65th ASH Annual Meeting & Exposition: 801. Gene Therapies: New Approaches From Bench to Bedside

Symposia: Gene Therapies Oral and Poster Abstracts
Oral

Ballroom 20CD (San Diego Convention Center)

Moderators:

Allison P Wheeler, MD, The Vanderbilt University and Angela C Weyand, MD,

Disclosures:

No relevant conflicts of interest to declare.

9:30 AM

Assessment of Transduction of CD34+ Human Hematopoietic Stem Cells from Patients with Severe Hemophilia-Α with Lentiviral Vector Carrying a High Expression FVIII Transgene (CD68-ET3-LV)

Gurbind Singh, PhD¹^*, Mohanashankar A M, MSc²^*, Shaji R Velayudhan, PhD^3,4^*, Gabriela Denning, PhD⁵^*, Abraham Sunder Singh, RN⁴^*, Anbu Jeba, RN⁶^*, Aby Abraham, MD, DM⁶^*, Christopher B Doering, PhD⁷, H Trent Spencer, PhD⁸^* and Alok Srivastava, MD, FRACP, FRCPA, FRCP^9,10

¹Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Vellore, India
²Centre for Stem Cell Research (a unit of inStem, Bengaluru), Vellore, India
³Centre for Stem Cell Research (a unit of inStem, Bengaluru), Vellore, Tamil Nadu, India
⁴Christian Medical College, Vellore (Ranipet Campus), Vellore, India
⁵Expression Therapeutics, Inc., Tucker, GA
⁶Christian Medical College Vellore (Ranipet Campus), Vellore, India
⁷Department of Pediatrics, Aflac Cancer and Blood Disorders Center, Emory University and Children's Healthcare of Atlanta, Atlanta, GA
⁸Emory University School of Medicine and Children’s Healthcare of Atlanta, Atlanta, GA
⁹Department of Haematology, Christian Medical College, Vellore, India
¹⁰Unit of inStem Bengaluru, Centre for Stem Cell Research, Vellore, India

9:45 AM

482

AAV-Mediated Expression of a Single-Chain FVIII Mimetic Antibody Efficiently Corrects Bleeding in Haemophilia a Mice

Vincent Muczynski, PhD¹^*, Eric Bun, MSc²^*, Lewis Tanner, PhD¹^*, Jenny H. McIntosh, PhD¹^*, Olivier D. Christophe, PhD²^* and Amit C. Nathwani, MD, PhD^3,4

¹Cancer Institute, University College London, London, United Kingdom
²UMR-S U1176 - HITh, INSERM, Le Kremlin Bicetre, France
³Royal Free London - NHS Foundation Trust, London, United Kingdom
⁴Cancer Institute, UCL, London, ENG, United Kingdom

10:00 AM

483

Trem-Cel, a CRISPR/Cas9 Gene-Edited Allograft Lacking CD33, Shows Rapid Primary Engraftment with CD33-Negative Hematopoiesis in Patients with High-Risk Acute Myeloid Leukemia (AML) and Avoids Hematopoietic Toxicity during Gemtuzumab Ozogamicin (GO) Maintenance Post-Hematopoietic Cell Transplant (HCT)

John DiPersio¹, Brenda W Cooper, MD², Hyung C Suh, MD/PhD³, Divya Koura, MD⁴, Lea Bernard, MD⁵, Nirali N. Shah, M.D.⁶, Roland B. Walter, MD, PhD, MS⁷, Miguel-Angel Perales, MD⁸, Markus Mapara, MD⁹, Roni Tamari, MD⁸, Michael R. Loken, PhD¹⁰^*, Kyle Breitschwerdt¹¹^*, Sritama Nath, PhD¹¹^*, Glen D Raffel, MD¹² and Guenther Koehne, MD, PhD¹³

¹Division of Oncology, Washington University School of Medicine, Saint Louis, MO
²University Hospitals Seidman Cancer Center, Cleveland, OH
³Stem Cell Transplantation and Cellular Therapy Program, John Theurer Cancer Center, Hackensack Meridian Health, Hackensack, NJ
⁴UCSD Moores Cancer Center, La Jolla, CA
⁵Maisonneuve-Rosemont Hospital, Montreal, QC, CAN
⁶Pediatric Oncology Branch, National Institutes of Health, Bethesda, MD
⁷Translational Science and Therapeutics Division, Fred Hutchinson Cancer Research Center, Seattle, WA
⁸Adult Bone Marrow Transplantation Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY
⁹Blood and Marrow Stem Cell Transplantation Program, Division of Hematology & Oncology, Columbia University Irving Medical Center, New York, NY
¹⁰Hematologics, Inc., Seattle, WA
¹¹Vor Biopharma, Cambridge, MA
¹²Vor Biopharma, Waban, MA
¹³Baptist Health South Florida, Miami Cancer Institute, Miami, FL

