62nd ASH Annual Meeting and Exposition: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical:Novel Treatments for Sickle Cell Disease

Oral and Poster Abstracts
Oral

Adult, Biological, sickle cell disease, Diseases, Non-Biological, thalassemia, Therapies, cellular interactions, Pediatric, red blood cells, Biological Processes, Hemoglobinopathies, gene therapy, Young Adult, Cell Lineage, opioids, Study Population, Clinically relevant, pharmacology, inflammation, molecular interactions

Moderators:

Parul Rai, MD, MBBS, St Jude Children's Research Hospital and Kerry A Morrone, MD, Children's Hospital At Montefiore

Disclosures:

No relevant conflicts of interest to declare.

1:30 PM

677

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Alexis A. Thompson, MD^1,2, Mark C. Walters, MD³, Markus Y Mapara, MD, PhD⁴, Janet L. Kwiatkowski, MD, MSCE^5,6, Lakshmanan Krishnamurti, MD⁷, Banu Aygun, MD^8,9, Kimberly A. Kasow, DO¹⁰^*, Stacey Rifkin-Zenenberg, DO¹¹, Manfred Schmidt, PhD¹²^*, Jay DelCarpini¹³^*, Francis J. Pierciey Jr.¹³^*, Alexandra L. Miller¹³^*, Meghan E. Gallagher, MSc¹³^*, Ren Chen¹³^*, Sunita Goyal, MD¹³^*, Julie Kanter, MD¹⁴ and John F. Tisdale, MD^15,16

¹Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL
²Northwestern University Feinberg School of Medicine, Chicago, IL
³UCSF Benioff Children's Hospital Oakland, Oakland, CA
⁴Columbia University Medical Center, New York, NY
⁵University of Pennsylvania Perelman School of Medicine, Philadelphia, PA
⁶The Children's Hospital of Philadelphia, Philadelphia, PA
⁷Aflac Cancer and Blood Disorders Center, Children’s Healthcare of Atlanta, Emory University, Atlanta, GA
⁸Cohen Children’s Medical Center, New Hyde Park, NY
⁹Zucker School of Medicine at Hofstra/Northwell, Hempstead, NY
¹⁰University of North Carolina At Chapel Hill, Chapel Hill, NC
¹¹Joseph M. Sanzari Children’s Hospital, Hackensack University Medical Center, Livingston, NJ
¹²GeneWerk GmbH, Heidelberg, Germany
¹³bluebird bio, Inc., Cambridge, MA
¹⁴University of Alabama at Birmingham, Birmingham, AL
¹⁵Clinical and Molecular Therapeutics Branch, NHLBI / NIDDK, NIH, Bethesda, MD
¹⁶National Institutes of Health, Bethesda, MD

1:45 PM

678

Early Initiation of Treatment with Rivipansel for Acute Vaso-Occlusive Crisis in Sickle Cell Disease (SCD) Achieves Earlier Discontinuation of IV Opioids and Shorter Hospital Stay: Reset Clinical Trial Analysis

Carlton D. Dampier, MD¹, Marilyn J. Telen, MD², Ted Wun, MD³, Wally R Smith, MD⁴, R. Clark Brown, MD, PhD⁵, Payal Desai, MD⁶, Fuad A El Rassi, MD⁷, Julie Kanter, MD⁸, Beng R. Fuh, MD⁹, Yves D. Pastore, MD¹⁰, Jennifer A. Rothman, MD¹¹, James G. Taylor, MD¹², David Readett, BM, BS, MRCP¹³^*, Jay N Lozier, MD, PhD¹⁴, John L. Magnani, PhD¹⁵, Helen M Thackray, MD¹⁶ and Kathryn L. Hassell, MD¹⁷

¹Emory University, Atlanta, GA
²Duke University Medical Center, Duke Comprehensive Sickle Cell Center, Durham, NC
³Division of Hematology Oncology, UC Davis School of Medicine, Sacramento, CA
⁴Virginia Commonwealth University, Richmond, VA
⁵Department of Pediatrics, Aflac Cancer & Blood Disorders Center of Children’s Healthcare of Atlanta and the Department of Pediatrics, Emory University, Atlanta, GA
⁶The Ohio State University, Columbus, OH
⁷Department of Hematology and Medical Oncology, Winship Cancer Institute, Atlanta, GA
⁸University of Alabama, Director of the UAB Adult Sickle Cell Clinic Associate Professor in the Division of Hematology and Oncology, Birmingham, AL
⁹East Carolina University Comprehensive Sickle Cell Center, Greenville, NC
¹⁰CHU Sainte-Justine, University of Montreal, Montreal, QC, Canada
¹¹Duke University Medical Center, Durham, NC
¹²Center for Sickle Cell Disease, Howard University, Washington, DC
¹³Pfizer Inc., Groton, CT
¹⁴Jay Lozier Consulting, LLC, Olney, MD
¹⁵Glycomimetics, Inc., Rockville, MD
¹⁶GlycoMimetics Inc., Rockville, MD
¹⁷University of Colorado Denver HSC, Aurora, CO

