Explore the 26th Annual Meeting Abstracts Collection
Live now - May 02, 2023
On Tuesday, May 2, at 4:30 p.m. ET, ASGCT released 1,600 abstracts to the public. Read them all on our Annual Meeting website.
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Starting on May 2 at 4.30 p.m. ET, read the most innovative science in the field, with 1,600 pieces of original research—34% more than last year! The collection will be available digitally on the Annual Meeting site and as a PDF on the Molecular Therapy website.
Read the abstracts collection now
Between May 16-20, attend the 26th Annual Meeting virtually or in person in Los Angeles to hear oral presentations and view hundreds of posters. Don't miss the premier event for professionals in gene and cell therapy!
If you haven’t yet, register today to take advantage of abstract presentations, scientific symposia, networking opportunities, and more. View our interactive online program now (filter by session type for more on abstracts).
See top abstracts highlighted below, including those selected to be presented at the Presidential Symposium on Wednesday, May 17, at 1:30 p.m., and the Clinical Trials Spotlight on Thursday, May 18, at 8 a.m.
Wednesday, May 18, 1:30-3:15 p.m. PT
Following talks by Jennifer Doudna, PhD, and David R. Liu, PhD
1. Efficacy and Safety of a Single Dose of Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Haydar Frangoul, Children’s Hospital Tristar Centennial
2. Split Intein-Mediated Protein trans-Splicing to Express Large Dystrophins
Hichem Tasfaout, University of Washington School of Medicine
3. Novel AAV-Capsid-Mediated Delivery of an RNAi Targeting Atxn2 Extends Survival and Improves Strength and Neuroinflammation in a Mouse Model of Sporadic ALS
Defne Amado, Neurology University of Pennsylvania
Thursday, May 18, 8-9:45 a.m. PT
4. Efficacy and Safety at Week 52 and up to Four Years in Adults with Glycogen Storage Disease Type IA (GSDIa): Results from a Phase 1/2 Clinical Trial and Long-Term Follow-Up Study of DTX401, an AAV8-Mediated, Liver-Directed Gene Therapy
Rebecca Riba-Wolman, University of Connecticut
5. Early Skeletal Outcome after Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type I Hurler
Maria Ester Bernardo, San Raffaele Telethon Institute for Gene Therapy
6. Midbrain Gene Therapy for AADC Deficiency
Krystof Bankiewicz, The Ohio State University
7. Gene Therapy for Adenosine Deaminase Deficiency: Long-Term Outcome and Post-Marketing Experience
Maddalena Migliavacca, SR-TIGET
8. GPC3-CAR T Cells Co-Expressing IL15 Mediate Potent Antitumor Activity in Liver Cancer Patients Associated with Toxicity That Can Be Mitigated Using iC9 Safety Switch
David Steffin, Baylor College of Medicine
9. Danon Disease Phase 1 RP-A501 Results: The First Single-Dose Intravenous (IV) Gene Therapy with Recombinant Adeno-Associated Virus (AAV9:LAMP2B) for a Monogenic Cardiomyopathy
Joseph Rossano, Children’s Hospital of Philadelphia
10. Clinical Dose-Response Relationship and Safety Profile of rAAVrh8-HexA/HexB
Terence Flotte, UMass Chan Medical School
Read the abstracts collection now
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