHEDULE • SATURDAY, MAY 13SA

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ASGCT Business Meeting 7:30 am - 8:00 amRoom: Maryland ABC

Coffee Break7:30 am - 8:00 am

Scientific Symposium 4008:00 am - 9:45 amRoom: Marriott Salon 2Clinical Trials Spotlight SymposiumCO-CHAIRS: Joy Cavagnaro, PhD, DABT, ATS and Helen E. Heslop, MDSPEAKERS

8:00 am – 8:17 am Giuliana Ferrari, PhD. San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milano, Italy 722: Gene Therapy for Beta Thalassemia: Initial Results from TIGET BTHAL Clinical Trial

8:17 am – 8:34 am Lindsey George, MD. The Children’s Hospital of Philadelphia, Philadelphia, PA723: SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B at the Low Vector Dose Achieved Sustained, Continuous Factor IX Activity Levels Adequate for Endogenous Prophylaxis Preventing Bleeding Episodes and Reducing the Risk of Immune Response

8:34 am – 8:51 am Joseph Fraietta, PhD. University of Pennsylvania, Philadelphia, PA724: TET2 Gene Disruption via Lentiviral Integration Promotes CAR T Cell Expansion and Persistence

8:51 am – 9:08 am Sattva Neelapu, MD. University of Texas MD Anderson Cancer Center, Houston, TX725: ZUMA-1 Pivotal Phase 2 Trial of Axicabtagene Ciloleucel (axi-cel, KTE-C19; Anti-CD19 CAR T Cells) in Patients (pts) with Refractory Aggressive Non-Hodgkin Lymphoma (NHL)



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9:08 am – 9:25 am Frank (Xiaohu) Fan, Nanjing Legend Biotech Co. Inc., Nanjing, China 726: A Potential Terminator of Multiple Myeloma: Myeloma: LCAR-B38M CAR-T Cells Achieved Unprecedented High Rate of Continuous Complete Remission (CCR) in Refractory or Relapsed Multiple Myeloma Patients

9:25 am – 9:42 am Bumsup Lee, PhD. Kolon Life Science, Seoul, Korea, Republic of727: A Phase III Clinical Results of INVOSSA™ (TissueGene-C): A Clues for the Potential Disease Modifying OA Drug

Scientific Symposium 4018:00 am - 9:45 amRoom: Thurgood Marshall NorthNovel Vaccine Vectors to Target Challenging Infectious Diseases - Organized by the Infectious Diseases and Vaccines CommitteeCO-CHAIRS: Dong Sung An, MD, PhD and Michael A. Barry, PhDSPEAKERS

8:00 am – 8:35 am Dan H. Barouch, MD, PhD. Beth Israel Deaconess Medical Center, Boston, MAHow Close are We to a Zika Virus Vaccine?

8:35 am – 9:10 am Klaus Früh. Oregon Health and Science University, Beaverton, ORCMV-based Vaccine Vectors

9:10 am – 9:45 am Michael A. Barry, PhD. Mayo Clinic, Rochester, MNSingle Cycle Viral Vaccines

Scientific Symposium 4028:00 am - 9:45 amRoom: Delaware ABGene Therapy Comes of Age in China - Organized by the International CommitteeCO-CHAIRS: Ian E. Alexander, MBBS, PhD and Renata Stripecke, PhDSPEAKERS

8:00 am – 8:35 am Xiaojun Huang, MD, PhD. Beijing University, Beijing, China, Peking University People’s Hospital, Beijing, ChinaProgress in Haploidentical HSC Transplantation



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8:35 am – 9:10 am Huajun Jin, PhD. Shanghai Cell Therapy Research Institute, Shanghai, China, Shanghai Cell Therapy Engineering Research Center, Shanghai, ChinaPD-1 Antibody Expressing CAR-T Cells for Advanced Solid Tumors

9:10 am – 9:45 amZhao Wu, PhD. Innovative Cellular Therapeutics, Shanghai, ChinaThe Regulatory and Commercialization Environment in China for Gene and Cell Therapies: The example of CAR-T

Scientific Symposium 4038:00 am - 9:45 amRoom: Virginia ABCAdvancement of Novel Nanoagent Formulations for Clinical Application - Organized by the Nanoagents & Synthetic Formulations CommitteeCO-CHAIRS: Millicent O. Sullivan, PhD and Assem G. Ziady, PhDSPEAKERS

