Now Available: 27th Annual Meeting Abstracts

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Presidential Symposium

Wednesday, May 8, 10:15 a.m.-12 p.m. ET

Following talks by Kevin Campbell, PhD, and Charles Murry, MD, PhD

1. Atidarsagene Autotemcel (Hematopoietic Stem Cell GeneTherapy) Preserves Cognitive and Motor Development in Metachromatic Leukodystrophy with up to 12 Years Follow-Up

Alessandro Aiuti, MD, PhD San Raffaele Telethon Institute for Gene Therapy (SR-Tiget)

2. In Vivo Generation of Both CAR T Cells and CAR NK Cells Using a CD7 Targeted Lentiviral Vector

Karla Mullen, PhD, Interius Biotherapeutics

3. A Recombinant Human Bocavirus Vector Delivers Therapeutic Levels of CTFR to Cystic Fibrosis Primary Human Airway Cells and Transduces Airway Epithelium In Vivo

Jeff Moffit, PhD, Carbon Biosciences

Clinical Trials Spotlight Symposium

Wednesday, May 8, 8-9:45 a.m. ET

4. Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RPL301

Julián Sevilla, Hematol y Hemoterapia, Fundación Invest Bioméd, Hosp Infantil Univ Niño Jesús (HIUNJ)

5. Personalized Neoantigen DNA Vaccine (GNOS-PV02) and Pembrolizumab Develop Antitumor Poly-Functional Neoantigen-Specific CD4+ and CD8+ Effector T Cells in Advanced Hepatocellular Carcinoma Therapy

Renzo Perales, PhD, Geneos Therapeutics

6. Allogeneic NKT Cells Expressing a CD19-Specific CAR in Patients with Relapsed or Refractory B cell Malignancies: An Interim Analysis

Leonid Metelitsa, MD, PhD, Baylor College of Medicine

7. Reprogramming of the Tumor Microenvironment in Glioblastoma Multiforme by Transplantation of Genetically-Engineered Hematopoietic Stem Cells

Bernhard Gentner, MD, PhD, CHUV

8. Long Term Safety and Integration Site Analysis Over a Large Cohort of Patients Treated with T Cells Modified by Lentiviral or Gammaretroviral Gene Addition

Frederic Bushman, PhD, UPenn

9. Safety and Efficacy of AB-1002 Gene Therapy in Patients with Advanced Heart Failure: Results from an Ongoing Phase 1 Clinical Trial

Luke Roberts, PhD, Asklepios BioPharmaceutical, Inc

10. Intracochlear Administration of DBOTO Gene Therapy in Pediatric Patients with Profound Hearing Loss Due to Otoferlin Mutations: The CHORD Phase 1/2 Open-Label Trial

Lawrence Lustig, MD, Columbia University Medical Center

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