Now Available: 28th Annual Meeting Abstracts

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Presidential Symposium

Wednesday, May 14, 10:15 a.m. - 12 p.m. CT

Following talks by Drew Weissman, MD, PhD; and Mike McCune, MD, PhD

1. CRISPR-hfCas12Max Genome Editing Therapy Demonstrates Preclinical Efficacy and Early Clinical Benefit in Duchenne Muscular Dystrophy

Alvin Luk, PhD, Huidagene Therapeutics Inc.

2. Sustained Brain-wide Reduction of Prion via Zinc Finger Repressors in Mice and Nonhuman Primates as a Potential One-Time Treatment for Prion Disease

Bryan Zeitler, PhD, Sangamo Therapeutics, Inc.

3. Comet LV: a lentiviral vector-based mRNA co-packaging technology for enhanced ex vivo and in vivo gene therapy

Chiara Bresesti, PhD, SR-Tiget

Clinical Trials Spotlight Symposium

Friday, May 16, 1:30 - 3:15 p.m. CT

4. Treatment effect of atidarsagene autotemcel (arsa-cel) in age-matched treated vs. untreated sibling pairs with early-onset metachromatic leukodystrophy (MLD)

Valeria Calbi, MD, SR-Tiget

5. RESET-Myositis^TM: Clinical Trial Evaluating Rese-cel (Resecabtagene Autoleucel), A Fully Human, Autologous 4-1BB Anti-CD19 CAR T Cell Therapy in Idiopathic Inflammatory Myopathies: Correlative Findings

Samik Basu, MD, Cabaletta Bio

6. Extensive detoxification and favorable effects on systemic clinical outcomes after Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type I-Hurler (OTL-203)

Maria Ester Bernardo, MD, PhD, SR-Tiget

7. Phase I Clinical Trial of Safety and Efficacy of the dAd5GNE Anti-Cocaine Vaccine

Stephen M. Kaminsky, PhD, Weill Cornell Medical College

8. Phase I/II Trial of Adipose Tissue Derived Mesenchymal Stem Cell Delivery of a Measles Virus Strain Engineered to Express the Sodium Iodine Symporter (MV-NIS) in Ovarian Cancer Patients

Evanthia Galanis, MD, Mayo Clinic

9. Safety and Efficacy of DYNE-101 in Adults with DM1 in the Phase 1/2 ACHIEVE Trial

Doug Kerr, MD, PhD, Dyne Therapeutics

10. Hemophagocytic Lymphohistiocytosis (HLH)/hyperinflammatory syndrome following high dose AAV9 therapy

Aimee Donald, MBChB, PhD, University of Manchester

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