Abstracts Released Ahead of 25th Annual Meeting

ASGCT Staff - May 02, 2022

We are proud to share our collection of research that will be presented at the 25th Annual Meeting!

Read the most innovative science in the field from more than 1200 pieces of research that are now available digitally on ASGCT.org and as a PDF on the Molecular Therapy website.

Below you'll find highlights from the research, including the Top Abstracts, which will be presented during the Presidential Symposium on May 18, and the Clinical Trials Spotlight abstracts, which will be presented during the Clinical Trials Spotlight Symposium on May 19.

New in 2022: Gene and Cell Therapy Trials in Progress abstracts are #50-56.

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Presentation of the Top Abstracts

Wednesday, May 18, 1:30-3:15 p.m. (ET)

808. Short- and Long-Term Hematopoietic Reconstitution After Transplantation is Stem Cell-driven and Stochastic: Implications for Gene Therapy
Stefan Radtke, Fred Hutchinson Cancer Research Center

809. Correction of the Sickle Cell Mutation by In Vivo HSC Prime Editing in a Mouse Model
Chang Li, University of Washington

810. Generation of Islet-specific Engineered Regulatory T Cells (EngTregs) for Immune Tolerance Induction in Type 1 Diabetes Using a Novel Dual-editing Strategy
Martina S. Hunt, Seattle Children's Research Institute

811. AAV-Meganuclease-mediated Gene Targeting Achieves Efficient and Sustained Transduction in Newborn and Infant Macaque Liver
Lili Wang, University of Pennsylvania

Clinical Trials Spotlight Symposium

Thursday, May 19, 8-9:45 a.m. (ET)

1188. Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)
Donald B. Kohn, UCLA

1189. Safety and Efficacy of SARS-COV-2-Specific T Cells as Adoptive Immunotherapy for High-Risk COVID-19 Patients: A Phase I/II, Randomized Clinical Trial
Evangelia Yannaki, George Papanikolaou Hospital

1190. Autologous Cell & Gene Therapy for the Therapeutic Targeting of Immune Payloads to the Solid Tumor Microenvironment: Preliminary Results of the TEM-GBM Study
Bernhard Gentner, San Raffaele Telethon Institute for Gene Therapy

1191. Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-Linked Severe Combined Immune Deficiency (XSCID) Results in the Development of a Normal and Sustained Immune System: Interim Results of an Ongoing Phase I/II Clinical Study
Ewelina Mamcarz, St. Jude Children's Research Hospital

1192. Stable Hemostatic Correction and Improved Hemophilia-Related Quality of Life: Final Analysis from the Pivotal Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec
Michael Recht, Oregon Health & Science University

1193. IGNITE DMD Study of SGT-001 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy: Long-Term Outcomes and Biomarker Update
Carl A. Morris, Solid Biosciences

1194. The OPTIC Study of Intravitreal Gene Therapy with ADVM-022 for Neovascular AMD (nAMD): The Role of Neutralizing Antibodies
Szilard Kiss, Weill Cornell Medical College

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