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Oral Abstract Session

A - Viral Vector Development -> AAV Vectors – Clinical Studies

Results of One Year Follow-Up After Treatment With Fordadistrogene Movaparvovec (PF-06939926) for Duchenne Muscular Dystrophy (DMD) in A Phase 1b, Open-label Study

Russell J. Butterfield1, Perry B. Shieh2, Florence Yong3, Michael Binks3, Tara G. McDonnell3, Kelly A. Ryan3, Marielle Delnomdedieu3, Beth A. Belluscio3, Srividya Neelakantan3, Daniel I. Levy3, Pamela F. Schwartz3, Edward C. Smith4

1University of Utah School of Medicine, Salt Lake City, UT,2University of California at Los Angeles (UCLA), Los Angeles, CA,3Pfizer Inc, New York, NY,4Duke University School of Medicine, Durham, NC

Oral Abstract Session

E - Disease Models and Clinical Applications -> Cancer – Oncolytic Viruses

Replication Competent Adenovirus-mediated cytotoxic and Interleukin-12 Gene Therapy in Stage IV Pancreatic Cancer: 36 Month Follow-Up Data from a Phase I Clinical Trial

Shyam Nyati1, Farzan Siddiqui1, Kenneth Barton1, Robert Pompa2, Svend O. Freytag1, Gazala Khan3, Irina Dobrosotskaya3, Munther Ajlouni1, Yingshu Zhang1, Jingfang Cheng1, Stephen Brown1, Benjamin Movsas1, David Kwon4

1Radiation Oncology, Henry Ford Health System, Detroit, MI,2Gastroenterology, Henry Ford Health System, Detroit, MI,3Oncology, Henry Ford Health System, Detroit, MI,4Henry Ford Pancreatic Cancer Center, Henry Ford Health System, Detroit, MI

Oral Abstract Session

I – Gene & Cell Therapy Trials in Progress -> Gene & Cell Therapy Trials in Progress

Interim Safety, Biomarker, and Efficacy Data From Imagine-1: A Phase 1/2 Open-label, Multicenter Study to Assess the Safety, Tolerability, and Efficacy of a Single Dose, ICM Administration of PBGM01 in Subjects with Type I (Early Onset) and Type IIa (Late Onset) Infantile GM1 Gangliosidosis (GM1)

David A. Weinstein1, Caroline A. Hastings2, Debra-Lynn Day-Salvatore3, Can Ficicioglu4, Chester B. Whitley5, Michal Inbar-Feigenberg6, Geneviève Bernard7,8, Roberto Giugliani9, Julien Baruteau10, Fatih S. Ezgü11, Jeanine R. Jarnes5, Yan G. Ni1, Pruthvi Nagilla1, Victoria L. Ballard1, Thomas F. Haws1, Michael H. Gelb12, Mark S. Forman1

1Passage Bio, Inc., Philadelphia, PA,2UCSF Benioff Children’s Hospital, Oakland, CA,3Saint Peter’s University Hospital, New Brunswick, NJ,4Children's Hospital of Philadelphia, Philadelphia, PA,5University of Minnesota, Minneapolis, MN,6The Hospital for Sick Children (SickKids), Toronto, ON, Canada,7McGill University, Montreal, QC, Canada,8McGill University Health Centre, Montreal, QC, Canada,9Federal University of Rio Grande do Sul, Porto Alegre, Brazil,10Great Ormond Street Hospital for Children, London, United Kingdom,11Gazi University, Gazi, Turkey,12University of Washington, Seattle, WA

Oral Abstract Session

I – Gene & Cell Therapy Trials in Progress -> Gene & Cell Therapy Trials in Progress

Myelodysplastic Syndromes after Eli-cel Gene Therapy for Cerebral Adrenoleukodystrophy (CALD)

David A. Williams1, Jacob R. Bledsoe2, Christine N. Duncan1, Florian S. Eichler3, Bartosz Grzywacz4, Ashish O. Gupta5, Troy Lund5, Paul J. Orchard5, Sarah Slauson6, Dustin Whitney6, Jakob T. Sieker6, Geoffrey Parsons6