8:00 am – 8:35 am James Dahlman, PhD. Georgia Tech, Atlanta, GADNA Barcoded Nanoparticles for High Throughput in vivo Assays

8:35 am – 9:10 am David V. Morrissey, PhD. Intellia Therapeutics, Cambridge, MARobust in vivo Gene Editing in Mouse Hepatocytes with Systemic Lipid Nanoparticle Delivery of CRISPR/Cas9 Components

9:10 am – 9:45 am Ugur Sahin, MD. University Medical Center of Johannes Gutenberg, Mainz, GermanyIndividualizing Cancer Immunotherapy with mRNA

Scientific Symposium 4048:00 am - 9:45 amRoom: Marriott Salon 3Preventing Disease and Fixing Pathology in the Brain and Eye - Organized by the Neurologic & Ophthalmic Gene & Cell Therapy CommitteeCO-CHAIRS: Alessandra Biffi, MD and Tonia S. Rex, PhDSPEAKERS

8:00 am – 8:35 am Florian Eichler, MD. Massachusetts General Hospital, Boston, MAEndothelial Dysfunction in the Brain of ALD Patients



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8:35 am – 9:10 am Dennis O. Clegg, PhD. University of California, Santa Barbara, Santa Barbara, CAHarnessing Pluripotent Stem Cells for the Treatment of Ocular Disease

9:10 am – 9:45 am Paul A. Sieving, MD, PhD. National Eye Institute/NIH, Bethesda, MDGene Therapy for Retinal Degeneration

Scientific Symposium 4058:00 am - 9:45 amRoom: Thurgood Marshall EastUpdate on Cell Therapies for Blood and Cardiac Diseases - Organized by the Stem Cell CommitteeCO-CHAIRS: Carolyn Lutzko, PhD and Eirini P. Papapetrou, MD, PhDSPEAKERS

8:00 am – 8:35 am Andrea Ditadi, PhD. San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), Milan, ItalyA View of Hematopoietic Development from the Petri Dish

8:35 am – 9:10 am John E. Wagner, MD. University of Minnesota, Minneapolis, MNHematopoetic Stem Cell Expansion with Stem Regenin - 1

9:10 am – 9:45 am Leo Luznik, MD. Johns Hopkins University School of Medicine, Baltimore, MDThe Haplotransplant Revolution

Scientific Symposium 4068:00 am - 9:45 amRoom: Maryland ABCTechnology Roadmaps for Achievable Manufacturing - Organized by the Translational Science and Product Development CommitteeCO-CHAIRS: Gwendolyn K. Binder-Scholl, PhD and Isabelle Riviere, PhDSPEAKERS

8:00 am – 8:35 am Krishnendu Roy, PhD. Georgia Institute of Technology, Atlanta, GAAchieving Large-Scale, Cost-Effective, Reproducible Manufacturing of Cells: A Roadmap to 2025

8:35 am – 9:10 am Michele M. Myers, PhD. GlaxoSmithKline, King of Prussia, PALessons Learned from Late Phase Product Development of Cell & Gene Therapies



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9:10 am – 9:45 am Marc Better, PhD. Kite Pharma, Santa Monica, CAOvercoming Challenges for Engineered Autologous T Cell Therapies

Oral Abstract Session 41010:00 am - 12:00 pmRoom: Virginia ABCRational Design and Analysis of AAV VectorsCO-CHAIRS: Guangping Gao, PhD and Junghae Suh, PhDPRESENTERS

10:00 AM 728: Analysis of the AAVrh.10 Capsid Structure and Its Antigenic and Receptor InteractionsMario Mietzsch, University of Florida, Gainesville, FL

10:15 AM 729: Using Novel Engineered AAV Capsids with High Efficiency Retrograde Transport to Map and Modulate the Function of Specific Neuronal Subpopulations Projecting to the Lateral StriatumPatrick Aldrin-Kirk, Lund University, Lund, Sweden

10:30 AM 730: HBoV1 Capsids Facilitate rAAV Genome Transduction in Newborn Ferret AirwaysZiying Yan, The University of Iowa, Iowa City, IA

10:45 AM 731: Facile Capsid Pseudotyping via Unnatural Amino Acid (UAA) Based Site-Specific Modification of AAV2 and AAV-DJ Capsids and Engineering of AAVsDhruva Katrekar, University of California San Diego, La Jolla, CA

