57th ASH Annual Meeting and Exposition (December 5-8, 2015): 801. Gene Therapy and Transfer: Gene Therapy for Hemoglobinopathies and Inherited Bleeding Disorders

Gene Therapy and Transfer Oral and Poster Abstracts
Oral

Tangerine 3 (WF3-4), Level 2 (Orange County Convention Center)

Moderators:

Naoya Uchida, National Institutes of Health and Stefan Karlsson, MD, PhD, Lund University

Disclosures:

No relevant conflicts of interest to declare.

7:30 AM

Adeno-Associated Viral Vector Delivery of Optimized Human Factor VIII Achieves Therapeutic Factor VIII Levels in Non-Human Primates

Xavier M. Anguela, PhD^1,2^*, Liron Elkouby, PhD^1,2^*, Raffaella Toso, PhD¹^*, Marti DiPietro, MS¹^*, Robert J. Davidson²^*, Katherine A. High, MD¹^* and Denise E. Sabatino, PhD^2,3

¹Spark Therapeutics, Inc., Philadelphia, PA
²The Children's Hospital of Philadelphia, Philadelphia, PA
³Department of Pediatrics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

7:45 AM

200

ZFN-Mediated Gene Targeting at the Albumin Locus in Liver Results in Therapeutic Levels of Human FIX in Mice and Non-Human Primates

Thomas Wechsler, PhD¹^*, Kathleen E. Meyer²^*, S. Kaye Spratt²^*, Judith Greengard, PhD¹^*, Yolanda Santiago³^*, Scott Sproul³^*, Richard Surosky⁴^*, David E. Paschon²^*, Nathalie Dubois-Stringfellow²^*, Dale Genji Ando, MD²^*, Geoff Nichol⁵^*, Edward J. Rebar, PhD¹^* and Michael C. Holmes, PhD¹^*

¹Sangamo BioSciences, Richmond, CA
²Sangamo Biosciences Inc, Richmond, CA
³Sangamo Biosciences Inc., Richmond, CA
⁴Sangamo Biosciences Inc, Richmond, CA, Afghanistan
⁵Sangamo Biosciences Inc, RIchmond, CA

8:00 AM

201

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

Mark C. Walters, MD¹, John Rasko, MBBS, PhD², Suradej Hongeng, MD³, Janet Kwiatkowski, MD⁴, Gary J Schiller, MD⁵, Morris Kletzel, MD⁶, P. Joy Ho, MBBS, DPhil⁷, Elliott Vichinsky, MD⁸, Christof von Kalle, MD⁹^*, Marina Cavazzana, MD, PhD¹⁰, Philippe Leboulch, MD^3,11,12^*, Alexandria Petrusich¹³^*, Sandeep Soni, MD¹³ and Alexis A. Thompson, MD¹⁴

¹UCSF Benioff Children's Hospital, Oakland, CA
²Cell & Molecular Therapies and Institute of Haematology, Royal Prince Alfred Hospital, Camperdown, Australia
³Ramathibodi Hospital, Mahidol University, Bangkok, Thailand
⁴Children's Hospital of Philadelphia, Philadelphia, PA
⁵University of California, Los Angeles, CA
⁶Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University, Chicago, IL
⁷Institute of Haematology, Royal Prince Alfred Hospital, Sydney, Australia
⁸Hematology/Oncology, UCSF Benioff Children's Hospital Oakland, Oakland, CA
⁹National Center for Tumor Diseases (NCT), German Cancer Research Center (DKFZ), Heidelberg, Germany
¹⁰Biotherapy Department, Hôpital Universitaire Necker -Enfants Malades, Paris, France
¹¹Brigham & Women’s Hospital and Harvard Medical School, Boston, MA
¹²CEA (iMETI) and University of Paris-Sud, Fontenay-aux-Roses, France
¹³bluebird bio, Inc., Cambridge, MA
¹⁴Ann and Robert H. Lurie Children’s Hospital of Chicago, Northwestern University, Chicago, IL

