66th ASH Annual Meeting: 801. Gene Therapies: Gene Therapies for Hemophilia, Cancer and Immunodeficiencies

Symposia: Gene Therapies Oral and Poster Abstracts
Oral

Research, Clinical trials, Fundamental Science, Bleeding and Clotting, Bleeding disorders, Translational Research, Hemophilia, Bone Marrow Failure Syndromes, Clinical Research, Platelet disorders, Genetic Disorders, Diseases, Immune Disorders, Immunodeficiency, Gene Therapy, Biological therapies, Treatment Considerations, Emerging technologies, Technology and Procedures, Gene editing, Study Population, Animal model

Room 33 (San Diego Convention Center)

Moderators:

Wolfgang A. Miesbach, MD, PhD, University Hospital Frankfurt and Sara Mastaglio, MD,

Disclosures:

Miesbach: Bayer, Biomarin, Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Regeneron, Roche, Sanofi, sobi, Takeda/Shire, uniQure: Consultancy; Bayer, Biotest, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, Takeda/Shire: Research Funding; Bayer, Biomarin, Biotest, CSL Behring, Chugai. LFB, Novo Nordisk, Octapharma, Pfizer, Roche,sobi, Takeda/Shire: Honoraria; Bayer, Biomarin, Biotest, CSL Behring, Chugai. LFB, Novo Nordisk, Octapharma, Pfizer, Roche,sobi, Takeda/Shire: Speakers Bureau.

4:30 PM

1051

Ex Vivo Lentiviral Hematopoietic Stem Cell (HSC) Gene Therapy May Represent a Curative Therapy for the Life-Threatening Inborn Error of Immunity Severe Combined Immunodeficiency Due to Adenosine Deaminase (ADA) Deficiency (ADA-SCID)

Katelyn Masiuk, MD, PhD^1,2^*, Jinhua Xu-Bayford, RN³^*, Augustine Fernandes, Ph.D.⁴^*, Konstantinos Vazouras, M.D.^5,6^*, Danilo Pellin, PhD^7,8^*, Havinder Hara, PhD⁹^*, Sohini Roy, Ph.D,²^*, Beatriz Campo Fernandez, PhD²^*, Thao Thai Dang, PhD¹⁰^*, Kimberly Gilmour, Ph.D.¹¹^*, Elena Alvarez Mediavilla, PhD⁶^*, Beatrice Curio-Penny¹²^*, Gráinne O'Toole, RN¹³^*, Rima Ahmed, R.N.¹³^*, Jordyn Arnold, R.N.¹⁴^*, Elizabeth Garabedian, RN¹⁵^*, Harry L Malech, MD¹⁶, Kenneth Cornetta, MD¹⁷^*, Claire Booth, MBBS, PhD^18,19^* and Donald B. Kohn, MD²⁰

¹David Geffen School of Medicine at UCLA, UCLA, Los Angeles, CA
²David Geffen School of Medicine at UCLA, University of California, Los Angeles, Los Angeles, CA
³Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital, London, London, United Kingdom
⁴Department of Microbiology, Immunology & Molecular Genetcis, University of California,-Los Angeles, Los Angeles, CA
⁵Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital UCL Great Ormond Street Institute of Child Health, London, United Kingdom
⁶UCL Great Ormond Street Institute of Child Health, London, United Kingdom
⁷Harvard Data Science Initiative, Harvard Medical School, Boston, MA
⁸Pediatric Oncology, Dana Farber Cancer Institute, Boston, MA
⁹UCL Great Ormond Street Institute of Child Health, London, GBR
¹⁰Clinical Medical and Molecular Genetics, Indiana University School of Medicine, Indianapolis, IN
¹¹Department of Immunology, Great Ormond Street Hospital, London, GBR
¹²David geffen School of Medicine, at UCLA, University of California, Los Angeles, Los Abgeles, CA
¹³Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital, London, United Kingdom
¹⁴David Geffen School of Medicine at UCLA, Department of Pediatrics, Los Angeles, CA
¹⁵National Human Genome Research Institute, National Institutes of Health, BETHESDA, MD
¹⁶National Institute of Allergy and Infectious Diseases, LHD, NIAID, NIH, Bethesda, MD
¹⁷National Gene vector Biorepository, Indiana University School of Medicine, Indianapolis, IN
¹⁸Institute of Child Health, London, ENG, United Kingdom
¹⁹UCL Great Ormond St Institute of Child Health, London, United Kingdom
²⁰David Geffen School of Medicine at UCLA, University of California - Los Angeles, Los Angeles, CA

