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, PhD^7,8,9, 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.