Resolution of Serious Vaso-occlusive Pain Crises: Results From The Ongoing Phase 1/2 HGB-206 Group C Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

被引:0
作者
Mapara, Markus [1 ]
Walters, Mark [2 ]
Thompson, Alexis [3 ,4 ]
Kwiatkowski, Janet [5 ,6 ]
Krishnamurti, Lakshmanan [7 ]
Aygun, Banu [8 ]
Kasow, Kimberly [9 ]
Rifkin-Zenenberg, Stacey [10 ]
Schmidt, Manfred [11 ]
Nnamani, Mauris [12 ]
VanNest, Sara [12 ]
Pierciey, Francis, Jr. [12 ]
Miller, Alex [12 ]
Chen, Ren [12 ]
Kim, Dennis [12 ]
Goyal, Sunita [12 ]
Kanter, Julie [13 ]
Tisdale, John [14 ]
机构
[1] Columbia Univ, Med Ctr, New York, NY USA
[2] UCSF Benioff Childrens Hosp Oakland, Oakland, CA USA
[3] Northwestern Univ, Feinberg Sch Med, Chicago, IL 60611 USA
[4] Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL USA
[5] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[6] Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA
[7] Emory Univ, Childrens Healthcare Atlanta, Atlanta, GA 30322 USA
[8] Cohen Childrens Med Ctr, Queens, NY USA
[9] Univ N Carolina, Chapel Hill, NC 27515 USA
[10] Hackensack Univ, Med Ctr, Hackensack, NJ USA
[11] GeneWerk GmbH, Heidelberg, Germany
[12] Bluebird Bio Inc, Cambridge, MA USA
[13] Univ Alabama Birmingham, Birmingham, AL USA
[14] NIH, Bldg 10, Bethesda, MD 20892 USA
关键词
D O I
暂无
中图分类号
Q6 [生物物理学];
学科分类号
071011 ;
摘要
O056
引用
收藏
页码:66 / 68
页数:3
相关论文
共 42 条
  • [31] Initial Results from the BEACON Clinical Study: A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises
    Gupta, Ashish O.
    Sharma, Akshay
    Frangoul, Haydar
    Dalal, Jignesh
    Kanter, Julie
    Alavi, Asif
    Dipersio, John
    Eapen, Mary
    Jaroscak, Jennifer J.
    Ayala, Ernesto
    Ziga, Edward D.
    Rifkin-Zenenberg, Stacey
    Minella, Alex C.
    Chen, Guo
    Chen, Yinzhong
    Chockalingam, Priya S.
    Lin, Ling
    Joseney-Antoine, Marcelyne
    Ianniello, Leanne
    Gardner, Beth
    Hartigan, Adam J.
    Ciaramella, Giuseppe
    Goyal, Sunita
    Simon, Amy
    Thompson, Alexis
    Heeney, Matthew M.
    [J]. BLOOD, 2024, 144 : 514 - 515
  • [32] GMI 1070: Reduction In Time To Resolution Of Vaso-Occlusive Crisis and Decreased Opioid Use In a Prospective, Randomized, Multi-Center Double Blind, Adaptive Phase 2 Study In Sickle Cell Disease
    Telen, Marilyn J.
    Wun, Ted
    McCavit, Timothy L.
    De Castro, Laura M.
    Krishnamurti, Lakshmanan
    Lanzkron, Sophie
    Hsu, Lewis L.
    Smith, Wally R.
    Rhee, Seungshin
    Magnani, John L.
    Thackray, Helen
    [J]. BLOOD, 2013, 122 (21)
  • [33] Preliminary Safety and Efficacy Results from Precizn-1: An Ongoing Phase 1/2 Study on Zinc Finger Nuclease-Modified Autologous CD34+HSPCs for Sickle Cell Disease (SCD)
    Alavi, Asif
    Krishnamurti, Lakshmanan
    Abedi, Mehrdad
    Galeon, Isobelle
    Reiner, David
    Smith, Sharon E.
    Wang, Lin
    Ramezi, Anne
    Rendo, Pablo
    Walters, Mark C.