4:45 PM

1052

Factor VIII Expression from a Novel F8 Transgene through a Lentiviral Vector Transduced CD34+ Autologous Hematopoietic Stem Cells for Gene Therapy of Severe Hemophilia Α: Final Results from a Phase 1 Clinical Trial

Alok Srivastava, MD, FRACP, FRCPA, FRCP^1,2, Aby Abraham, MD, DM¹^*, Fouzia N Aboobacker, DNB, DM³^*, Gurbind Singh, PhD⁴^*, Tulasi Geevar, MD⁵^*, Uday Kulkarni, MD, DM¹, Sushil Selvarajan, MD, DM⁶^*, Anu Korula, MD, DM, MRCP⁷^*, Rutvi Gautam Dave, MD⁸^*, Mohanashankar A M, MSc⁹^*, Abraham Sunder Singh, RN¹⁰^*, Anbu Jeba, RN⁶^*, Navien Kumaar, MSc⁶^*, Christopher Benjamin, B Pharm, Dip Clin Pharm⁶^*, Kavitha M Lakshmi, MSc⁶^*, Shaji R Velayudhan, PhD^6,11^*, Sukesh C Nair, MD¹²^*, Gabriela Denning, PhD¹³^*, Pete Lollar, MD¹⁴, Christopher B Doering, PhD¹⁵ and H. Trent Spencer, PhD¹⁵^*

¹Department of Haematology, Christian Medical College Ranipet Campus, Ranipet, India
²Unit of inStem Bengaluru, Centre for Stem Cell Research, Vellore, India
³Department of Haematology, Christian Medical College Vellore, Vellore, India
⁴Centre for Stem Cell Research (a Unit of inStem, Bengaluru), Vellore, India
⁵Christian Medical College, Vellore, Vellore, Tamil Nadu, India
⁶Department of Haematology, Christian Medical College, Vellore, India
⁷Department of Haematology, Christian Medical College & Hospital, Vellore, India
⁸Christian Medical College, Vellore, Vellore, IND
⁹Centre for Stem Cell Research (a unit of inStem, Bengaluru), Vellore, India
¹⁰Department of Haematology, Christian Medical College Vellore, Ranipet Campus, Ranipet, Tamil Nadu, India
¹¹Centre for Stem Cell Research (a unit of inStem, Bengaluru), Vellore, Tamil Nadu, India
¹²Department of Transfusion Medicine and Immunohematology, Christian Medical College, Vellore, India
¹³Expression Therapeutics, Inc., Tucker, GA
¹⁴Emory University, Children's Center, Atlanta, GA
¹⁵Department of Pediatrics, Aflac Cancer and Blood Disorders Center, Emory University and Children's Healthcare of Atlanta, Atlanta, GA

5:00 PM

1053

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults with Moderately Severe to Severe Hemophilia Α: Primary Analysis Results from the Phase 3 ΑFFINE Gene Therapy Trial

Andrew D. Leavitt, MD¹, Kaan Kavakli²^*, Laurent Frenzel, MD, PhD³^*, Ali Bülent Antmen, MD, PhD⁴^*, Margareth Ozelo, MD⁵, Davide Matino, MD, MSc^6,7^*, Hazza Alzahrani, MD⁸^*, Barbara A. Konkle, MD⁹, Steven W. Pipe, MD¹⁰, Jerome M. Teitel, MD¹¹, Li-Jung Tseng, PhD, MBA¹²^*, Annie F. Fang, MD, PhD¹²^*, Florence Ganne, PhD¹³^*, Gregory Dirusso, MD¹⁴, Jeremy Rupon, MD, PhD¹⁴, Pascal Klaus, MD¹⁵^*, Jasmine Healy, PhD¹⁶^*, Delphine Agathon, PhD¹³^*, Francesca Biondo, MD¹⁷^* and Frank Plonski, MA, RN, BS¹⁴^*