1Dana-Farber & Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA,2Dept of Pathology, Boston Children's Hospital, Boston, MA,3Massachusetts General Hospital & Harvard Medical School, Boston, MA,4Department of Laboratory Medicine and Pathology, University of Minnesota Medical Center, Minneapolis, MN,5Division of Blood and Marrow Transplantation, Dept. of Pediatrics, University of Minnesota, Minneapolis, MN,6bluebird bio Inc., Cambridge, MA

Oral Abstract Session

I – Gene & Cell Therapy Trials in Progress -> Gene & Cell Therapy Trials in Progress

AXO-AAV-GM2 Gene Therapy for Infantile- and Juvenile-onset GM2 Gangliosidosis: Preliminary Results from an Ongoing Phase 1/2 Trial

Florian Eichler1, Terence Flotte2, Haley Andonian1, Oguz Cataltepe2, Rebecca Artinian2, Albert Misko1, Benjamin Thorp3, John Jameson3, Michael Sheehan3, Toby Vaughn3, Donna Valencia3, Erika De Boever3

1Massachusetts General Hospital, Center for Rare Neurologic Diseases, Boston, MA,2University of Massachusetts Chan Medical School, Worcester, MA,3Sio Gene Therapies, Inc, New York, NY

Oral Abstract Session

A - Viral Vector Development -> AAV Vectors – Virology and Vectorology

Evolving AAV Capsids with Broad Biodistribution in Deep Brain Structures in Adult Rhesus Macaques

David E. Leib1, Megan S. Keiser1, Yong Hong Chen1, Paul T. Ranum1, Luis Tecedor1, Congsheng Cheng1, Laurence A. Busque1, Beverly L. Davidson1,2

1The Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children's Hospital of Philadelphia, Philadelphia, PA,2Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA

Oral Abstract Session

I – Gene & Cell Therapy Trials in Progress -> Gene & Cell Therapy Trials in Progress

Preliminary Safety, Tolerability and Efficacy of Direct Epicardial Administration of Encoberminogene Rezmadenovec to Ischemic Myocardium in Patients with Refractory Angina: Six Month Phase 1 Data

Thomas J. Povsic1, Jay H. Traverse2, Timothy D. Henry3, Todd K. Rosengart4, Ronald G. Crystal5, Howard C. Dittrich6, Elizabeth A. Tarka6, Nahush A. Mokadam7

1Duke University Medical Center, Durham, NC,2Minneapolis Heart Institute, Minneapolis, MN,3The Christ Hospital, Cincinnati, OH,4Baylor College of Medicine, Houston, TX,5Weill Cornell Medicine, New York, NY,6XyloCor Therapeutics, Wayne, PA,7The Ohio State University Wexner Medical Center, Columbus, OH

Oral Abstract Session

G - Cell Therapies -> Cell Therapies (Includes development of somatic, embryonic and induced pluripotent stem cells or other therapeutic cell populations, and issues related to cell expansion or processing)

Long-term Follow-up of Subjects With Diabetes 2 Type Treatment with ex vivo Gene Therapy

Svitlana Gramatiuk1, Julia Ivanova2

1Ukraine Association of Biobank, Kharkiv, Ukraine,2Kharkiv National Medical University, Kharkiv, Ukraine

Oral Abstract Session

E - Disease Models and Clinical Applications -> Cancer – Oncolytic Viruses

Virotherapy for Diffuse Intrinsic Pontine Glioma: Results from a Phase I Clinical Trial with DNX-2401

Marc Garcia-Moure1, Jaime Gallego Perez-Larraya1, Ana Patiño-Garcia1, Sara Labiano1, Jasper van der Lugt2, Jessica Dobbs3, Joan Robbins4, Ricardo Diez-Valle5, Fred F. Lang6, Candelaria Gomez-Manzano7, Juan Fueyo7, Sonia Tejada-Solis5, Marta M. Alonso1

1Pediatrics, University Hospital of Navarra, Pamplona, Spain,2Pediatrics, Princess Maxima, Utrech, Netherlands,3DNATrix, SanDiego, CA,4DNatrix, SanDiego, CA,5Neurosurgery, Fundación Jimenez Díaz, Madrid, Spain,6Neurosurgery, MD Anderson Cancer Center, Houston, TX,7NeuroOncology, MD Anderson Cancer Center, Houston, TX