11:00 AM 732: Bioinformatics Design of rAAV-Compatible MiniPromoters for Cell Type-Restricted ExpressionRachelle Farkas, University of British Columbia, Vancouver, BC, Canada

11:15 AM 733: Structural Mapping of AAV9 Antigenic Sites and the Engineering of Immune Escape VariantsJ. Smith, University of Florida, Gainesville, FL

11:30 AM 734: Efficient Protection from Pre-Existing Antibodies and Superior Correction of Hemophilia B with Exosome-AAV Vector Mediated Liver Gene TransferAmine Meliani, Genethon and INSERM U951, Evry, France

11:45 AM 735: Design of an AAV9-Based Gene Therapy Vector Able to Evade Neutralization by a Novel, Potent α-AAV9 Neutralizing AntibodyApril Giles, University of Pennsylvania, Philadelphia, PA



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Oral Abstract Session 41110:00 am - 12:00 pmRoom: Delaware ABSomatic Stem Cell TherapiesCO-CHAIRS: Jennifer E. Adair, PhD and Anthony Boitano, PhDPRESENTERS

10:00 AM 736: Early and Stable Engraftment of a Population of Highly-Enriched Long-Term Multi-Potent Hematopoietic Stem CellsJennifer Adair*, Fred Hutch, Seattle, WA

10:15 AM 737: Successful Microglia Reconstitution with Allogeneic Hematopoietic Stem and Progenitor Cells Through Intracerebroventricular Injection in MiceHui Yu, Dana Farber Cancer Institute, Boston, MA

10:30 AM 738: Novel Molecular and Functional Insight into Cyclosporine-Mediated Enhancement of Human Hematopoietic Stem Cell Gene TherapyC. Petrillo, San Raffaele Telethon Institute for Gene Therapy, Milan, Italy

10:45 AM 739: Ex Vivo Blood Conditioning Using Tissue Engineered ReactorsBiju Parekkadan, Harvard Medical School, Boston, MA

11:00 AM 740: Development of an Autologous Gene-Modified Cell Therapy for the Treatment of Localized SclerodermaDarby Thomas, Intrexon Corp., Germantown, MD

11:15 AM 741: Reprogramming Human Gallbladder Cells into Insulin-Producing Beta-Like CellsFeorillo Galivo, Oregon Health & Science University, Portland, OR

11:30 AM 742: Intra-Cavity Neural Stem Cell/Polymer Scaffold Composite Therapy Targets Post-Operative Tumor Foci in a Novel Surgical Resection Model of MedulloblastomaShawn Hingtgen, The University of North Carolina at Chapel Hill, Chapel Hill, NC

11:45 AM 743: Assessment of a Cell Therapy Approach for Duchenne Muscular Dystrophy Using Mesoangioblasts and Transposable VectorsPavithra Iyer, University of Lausanne, Lausanne, Switzerland



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Oral Abstract Session 41210:00 am - 12:00 pmRoom: Marriott Salon 2Ex Vivo Gene EditingCO-CHAIRS: Dirk Grimm, PhD and Ko Mitani, PhDPRESENTERS

10:00 AM 744: Genome Editing of Hematopoietic Stem Cells to Increase Fetal Hemoglobin in Sickle Cell Disease and β-ThalassemiaJean-Yves Metais, St Jude Children’s Research Hospital, Memphis, TN

10:15 AM 745: Multiplexing CRISPR-Cas9 Genome Editing in Human Hematopoietic Stem and Effector CellsDaniel Dever, Stanford University, Palo Alto, CA

10:30 AM 746: AAVHSC Vectors Mediate Highly Precise and Efficient Homologous Recombination-Based Gene EditingLaura Smith, Beckman Research Institute of City of Hope, Duarte, CA

10:45 AM 747: Correction of SCID-X1 by Targeted Genome Editing of Hematopoietic Stem/Progenitor Cells (HSPC) in a Humanized Mouse ModelGiulia Schiroli, SR-TIGET, Milan, Italy

11:00 AM 748: Nuclease-Free AAV-Mediated Il2rg Gene Editing in X-SCID MiceTakafumi Hiramoto, University of Washington, Seattle, WA

11:15 AM 749: Correction of X-Linked Severe Combined Immunodeficiency in Human Hematopoietic Stem and Progenitor CellsMara Pavel-Dinu, Stanford University, Stanford, CA

