Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People With Hemophilia A
Highlights From the 64th ASH Annual Meeting and Exposition

Rapid Clearance of Vector Following AAV-Mediated FVIII Gene Transfer in the Phase I/II Trial of SPK-8011 in People With Hemophilia A

Presented by: Benjamin J. Samelson Jones, MD, PhD, Children’s Hospital of Philadelphia, Philadelphia, PA

Huyen Tran, MD, PhD1, M. Elaine Eyster, MD2, Stacy E. Croteau, MD, MMS3, Margaret V. Ragni, MD, MPH4, Benjamin J. Samelson‑Jones, MD, PhD5, Spencer Sullivan, MD6, John E. J. Rasko, MBBS, PhD7,8,9, Kristen Jaworski, MSN, NP10, Amy MacDougall, BSN10, Savina Jaeger, PhD10, Magdalena Hofer, PhD10, Charlie Li, PhD10, Federico Mingozzi, PhD10, Tiffany Chang, MD, MAS,10 and Gallia Levy, MD, PhD10

1Australian Centre for Blood Diseases, Monash University, Melbourne, VIC, Australia
2Penn State Health Milton S. Hershey Medical Center, Hershey, PA
3Boston Children’s Hospital, Boston Hemophilia Center, Boston, MA
4University of Pittsburgh Medical Center, Pittsburgh, PA
5Children’s Hospital of Philadelphia, Philadelphia, PA
6Mississippi Center for Advanced Medicine, Madison, MS
7Gene and Stem Cell Therapy Program Centenary Institute, The University of Sydney, Camperdown, NSW, Australia
8Faculty of Medicine and Health, The University of Sydney, Sydney, NSW, Australia
9Cell & Molecular Therapies, Royal Prince Alfred Hospital, Sydney, NSW, Australia
10Spark Therapeutics, Inc., Philadelphia, PA

 

Data Points

Proportion of participants with detectable vector shedding over time

AAV vector clearance following infusion in the phase 1/2 trial of SPK-8011. Study participants were infused with one of four doses ranging from 5×1011 to 2×1012 vg per kg of body weight. Vector shedding from body fluids was determined by qPCR. Results show that vector is below detectable levels in saliva, semen, serum, and urine by 3 weeks and in peripheral blood mononuclear cells (PBMC) by 12 weeks following infusion.

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