Oral Abstract Session

E - Disease Models and Clinical Applications -> Neurologic Diseases

Successful Prenatal Delivery of a Therapeutic Antisense Oligonucleotide for Treatment of Angelman Syndrome in Mice

Maria T. Clarke1, Lea Lentz1, Laura Remesal Gomez1, Georgia Kirn1, Jasmine Wu1, Antonia Varthaliti1, Danielle Tan2, Stephan J. Sanders1, Scott Dindot3, Saul Villeda1, Tippi C. MacKenzie1

1UCSF, San Francisco, CA,2BioMarin, Marin, CA,3Texas A&M, College Station, TX

Oral Abstract Session

E - Disease Models and Clinical Applications -> Cancer – Immunotherapy, Cancer Vaccines

Single-cell Antigen-specific Activation Profile of CAR-T Infusion Product Identifies Th2 Deficiency in CD19-Positive Relapsed ALL Patients

Zhiliang Bai

Yale University, New Haven, CT

Oral Abstract Session

B - Gene Targeting and Gene Correction -> Gene Targeting and Gene Correction (Basic development of novel technologies for genome editing, with or without site-specific endonuclease. Abstracts focused on specific disease applications should be submitted to the suitable tissue/disease category)

MOSAIC Enables In Situ Saturation Mutagenesis of Genes and Optimization of CRISPR Prime Editor Activities in Human Cells

Jonathan Y. Hsu1,2,3,4, Kin Chung Lam2,3,4, Justine Y. Shih2,3,4, Luca Pinello2,3,4, Keith Joung2,3,4

1Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA,2Molecular Pathology Unit, Massachusetts General Hospital, Charlestown, MA,3Center for Cancer Research and Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, MA,4Department of Pathology, Harvard Medical School, Boston, MA

Oral Abstract Session

H - Clinical Translation of Gene and Cell Therapies -> Vector Product Engineering, Development or Manufacturing

Genome Editing of Human Hematopoietic Stem Cells to Induce Fetal Hemoglobin for Autologous Cellular Therapy of Sickle Cell Disease

Varun Katta1, Kiera O’Keefe1, Racheal Wood1, Cicera R. Lazzarotto1, Thiyagaraj Mayuranathan1, Jonathan Yen1, GaHyun Lee1, Yichao Li1, Naoya Uchida2, Shondra M. Pruett-Miller3, John Tisdale2, Akshay Sharma4, Mitchell J. Weiss1, Shengdar Q. Tsai1

1Hematology, St Jude Children's Research Hospital, Memphis, TN,2Molecular and Clinical Hematology Branch, National Heart, Lung and Blood Institute, Bethesda, MD,3Cell and Molecular Biology, St Jude Children's Research Hospital, Memphis, TN,4Bone Marrow Transplantation and Cellular Therapy, St Jude Children's Research Hospital, Memphis, TN

Oral Abstract Session

F - Immunological Aspects of Gene Therapy and Vaccines -> Immunological Aspects of Gene Therapy and Vaccines (Includes host responses, therapy/prevention of infectious diseases; excludes cancer immunotherapy and cancer vaccines)

A Collaborative Analysis by Clinical Trial Sponsors and Academic Experts of Anti-transgene SAEs in Studies of Gene Therapy for DMD

Carsten G. Bonnemann1, Beth A. Belluscio2, Serge Braun3, Carl Morris4, Teji Singh5, Francesco Muntoni6

1Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD,2Pfizer, New York, NY,3Genethon, Evry-Courcouronnes, France,4Solid Biosciences, Cambridge, MA,5Sarepta Therapeutics, Cambridge, MA,6Great Ormond Street Institute of Child Health, University College London, London, United Kingdom

Oral Abstract Session

A - Viral Vector Development -> AAV Vectors – Preclinical and Proof-of-Concept Studies

1: In vivo Selection of Randomly Integrated rAAV Vectors

Amita Tiyaboonchai, Anne Vonada, Markus Grompe

Oregon Health and Science University, Portland, OR

Oral Abstract Session

A - Viral Vector Development -> AAV Vectors – Preclinical and Proof-of-Concept Studies

