65th ASH Annual Meeting & Exposition: 801. Gene Therapies: Poster I

Symposia: Gene Therapies Oral and Poster Abstracts
Poster

Halls G-H (San Diego Convention Center)

Childhood Treatment with Adeno-Associated Viral Gene Therapy Results in Stable FVIII Expression and Improved Bleeding Phenotype in Adult Severe Hemophilia A Dogs

Paul Batty, MBBS, PhD^1,2^*, Alex Menard, MD³^*, Aomei Mo¹^*, Christine Brown¹^*, Ashrafali M Ismail⁴^*, Bridget Yates, BS⁴^*, Laura Swystun, BSc, PhD⁵^*, Lori Harpell¹^*, Abbey Pender¹^*, Andrew Winterborn¹^*, Sylvia Fong⁴^* and David Lillicrap, MD¹

¹Queen's University, Kingston, Canada
²Cancer Institute, University College, London, United Kingdom
³Kingston Health Sciences Centre, Kingston, Canada
⁴BioMarin Pharmaceutical, Novato
⁵Queen's University, Kingston, ON, Canada

2249

Gene Therapy Expressing Platelet‑Derived Factor VIII for Correction of Hemophilia Α with a History of Inhibitors

David A. Wilcox, PhD^1,2,3, Myriam Armant, PhD⁴^*, Lynn Malec, MD^1,2,3,5, Qizhen Shi, MD, PhD^1,2,3, Bryon D. Johnson, PhD⁶^*, Huiqing Xu, MD⁶^*, Lily M. Du, MD¹^*, Lisa J. Duffy, BS⁷^*, Frederic D. Bushman, PhD⁸^*, James H. Jerkins, MD⁶^*, Peiman Hematti, MD⁶, Ruta Brazauskas, PhD⁹^*, Shawn M. Jobe, MD, PhD³, Parameswaran N. Hari, MD, MBBS⁶ and Mary Eapen, MBBS, MS⁶

¹Department of Pediatrics, Medical College of Wisconsin, Milwaukee, WI
²Children's Research Institute, Children's Wisconsin, Milwaukee
³Versiti Blood Research Institute, Milwaukee, WI
⁴Translab, Boston Children's Hospital, Boston, MA
⁵Department of Medicine, Medical College of Wisconsin, Milwaukee, WI
⁶Department of Medicine, Medical College of Wisconsin, Milwaukee
⁷Gene Therapy Testing Laboratory, Indiana University, Indianapolis
⁸Department of Microbiology, University of Pennsylvania, Philadelphia, PA
⁹Institute for Health and Society, Medical College of Wisconsin, Milwaukee

2250

Bleeding Outcomes in Participants with Factor VIII Activity <5 IU/DL Post–Gene Transfer in GENEr8-1

Johnny Mahlangu, MBBCh, MMed¹, Margareth Castro Ozelo, MD², Chee Wee Tan, MD^3,4^*, Adam Giermasz, MD, PhD⁵^*, Jiaan-Der Wang, MD, PhD⁶^*, Dane Osmond, PharmD⁷^*, Hua Yu, PhD⁷^* and Tara M. Robinson, MD, PhD⁷^*

¹University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa
²Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, SP, BRA
³Department of Haematology, Royal Adelaide Hospital, Adelaide, SA, Australia
⁴University of Adelaide, Adelaide, SA, Australia
⁵University of California, Davis, Sacramento, CA
⁶Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan
⁷BioMarin Pharmaceutical Inc., Novato, CA

2251

Evaluation of the Impact of a Naturally Occurring Beta Hemoglobin Variant, Hb G-Makassar, on Mature Red Blood Cell Function and Pathology in a Sickle Cell Disease Mouse Model