11:30 AM 750: Ex Vivo Protein Replacement Using Homology Driven Genome Editing in Human B Cells by Combining Zinc Finger Nuclease mRNA and AAV6 Donor DeliveryBrigit Riley, Sangamo Therapeutics, Inc., Richmond, CA

11:45 AM 751: Nuclease-Targeted Gene-Editing of FOXP3 in Primary CD4+ T Cells to Generate Stable and Functional Engineered Treg ProductsYuchi Honaker, Seattle Children’s Research Institute, Seattle, WA



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Oral Abstract Session 41310:00 am - 12:00 pmRoom: Marriott Salon 3Gene and Cell Therapies for Hematologic and Immunologic Diseases IICO-CHAIRS: Philip D. Gregory, DPhil and Shengdar Q. Tsai, PhDPRESENTERS

10:00 AM 752: HSPC Mobilization in Association with In Vivo Foamy Virus Vector Delivery of Common Gamma Chain Promote Rapid Immune Reconstitution in the SCID-X1 Canine ModelOlivier Humbert, Fred Hutchinson Cancer Research Center, Seattle, WA

10:15 AM 753: LMO2 Associated Clonal T Cell Proliferation 15 Years After Gamma-Retrovirus Mediated Gene Therapy for SCIDX1Emmanuelle Six, Laboratory of Human Lymphohematopoiesis, Paris, France

10:30 AM 754: Delivery and Tracking of CCR5-Disrupted, HIVCAR T Cells in SHIV-Infected, Non-Human PrimatesCourtnee Clough, Seattle Children’s Research Institute, Seattle, WA

10:45 AM 755: First Evidence of Engraftment and Repopulation Advantage of Gene-Corrected Hematopoietic Precursors in Non-Conditioned Fanconi Anemia PatientsPaula Rio, CIEMAT/CIBERER/IIS-FJD, Madrid, Spain

11:00 AM 756: STAT3 Inhibition Unleashes TLR9-Induced B Cell Lymphoma Immunogenicity and T Cell-Dependent Antitumor Immune ResponsesMarcin Kortylewski, City of Hope, Duarte, CA

11:15 AM 757: AAV Gene Therapy Correction of Systemic Leak and Stress-Induced Edema in a Heterozygote Mouse Model of Hereditary AngioedemaOdelya Pagovich, Weill Cornell Medical College, New York, NY

11:30 AM 758: Hematopoietic Stem Cell Gene Therapy for Ifngr1 Deficiency Protects Mice from Mycobacterial InfectionsMiriam Hetzel, Hannover Medical School, Hannover, Germany

11:45 AM 759: Intravenously Administered FVIII Encoding Lenti-Viral Vectors Mediate up to 5000% of Normal and Persisting FVIII Expression in HemA Neonatal MiceTongyao Liu, Biogen, Cambridge, MA



TURDAY, M

AY 13, 2017

Oral Abstract Session 41410:00 am - 12:00 pmRoom: Thurgood Marshall NorthPreclinical Progress Towards Therapies for Neurosensory DisordersCO-CHAIRS: Beverly L. Davidson, PhD and Steven J. Gray, PhDPRESENTERS

10:00 AM 760: Application of CRISPR Technology as Treatment for Genetic Hearing LossZheng-Yi Chen, Massachusetts Eye & Ear Infirmary, Boston, MA

10:15 AM 761: Gene Therapy Restores Auditory and Vestibular Function in a Mouse Model for Usher Syndrome Type 1CCharles Askew, Boston Children’s Hospital, Harvard Medical School, Boston, MA

10:30 AM 762: Allele-Specific Gene Editing for the Treatment of Autosomal Dominant Retinitis PigmentosaGene Liau, Precision BioSciences, Durham, NC

10:45 AM 763: A Potential Therapeutic Strategy for the Treatment of Autosomal Dominant Retinitis PigmentosaCatherine O’Riordan, Sanofi, Framingham, MA

11:00 AM 764: Efficient In Vivo Gene Editing of Inherited Retinal Disease Genes in Mice and Non-Human PrimatesMorgan Maeder, Editas Medicine, Cambridge, MA

11:15 AM 765: A C-Terminal Fragment of CEP290 Is Sufficient for Rescuing Vision in Mouse Model for LCA Due to Cep290 MutationSuddhasil Mookherjee, NEI,NIH, Bethesda, MD

11:30 AM 766: Gene Supplementation Therapy for CNGA3-Linked AchromatopsiaStylianos Michalakis, Ludwig-Maximilians-Universität München, München, Germany