2: Bicistronic AAV Gene Therapy for Tay-Sachs and Sandhoff Diseases in the Sheep Model of Tay-Sachs

Toloo Taghian1, William C. Baker1, Stephanie Bertrand2, Abigail W. McElory1, Hector R. Benatti1, Jillian Gallagher1, Erin Hall1, Deborah Fernau1, Kalajan L. Mercado3, Lauren Ellis3, Elise Diffie4, Amanda Gross3, Anne S. Maguire3, Monique Otero1, Rachel Prestigiacomo2, Rachael Gately2, Hannah Lahey1, Ana Rita Batista1, Amanda Taylor3, Jey Koehler3, Douglas R. Martin3, Miguel Sena-Esteves1, Heather L. Gray-Edwards1

1UMass Chan Medical School, Worcester, MA,2Tufts Cummings School of Veterinary Medicine, Grafton, MA,3Auburn University, Auburn, AL,4Auburn Univeristy, Auburn, AL

Oral Abstract Session

A - Viral Vector Development -> AAV Vectors – Preclinical and Proof-of-Concept Studies

3: Vagus Nerve Delivery of AAV9 to Treat Autonomic Nervous System Dysfunction in Giant Axonal Neuropathy

Rachel M. Bailey1, Diane Armao2, Irvin Garza1, Yuhui Hu1, Sydni Holmes1, Alejandra Rozenberg2, Angela Price1, Steven J. Gray1

1UT Southwestern Medical Center, Dallas, TX,2University of North Carolina at Chapel Hill, Chapel Hill, NC

Oral Abstract Session

A - Viral Vector Development -> AAV Vectors – Preclinical and Proof-of-Concept Studies

4: Assessment of Gene Therapy Treatment in the Pompe Disease Canine Model

Megan K. Pope1, Kirsten Coleman1, Megan Wichman1, Denise Cloutier1, Christina Cousins2, Lee Ann Mueller1, Victoria Whitehead3, Naresh Meena4, Nina Raben4, Manuela Corti1, Barry Byrne1

1Pediatrics, University of Florida, Powell Gene Therapy Center, Gainesville, FL,2Pediatrics, University of Florida, Gainesville, FL,3ProtoKinetics, Haverton, PA,4National Institutes of Health, Bethesda, MD

Oral Abstract Session

A - Viral Vector Development -> AAV Vectors – Preclinical and Proof-of-Concept Studies

6: The Porcine Model for In Utero Gene Therapy

Apeksha Dave, Cara L. Berkowitz, Valerie L. Luks, Pallavi V. Menon, Brandon M. White, Rohan Palanki, Haiying Li, Philip W. Zoltick, William H. Peranteau

CHOP, Philadelphia, PA

Oral Abstract Session

A - Viral Vector Development -> AAV Vectors – Preclinical and Proof-of-Concept Studies

7: Temporary Mechanical Support Improves Cardiac AAV Gene Transfer Efficacy in a Pig HF Model

Renata Mazurek, Tomoki Sakata, Spyros Mavropoulos, Anjali J. Ravichandran, Francisco Romeo, Kiyotake Ishikawa

Mount Sinai, New York, NY

Abstract Information


Abstract authors will select a topic that best describes their abstract from this list.

Submission Information

Here's what you need to know about submitting and presenting your abstract.

Awards & Incentives

You might be eligible for one of these awards when you submit an abstract.

Presentation Information

Abstracts at the 25th Annual Meeting will be split between oral presentations and posters. All poster presenters will also have the ability to create a digital presentation for the virtual component of the meeting.

Trials in Progress and Late-Breaking Abstracts

In 2022, ASGCT is offering a new topic category as well as the opportunity to submit late-breaking abstracts beginning Feb. 14.

Volunteer to Review Abstracts

Recommend yourself or a colleague to review abstracts for the Annual Meeting. This is a great way for early- to mid-career stage members to get involved with ASGCT!

Embargo Policy

Here's the embargo information for research presented at the 25th Annual Meeting.

Review Posters for Awards

ASGCT offers the opportunity to self-nominate or submit recommendations to review poster presentations at the Annual Meeting. Attendance at the Annual Meeting is required to participate.

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