Zachary Kostamo, BS¹^*, Jawa Darazim, BS¹^*, Britney Hernandez, BS¹^*, Alex Pendergast, BSc¹^*, Jordan Zgodny, BA¹^*, Erica N Evans¹^*, Yankai Zhang, PhD¹^*, Ashwin P Patel, MD, PhD, MPH¹, Celeste K. Kanne, MS¹, Elizabeth Budak²^*, Rebecca Jenkins²^*, Daniel Haupt²^*, Valerie Winton, PhD²^*, Bo Yan, PhD²^*, Adam Hartigan, PhD²^*, Haihua Chu, PhD²^*, Giuseppe Ciaramella, PhD, BSc²^* and Vivien A. Sheehan, MD, PhD¹

¹Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
²Beam Therapeutics, Cambridge, MA

2252

Efficient and Specific In Vivo Genetic Engineering of Human Hematopoietic Stem Progenitor Cells without Selective Conditioning

Vladimir V Botchkarev Jr., PhD^*, Sean Harrington^*, Alexander Justen, PhD^*, Casey Kimber^*, Anjali Kapuria^*, Yuanxin Xu^*, Kelsey Haugh, PhD^*, Ramya Ankala, MS^*, Nathan Kipniss, PhD^*, Rowena de Jesus, MS^*, Funmi Adewale^*, Kathy Daniels^*, Samantha Crocker^*, Anna Liang^*, Shannon Joyce^*, Derek Smith, PhD^*, Athena Wong, PhD^*, Valeria Berlfein^*, Shirisha Amatya, MS^*, Patricia Cruite, MS^*, Shariq Usmani, PhD, MSc, BSc^*, Albert Ruzo, PhD^*, Benjamin Ferland^*, Sundeep Chandra^*, Matteo Stoppato, PhD^*, Kyle Trudeau, PhD^*, Edward Rebar, PhD^*, Jagesh Shah, PhD^* and Luca Biasco, PhD

Sana Biotechnology, Seattle, WA

2253

Novel Bone Marrow/HSC-Targeted, Megakaryocyte-Derived Extracellular Vesicle Delivery Modality for In Vivo Gene Therapy

Laura R Goldberg, MD, PhD¹, Qiuyan W Ma, PhD²^*, Navdeep Malik, PhD²^*, Daniel Bode, PhD²^*, Samik Das, PhD³^*, Will Thompson, PhD³^*, Jenna C Harris, PhD³^*, Joseph R Inigo, PhD²^*, Pragati Jain, PhD²^*, Emily S Day, PhD³^*, David M Raiser, PhD²^*, Sedattin S Ozturk, PhD²^*, Eleftherios T Papoutsakis, PhD³, Iain R Thompson, PhD²^* and Jonathan N. Thon, PhD²^*

¹STRM.BIO, Providence, RI
²STRM.BIO, Cambridge, MA
³University of Delaware, Newark, DE

2254

CD90-Targeted Cocal-Pseudotyped Lentivirus As a Robust Platform for Human HSC Gene Therapy

Justin Thomas^*, Kurt Burkmueller^*, Greta Kanestrom^*, Stefan Radtke and Hans-Peter Kiem

Fred Hutchinson Cancer Center, Seattle, WA

2255

Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

Jenny H. McIntosh, PhD¹^*, Saira Mohamed, MSc¹^*, Lydia Ireland, BSc¹^*, Christopher L Morton²^*, Ulrike M. Reiss, MD³, Pratima Chowdary^4,5^*, Edward G. D. Tuddenham, MD^4,5^*, Andrew M. Davidoff, MD⁶ and Amit C. Nathwani, MD, PhD^7,8

¹Cancer Institute, UCL, London, United Kingdom
²Department of Surgery, St Jude Children's Research Hospital, Memphis, TN
³Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁴UCL, London, United Kingdom
⁵Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital, London, United Kingdom
⁶Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN
⁷Royal Free London - NHS Foundation Trust, London, United Kingdom
⁸Cancer Institute, UCL, London, ENG, United Kingdom

2256

HIV Comorbid Infection and Liver-Directed AAV-Based Gene Therapy in Adults with Severe and Moderately Severe Hemophilia B: Efficacy and Safety Results from Phase 2b and the Pivotal Phase 3 HOPE-B Trials 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec

Steven Pipe, MD¹, Esteban Gomez, MD², Cedric R. Hermans, MD, MRCP, PhD³, Adam Giermasz, MD, PhD⁴^*, Peter Kampmann, MD⁵^*, Richard Lemons, MD⁶^*, Nicholas Galante, PhD⁷^*, Sandra LeQuellec, PhD⁷^* and Paul Monahan⁷

¹University of Michigan, Ann Arbor, MI
²The Center For Inherited Blood Disorders, Orange, CA
³Haemostasis and Thrombosis Unit, Division of Haematology, Cliniques Universitaires Saint-Luc, Université Catholique de Louvain (UCLouvain), Brussels, BEL
⁴University of California, Davis, Sacramento, CA
⁵Department of Haematology Rigshospitalet – National University Hospital Copenhagen, Copenhagen, Denmark
⁶University of Utah, Salk Lake City, UT
⁷CSL Behring, King of Prussia, PA

2257

Characterizing a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec from the Phase 3 Benegene-2 Study Who Returned to Factor IX Prophylaxis

Laurent Frenzel, MD, PhD¹^*, Kaan Kavakli, MD²^*, Robert Klamroth, MD³^*, Shyh-Shin Chiou, MD, PhD⁴^*, Amy D. Shapiro, MD⁵, Pengling Sun, PhD⁶^*, Joanne Fuiman, MS⁷^*, John McKay, MS⁸^*, Annie F. Fang, MD, PhD⁹^*, Francesca Biondo, MD⁸^*, Frank Plonski, MA, RN, BS⁷^* and Jeremy Rupon, MD, PhD⁷

¹Department of Haematology, Institut Necker, Paris, FRA
²Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey
³Internal Medicine, Vascular Medicine and Coagulation Disorders, Vivantes Clinic Friedrichshain, Berlin, Germany
⁴Division of Pediatric Hematology and Oncology, Department of Pediatrics, Kaohsiung Medical University, Kaohsiung, Taiwan
⁵Indiana Hemophilia & Thrombosis Center, Inc., Indianapolis, IN
⁶Pfizer Inc., Cambridge, MA
⁷Pfizer Inc., Collegeville, PA
⁸Pfizer Inc., Groton, CT
⁹Pfizer Inc., New York, NY

2258

Adult Patients with Hemophilia B and with a History of Chronic HCV/HBV Infection Receiving Liver-Directed Gene Therapy Demonstrated Long-Term Bleeding Protection and Sustained FIX Activity: Efficacy and Safety Results from the HOPE-B Trial 3 Years after Administration of a Single Dose of Etranacogene Dezaparvovec

Annette von Drygalski, MD, PharmD¹, Niamh O'Connell²^*, Peter Verhamme³^*, Karina Meijer, MD, PhD⁴^*, Paul van der Valk, MD⁵^*, Rashid Saeed Kazmi, MBBS, FRCP, FRCPath⁶^*, Priyanka Raheja⁷^*, Nicholas Galante, PhD⁸^*, Sandra LeQuellec, PhD⁸^*, Rachel Church⁸^*, Samantha Lucas, BS⁸^*, Giancarlo Castaman, MD⁹^* and Paul Monahan⁸

¹Department of Medicine, Division of Hematology/Oncology, University of California San Diego, SAN DIEGO, CA
²National Coagulation Centre, St James’s Hospital, Dublin, Dublin, Ireland
³University Hospitals Leuven, Belgium, Leuven, Belgium
⁴Department of Hematology, University Medical Center Groningen, Groningen, Groningen, Netherlands
⁵Van Creveldkliniek, University Medical Center Utrecht, Utrecht, Netherlands
⁶University Hospital Southampton NHS Foundation Trust, Southampton, United Kingdom
⁷Royal London Haemophilia Centre, Bart health NHS trust, London, United Kingdom
⁸CSL Behring, King of Prussia, PA
⁹SOD Malattie Emorragiche, AOU Careggi, Firenze, Italy

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^*signifies non-member of ASH

801.P1.33 801. Gene Therapies: Poster I