8:15 AM

202

Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector

Marina Cavazzana, MD, PhD¹, Jean-Antoine Ribeil, MD, PhD^1,2^*, Emmanuel Payen, MD³^*, Felipe Suarez, MD, PhD^2,4, Yves Beuzard, MD³^*, Fabien Touzot, MD, PhD⁵^*, Resy Cavallesco, PhD⁶, Francois Lefrere, MD^1,2^*, Stany Chretien, MD³^*, Philippe Bourget, PharmD, PhD⁷^*, Fabrice Monpoux, MD⁸^*, Corinne Pondarre⁹^*, Benedicte Neven¹^*, Manfred Schmidt, PhD¹⁰^*, Christof von Kalle, MD¹¹^*, Frans A. Kuypers, Ph.D.¹², Laura Sandler, MPH¹³^*, Sandeep Soni, MD¹³, Olivier Hermine¹⁴^*, Stephane Blanche, MD¹^*, Mariane De Montalembert, MD^1,2, Salima Hacien-Bey-Abina, PharmD, PhD^1,15^* and Philippe Leboulch, MD^3,6,16^*

¹Hôpital Universitaire Necker -Enfants Malades, Paris, France
²Paris Descartes Sorbonne Paris Cite University, Paris, France
³CEA (iMETI) and University of Paris-Sud, Fontenay-aux-Roses, France
⁴Department of Hematology, Necker - Enfants Malades Hospital, Paris, France
⁵Biotherapy Department, Hôpital Universitaire Necker -Enfants Malades, Paris, France
⁶Brigham & Women’s Hospital and Harvard Medical School, Boston, MA
⁷Clinical Pharmacy Department, University Hospital Necker-Enfants Malades, Paris, France
⁸Centre Hospitalier de Nice Sophia-Antipolis, Nice, France
⁹SCD Referral Centre Hospitalier lntercommunal de Creteil, Creteil, France
¹⁰Department of Translational Oncology, National Center for Tumor Diseases and German Cancer Research Center, Heidelberg, Germany
¹¹National Center for Tumor Diseases (NCT), German Cancer Research Center (DKFZ), Heidelberg, Germany
¹²Children's Hospital Oakland Research Institute, Oakland, CA
¹³bluebird bio, Inc., Cambridge, MA
¹⁴Department of Hematology, Necker Children's hospital, APHP, Paris, France
¹⁵Groupe Hospitalier Universitaire Paris-Sud, Paris, France
¹⁶Mahidol University, Bangkok, Thailand

8:30 AM

203

Genome Editing of the Bcl11A Erythroid Specific Enhancer in Bone Marrow Derived Hematopoietic Stem and Progenitor Cells for the Treatment of Sickle Cell Disease

Siyuan Tan, PhD¹^*, Kai-Hsin Chang, PhD¹^*, Sarah Smith, BS¹^*, Kai Chen, MS¹^*, Timonthy Sullivan, BS¹^*, Qianhe Zhou, PhD¹^*, Andreas Reik, PhD²^*, Fyodor D Urnov, PhD²^*, Edward J. Rebar, PhD²^*, Olivier Danos, PhD¹^* and Haiyan Jiang, PhD¹

¹Biogen, Cambridge, MA
²Sangamo BioSciences, Richmond, CA

8:45 AM

204

Clinical-Scale Genome Editing of the Human BCL11A Erythroid Enhancer for Treatment of the Hemoglobinopathies

Fyodor D Urnov, PhD¹^*, Andreas Reik, PhD¹^*, Jeff Vierstra, PhD²^*, Kai-Hsin Chang, PhD³^*, Yuanyue Zhou¹^*, Andrea Mich¹^*, Jennifer Adrian¹^*, Lisa Fox¹^*, Nikoletta Psatha, PhD²^*, Siyuan Tan, PhD³^*, Evangelia Yannaki, MD⁴, Stewart Craig, PhD¹^*, Edward J. Rebar, PhD¹^*, John Stamatoyannopoulos, MD²^*, Thalia Papayannopoulou, MD², George Stamatoyannopoulos, MD, DSci² and Haiyan Jiang, PhD³

¹Sangamo BioSciences, Richmond, CA
²University of Washington, Seattle, WA
³Biogen, Cambridge, MA
⁴George Papanicolaou Hospital, Thessaloniki, Greece

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801.O1.6 801. Gene Therapy and Transfer: Gene Therapy for Hemoglobinopathies and Inherited Bleeding Disorders