2:00 PM

679

FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease

R. Clark Brown, MD, PhD¹, Kimberly Cruz, MD, MS²^*, Theodosia A. Kalfa, MD, PhD³, Frans A. Kuypers, PhD⁴^*, Santosh L. Saraf, MD⁵, Jeremie H. Estepp, MD⁶, Luke R. Smart, MD³, Punam Malik, MD⁷, Mark Lerman, MD⁸^*, Renae Mayer, MD⁹^*, Maria D. Ribadeneira, PhD¹⁰^*, Sanjeev Forsyth, MS¹⁰^*, Patricia Schroeder, PhD¹⁰^*, Eric Wu, PhD¹⁰^*, Patrick Kelly, MD¹⁰ and Marilyn J. Telen, MD¹¹

¹Department of Pediatrics, Aflac Cancer & Blood Disorders Center of Children’s Healthcare of Atlanta and the Department of Pediatrics, Emory University, Atlanta, GA
²Advanced Pharma, Miami, FL
³Division of Hematology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁴Children's Hospital Oakland Research Institute, Oakland, CA
⁵Department of Medicine, University of Illinois at Chicago, Chicago, IL
⁶Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN
⁷Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁸Atlanta Center for Medical Research, Atlanta, GA
⁹Lynn Institute, Tulsa, OK
¹⁰Forma Therapeutics, Inc., Watertown, MA
¹¹Duke Univ. Med. Ctr., Durham, NC

2:15 PM

680

Early Results from a Phase 1/2 Study of Aru-1801 Gene Therapy for Sickle Cell Disease (SCD): Manufacturing Process Enhancements Improve Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant

Michael Grimley, MD¹, Monika Asnani, PhD²^*, Archana Shrestha³^*, Sydney Felker^4,5^*, Carolyn Lutzko, PhD⁶^*, Paritha I. Arumugam, PhD⁴^*, Scott Witting, PhD⁷^*, Jennifer Knight-Madden, MBBS, PhD⁸^*, Omar Niss, MD⁹, Charles T. Quinn, MD⁶, Chris Lo, BS¹⁰^*, Courtney R Little, BSN¹¹^*, Joseph W. McIntosh, MD¹⁰^* and Punam Malik, MD⁴

¹Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
²Carribean Health Research Institute, Kingston, Jamaica
³Cincinnati Children's Hospital and Medical Center, Cincinnati, OH
⁴Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁵Immunology Graduate Program, University of Cincinnati College of Medicine, Cincinnati, OH
⁶Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH
⁷Cincinnati Children's Hospital, Cincinnati, OH
⁸Caribbean Institute for Health Research, The University of the West Indies, Kingston, Jamaica
⁹Division of Hematology, Cincinnati Children's Hospital, Cincinnati, OH
¹⁰Aruvant Sciences, New York, NY
¹¹Aruvant Sciences, Felicity, OH

2:30 PM

681

Phase 1 Multiple Ascending Dose Study of Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Mitapivat (AG-348) in Subjects with Sickle Cell Disease

Julia Z. Xu, MD, MS¹, Anna Conrey, NP¹^*, Ingrid Frey, RN, BSN, MS¹^*, James Nichols, RN¹^*, Laurel A Menapace, MD¹, Laxminath Tumburu, PhD¹^*, Timothy Lequang, MSc¹^*, Quan Li, PhD²^*, Emily Dunkelberger, PhD²^*, Eric Henry, PhD²^*, Troy Cellmer, PhD²^*, Varsha Iyer³^*, Heidi Mangus, BS³^*, Charles Kung, PhD³^*, Lenny Dang, PhD³^*, Penelope A. Kosinski, MS³^*, Peter Hawkins, PhD³^*, Neal Jeffries, PhD⁴^*, William Eaton, MD² and Swee Lay Thein, DSc, FRCP, MBBS, MRCP¹

¹Sickle Cell Branch, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD
²National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD
³Agios Pharmaceuticals, Inc., Cambridge, MA
⁴Office of Biostatistics Research, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD

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^*signifies non-member of ASH

114.O3.6 114. Hemoglobinopathies, Excluding Thalassemia—Clinical:Novel Treatments for Sickle Cell Disease