11:45 AM 767: Directed Evolution of Intravitreally Delivered Adeno-Associated Viral Vectors in the Non-Human Primate RetinaTimothy Day*, University of California, Berkeley, CA

Oral Abstract Session 41510:00 am - 12:00 pmRoom: Thurgood Marshall EastEngineering and Manufacturing Vectors and Cells IICO-CHAIRS: Johannes C.M. van der Loo, PhD and J. Fraser Wright, PhDPRESENTERS

10:00 AM 768: Donor-Derived Sleeping Beauty Engineered CAR CD19 Cells for B-Acute LeukemiaChiara Magnani, University of Milano-Bicocca, Monza, Italy



TURD

AY,

MA

Y 13

, 201

7

10:15 AM 769: CD46 Receptor Knockout in Packaging Cell Lines Improves Production of Measles Glycoprotein Pseudotyped Lentiviral Vectors and Transduction Efficiency of Human CD34+ CellsStosh Ozog, The Scripps Research Institute, La Jolla, CA

10:30 AM 770: Advancing rAAV QC Pipelines with AAV-GPseq: Abundant Detection of Chimeric Genomes in Preparations and Its Implications for Vector BiologyPhillip Tai*, University of Massachusetts, Medical School, Worcester, MA

10:45 AM 771: Development and Optimization of a Serotype-Independent Method of Adeno-Associated Virus Harvest and PurificationBryan Piras, St. Jude Children’s Research Hospital, Memphis, TN

11:00 AM 772: RNA Interference Mediated Ablation of Cytotoxic Transgenes Enables Productive Viral Vector PackagingJun Xie, Umass Medical School, Gene The, Worcester, MA

11:15 AM 773: Microfluidic-Enabled Manufacture of Nucleic Acid-Based Lipid Nanoparticles: Modulating Gene Expression from Bench Towards the ClinicMariam Assadian, Precision NanoSystems Inc., Vancouver, BC, Canada

11:30 AM 774: Optimizations in the Production of DNA Ministrings, Linear-Covalently Closed DNA Minivectors for Use in Non-Viral Gene TherapyShirley Wong, University of Waterloo, Waterloo, ON, Canada

11:45 AM 775: Persistent Genetic Modification of Dividing and Differentiating Cells with Non-Integrative and Autonomously Replicating DNA VectorsAlicia Roig-Merino, DKFZ, Heidelberg, Germany

Oral Abstract Session 41610:00 am - 12:00 pmRoom: Maryland ABCCancer Immunotherapies via Bacteria, Viruses and Novel ModalitiesCO-CHAIRS: David Kirn, MD and Ianko D. Iankov, MD, PhDPRESENTERS

10:00 AM 776: Development of an Effective Cancer Vaccine Platform Using Attenuated Salmonella typhiXin Xu, Baylor College of Medicine, Houston, TX

10:15 AM 777: Non-Clinical Safety, Immunogenicity and Antitumor Efficacy of Live Attenuated Salmonella Typhimurium-Based Oral T-Cell Vaccines VXM01m, VXM04m and VXM06mSébastien Wieckowski, VAXIMM AG, Basel, Switzerland



TURDAY, M

AY 13, 2017

10:30 AM 778: Targeted Interferon Gene Delivery Reprograms the Tumor Microenvironment and Induces Protective Immunity Against Multiple NeoAntigensGiulia Escobar, SR-TIGET, Milan, Italy

10:45 AM 779: Glioblastoma Eradication by Combination Oncolytic Immunovirotherapy and Immune Checkpoint BlockadeDipongkor Saha, Harvard Medical School, Boston, MA

11:00 AM 780: Understanding and Exploiting Immune Checkpoint Blockade in Animal Models of Gene and ViroimmunotherapiesRichard Vile, Mayo Clinic, Rochester, MN

11:15 AM 781: CART Cells Secreting IL18 Augment Antitumor Immunity and Increase T Cell ProliferationBiliang Hu, Upenn, Philadelphia, PA

11:30 AM 782: Improving CAR-T Cell Therapy with Oncolytic Virus-Driven Production of Bispecific T-Cell EngagerAnna Wing, Perelman School of Medicine, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA

11:45 AM 783: Gene Modification of Human Hematopoietic Stem Cells with Suicide Genes as a Safety Control SystemSatiro De Oliveira, UCLA, Los Angeles, CA


NOTES


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