10:15 AM

484

Efficient and Stable Makassar Base Editing in Nonhuman Primates

Stefan Radtke¹, Emily Fields²^*, Gregory A. Newby, PhD³^*, Jonathan S Yen, PhD⁴, Mitchell Weiss, MD, PhD⁵, Olivier Humbert, PhD²^*, Dnyanada Pande²^*, Mark Enstrom, PhD²^*, David Liu, PhD⁶^* and Hans-Peter Kiem¹

¹Fred Hutchinson Cancer Center, Seattle, WA
²Fred Hutchinson Cancer Center, Seattle
³Broad Institute of Harvard and MIT, Harvard University, Cambridge, MA
⁴Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁵St. Jude Children's Research Hospital, Memphis, TN
⁶Harvard University, Cambridge, MA

10:30 AM

485

Dual α-Globin and Truncated EPO Receptor Knockin Restores Hemoglobin Production in α-Thalassemia-Derived Hematopoietic Stem and Progenitor Cells

Simon N Chu, MD, MS^1,2^*, Eric Soupene, PhD³^*, Beeke Wienert, PhD⁴^*, Han Yin, BS⁵^*, Devesh Sharma, MBBS^1,2^*, Kun Jia, PhD^1,2^*, Shota Homma, BA, BS⁶^*, Jessica P Hampton, BS⁷, Bruce Conklin, MD⁸^*, Tippi C. MacKenzie, MD^1,2^*, Matthew Porteus, MD, PhD⁹ and Kyle Cromer, PhD, MPhil, BS^1,2,10

¹Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA
²Department of Surgery, University of California, San Francisco, San Francisco, CA
³Children’s Hospital of Oakland Research Institute, Oakland, CA
⁴Department of Medicine, University of California, South San Francisco, CA
⁵School of Medicine, University of California, San Francisco, San Francisco, CA
⁶Department of Genetics, Stanford University, Palo Alto, CA
⁷Stanford University, Stanford, CA
⁸Department of Medicine, University of California, San Francisco, San Francisco, CA
⁹Department of Pediatrics, Stanford University, Stanford, CA
¹⁰Department of Bioengineering & Therapeutic Sciences, University of California, San Francisco, San Francisco, CA

10:45 AM

486

CD117 Antibody-Drug Conjugate Conditioning Allows Efficient Engraftment of Gene-Modified CD34+ Cells with Fertility Preservation in a Rhesus Gene Therapy Model

Naoya Uchida, MD, PhD^1,2, Selami Demirci, PhD³^*, Anh Le³^*, Rebecca Chu³^*, Xiong Liu, PhD³^*, Ling Su⁴^*, Xiaolin Wu, PhD⁴^*, Allen Krouse⁵^*, N Seth Linde⁵^*, Aylin Bonifacino⁵^*, So Gun Hong, DVM, PhD⁶, Cynthia E. Dunbar, MD⁵, Leanne Lanieri⁷^*, Anjali Bhat⁷^*, Rahul Palchaudhuri, PhD⁷^*, Bindu Bennet⁷^*, Megan D. Hoban, PhD⁷^*, Kirk Bertelsen, PhD⁷^*, Lisa Olson, PhD⁷^*, Robert Donahue, DVM³^* and John F. Tisdale, MD³

¹Cellular and Molecular Therapeutics Branch, NHLBI/NIDDK, NIH, NHLBI, MCHB, Bethesda, MD
²Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan
³Cellular and Molecular Therapeutics Branch, NHLBI/NIDDK, NIH, Bethesda, MD
⁴Genomics Technology Laboratory, Leidos Biomedical Research, Inc., Frederick National Laboratory for Cancer Research, Frederick, MD
⁵Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD
⁶Translational Stem Cell Biology Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
⁷Magenta Therapeutics, Cambridge, MA

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^*signifies non-member of ASH

801.O2.6 801. Gene Therapies: New Approaches From Bench to Bedside