¹Department of Medicine and Department of Laboratory Medicine, University of California San Francisco, San Francisco, CA
²Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey
³Department of Haematology, Hemophilia Care and Research, Necker Hospital, Institut Imagine, Paris, France
⁴Department of Pediatric Hematology, Acibadem Adana Hospital, Adana, Turkey
⁵Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Brazil
⁶Thrombosis and Atherosclerosis Research Institute (TaARI), McMaster University, Hamilton, ON, Canada
⁷Department of Medicine, McMaster University, Hamilton, ON, Canada
⁸Adult Hematology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
⁹Washington Center for Bleeding Disorders and the University of Washington, Seattle, WA
¹⁰University of Michigan, Ann Arbor, MI
¹¹Division of Hematology, St. Michael's Hospital, University of Toronto, Toronto, ON, Canada
¹²Pfizer Inc, New York, NY
¹³Pfizer Inc, Paris, France
¹⁴Pfizer Inc, Collegeville, PA
¹⁵Pfizer Pharma GmbH, Berlin, Germany
¹⁶Pfizer Canada ULC, Kirkland, QC, Canada
¹⁷Pfizer S.r.l., Rome, Italy

5:15 PM

1054

Long-Term Factor VIII Expression Post AAV in Adult Hemophilia a Mice Demonstrates No Evidence of Adverse Liver Health or Tumorigenesis

Cristina Martos-Rus, MSc^1,2^*, Robert Davidson, BS^1,2^*, Charles-Antoine Assenmacher, DVM, MSc, DACVP³^* and Lindsey A. George, MD^1,2,4

¹Raymond G. Perelman Center for Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia, Philadelphia, PA
²Division of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA
³Comparative Pathology Core, Department of Pathobiology, University of Pennsylvania, School of Veterinary Medicine, Philadelphia, PA
⁴Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA

5:30 PM

1055

Site-Specific Insertion of Factor VIII Gene Results in Durable Factor VIII Expression in Nonhuman Primates

Alan Brooks, PhD¹^*, Janet Mei¹^*, Kalya Stanten¹^*, Luis Gamboa¹^*, Megan Van Overbeek, PhD¹^*, Paul Szymanski, PhD¹^*, Glenn Pierce, MD, PhD² and Sarah Noonberg, MD, PhD¹^*

¹Metagenomi, Emeryville, CA
²Independent Consultant, La Jolla, CA

5:45 PM

1056

Using Gene Therapy to Solve Challenges with CAR-T Cell Immunotherapy: Lead Selection and Preclinical Development of an Adeno-Associated Virus with Reduced Immunogenicity Exhibiting Efficient and Long-Term Expression of an Anti-CD19 T-Cell Engager

Mark A Currier, MS¹^*, Allen Reha, MS²^*, Brian Hutzen, PhD¹^*, Pin-Yi Wang, PhD¹^*, Chun-yu Chen, PhD¹^*, Andrea Glaspell, BS¹^*, Siddhi Nath Paudel, PhD¹^*, Akila S Venkataramany, BS¹^*, Debashree Das, MS²^*, Danielle Hanlon, BS²^*, Allan A. Kaspar, PhD²^*, Samit Varma, BS, MBA²^*, Brian K. Kaspar, PhD²^*, Jacob G. Scott, MD, DPhil³^*, Pablo Morales, DVM, DACLAM⁴^*, Peter Ralph, PhD⁵^*, Tonny Johnson, PhD⁵^*, Tania L. Weiss, PhD⁵^* and Timothy P Cripe, MD, PhD⁶^*

¹Center for Childhood Cancer Research, Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH
²Vironexis Biotherapeutics, Inc., San Diego, CA
³Department of Translational Hematology and Oncology Research, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH
⁴The Mannheimer Foundation, Inc., Homestead, FL
⁵Marin Biological Laboratories, Novato, CA
⁶Division of Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, OH

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^*signifies non-member of ASH

801.O2.6 801. Gene Therapies: Gene Therapies for Hemophilia, Cancer and Immunodeficiencies