    [J]. BLOOD, 2021, 138
  • [34] Early Results from a Phase 1/2 Study of ARU-1801 Gene Therapy for Sickle Cell Disease (SCD): Safety and Efficacy of a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant
    Grimley, Michael
    Asnani, Monika
    Shrestha, Archana
    Felker, Sydney
    Lutzko, Carolyn
    Arumugam, Paritha I.
    Witting, Scott
    Knight-Madden, Jennifer
    Niss, Omar
    Quinn, Charles T.
    Lo, Christopher
    Little, Courtney R.
    McIntosh, Joseph W.
    Malik, Punam
    [J]. MOLECULAR THERAPY, 2021, 29 (04) : 39 - 40
  • [35] Trials in Progress: The Randomized, Double-Blind, Placebo-Controlled Phase 1b CROSSWALK-a and Phase 2a CROSSWALK-c Trials - Crovalimab for the Treatment and Prevention of Vaso-Occlusive Episodes in Sickle Cell Disease
    Salvino, Marco Aurelio
    Antmen, Bulent
    Asirwa, Frederick Chite
    Chonat, Satheesh
    De Franceschi, Lucia
    Eleftheriou, Perla
    Inati, Adlette
    Mahlangu, Johnny
    Nur, Erfan
    Payan-Pernia, Salvador
    Charania, Asad
    Gradinaru, Diana
    Hsia, Allison
    Luder, Yves
    Perretti, Thomas
    Srekovic, Sasha
    Bartolucci, Pablo
    [J]. BLOOD, 2023, 142
  • [36] Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)
    Hopkin, Robert J.
    Ganesh, Jaya
    Bernat, John
    Goker-Alpan, Ozlem
    Nicholls, Kathleen
    Pahl, Madeleine V.
    Deegan, Patrick B.
    Whitley, Chester B.
    Hughes, Derralynn
    Cao, Liching
    Chen, Michael
    Shiue, Lisa
    Rojkjaer, Lisa
    Wilcox, William R.
    [J]. MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
  • [37] Isaralgagene civaparvovec (ST-920) gene therapy in adults with Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)
    Hughes, Derralynn
    Wilcox, William
    Hopkin, Robert J.
    Ganesh, Jaya
    Bernat, John
    Goker-Alpan, Ozlem
    Nicholls, Kathy
    Deegan, Patrick
    Pahl, Madeleine
    Whitley, Chester B.
    Chen, Michael
    Cao, Liching
    Schreeb, Katharina H.
    [J]. MOLECULAR GENETICS AND METABOLISM, 2025, 144 (02)
  • [38] 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
    Grimley, Michael
    Asnani, Monika
    Shrestha, Archana
    Felker, Sydney
    Lutzko, Carolyn
    Arumugam, Paritha I.
    Witting, Scott
    Knight-Madden, Jennifer
    Niss, Omar
    Quinn, Charles T.
    Lo, Chris
    Little, Courtney R.
    McIntosh, Joseph W.
    Malik, Punam
    [J]. BLOOD, 2020, 136
  • [39] Safety and Efficacy of Aru-1801 in Patients with Sickle Cell Disease: Early Results from the Phase 1/2 Momentum Study of a Modified Gamma Globin Gene Therapy and Reduced Intensity Conditioning
    Grimley, Michael
    Asnani, Monika
    Shrestha, Archana
    Felker, Sydney
    Lutzko, Carolyn
    Arumugam, Paritha I.
    Witting, Scott
    Knight-Madden, Jennifer
    Niss, Omar
    Quinn, Charles T.
    Lo, Chris
    Little, Courtney R.
    Dong, Alisa
    Malik, Punam
    [J]. BLOOD, 2021, 138
  • [40] Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral βA-T87Q Globin Vector (LentiGlobin BB305 Drug Product)
    Thompson, Alexis A.
    Rasko, John E.
    Hongeng, Suradej
    Kwiatkowski, Janet L.
    Schiller, Gary
    von Kalle, Christof
    Cavazzana, Marina
    Leboulch, Philippe
    Petrusich, Alexandria
    Soni, Sandeep
    Walters, Mark C.
    [J]. BLOOD, 2014, 124 (21)