Interim Results of Betibeglogene Autotemcel Gene Therapy in Pediatric Patients With Transfusion-dependent B-Thalassemia (TDT) Treated in The Phase 3 Northstar-2 (HGB-207) And Northstar-3 (HGB-212) Studies

被引：0
|
作者：
Thuret, Isabelle [1 ]
Thompson, Alexis [2 ,3 ]
Kwiatkowski, Janet [4 ,5 ]
Porter, John [6 ]
Hongeng, Suradej [7 ]
Yannaki, Evangelia [8 ]
Kulozik, Andreas [9 ]
Sauer, Martin [10 ]
Thrasher, Adrian [11 ,12 ]
Lal, Ashutosh [13 ]
Guo, Ruiting [14 ]
Liu, Weijian [14 ]
Colvin, Richard [14 ]
Walters, Mark [13 ]
Locatelli, Franco [15 ]
机构：
[1] Hop La Timone, Marseille, France
[2] Ann & Robert H Lurie Childrens Hosp, Chicago, IL USA
[3] Northwestern Univ, Feinberg Sch Med, Chicago, IL 60611 USA
[4] Univ Penn, Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA
[5] Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA
[6] Univ Coll London Hosp, London, England
[7] Mahidol Univ, Ramathibodi Hosp, Bangkok, Thailand
[8] G Papanikolaou Hosp, Thessaloniki, Greece
[9] Heidelberg Univ, Heidelberg, Germany
[10] Hannover Med Sch, Hannover, Germany
[11] UCL, Great Ormond St Hosp Inst Child Hlth, London, England
[12] Great Ormond St Hosp NHS Trust, London, England
[13] UCSF Benioff Childrens Hosp, Oakland, CA USA
[14] Bluebird Bio Inc, Cambridge, MA USA
[15] IRCCS Bambino Gesu Childrens Hosp, Rome, Italy
来源：
BONE MARROW TRANSPLANTATION | 2021年 / 56卷 / SUPPL 1期
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：
O054
引用
收藏
页码：64 / 65
页数：2
相关论文
共 27 条
  • [1] Interim results of betibeglogene autotemcel gene-additiontherapy in pediatric patients with transfusion-dependent β-thalassemia (TDT) treated in the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies
    Kulozik, A. E.
    Kunz, J.
    Thuret, I
    Thompson, A. A.
    Kwiatkowski, J. L.
    Porter, J. B.
    Hongeng, S.
    Yannaki, E.
    Sauer, M. G.
    Thrasher, A. J.
    Lal, A.
    Guo, R.
    Colvin, R. A.
    Walters, M. C.
    Locatelli, F.
    ONCOLOGY RESEARCH AND TREATMENT, 2021, 44 : 8 - 8
  • [2] Interim Results from the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy (LentiGlobin) for the Treatment of Transfusion-Dependent β-Thalassemia
    Schneiderman, Jennifer
    Thompson, Alexis A.
    Walters, Mark C.
    Kwiatkowski, Janet L.
    Kulozik, Andreas E.
    Sauer, Martin G.
    Porter, John B.
    Thuret, Isabelle
    Hongeng, Suradej
    Lal, Ashutosh
    Thrasher, Adrian J.
    Yannaki, Evangelia
    Elliot, Heidi
    Tao, Ge
    Liu, Weijian
    Colvin, Richard A.
    Locatelli, Franco
    BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2020, 26 (03) : S87 - S88
  • [3] Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia
    Thompson, Alexis A.
    Kwiatkowski, Janet L.
    Porter, John B.
    Hongeng, Suradej
    Yannaki, Evangelia
    Kulozik, Andreas E.
    Sauer, Martin G.
    Thrasher, Adrian J.
    Thuret, Isabelle
    Lal, Ashutosh
    Guo, Ruiting
    Liu, Weijian
    Colvin, Richard A.
    Walters, Mark C.
    Locatelli, Franco
    BLOOD, 2020, 136
  • [4] Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies
    Locatelli, Franco
    Walters, Mark C.
    Kwiatkowski, Janet L.
    Porter, John
    Sauer, Martin G.
    Thuret, Isabelle
    Hongeng, Suradej
    Kulozik, Andreas E.
    Lal, Ashutosh
    Thrasher, Adrian J.
    Yannaki, Evangelia
    Elliot, Heidi
    Tao, Ge
    Asmal, Mohammed
    Thompson, Alexis A.
    BLOOD, 2018, 132
  • [5] Safety and Efficacy of LentiGlobin Gene Therapy for Transfusion-Dependent β-thalassemia (TDT) in Patients with non-β0/β0 Genotypes: Interim results from the HGB-204 (Northstar) and HGB-207 (Northstar-2) Trials
    Porter, J. B.
    Walters, M. C.
    Kwiatkowski, J. L.
    Rasko, J. E. J.
    Hongeng, S.
    Schiller, G. J.
    Locatelli, F.
    Sauer, M.
    Thrasher, A.
    Thuret, I.
    Yannaki, E.
    Elliot, H.
    Deary, B.
    Gayron, M.
    Ramirez-Santiago, A.
    Thompson, A. A.
    BRITISH JOURNAL OF HAEMATOLOGY, 2018, 181 : 30 - 30
  • [6] FINAL RESULTS OF THE PHASE 3 STUDIES NORTHSTAR-2 AND NORTHSTAR-3 IN PATIENTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA (TDT): SUBGROUP ANALYSIS OF ITALIAN PATIENTS TREATED WITH BETIBEGLOGENE AUTOTEMCEL (BETI-CEL), LENTIGLOBIN FOR THALASSEMIA
    Locatelli, F.
    Pagliara, D.
    Gaspari, S.
    Galaverna, F.
    Pira, G. Li
    Coletti, V.
    Merli, P.
    Palumbo, G.
    Leone, G.
    Guo, R.
    Colvin, R. A.
    Algeri, M.
    HAEMATOLOGICA, 2021, 106 (10) : 32 - 32
  • [7] CLINICAL OUTCOMES FOLLOWING AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION WITH BETIBEGLOGENE AUTOTEMCEL GENE THERAPY IN THE PHASE 3 NORTHSTAR-2 AND NORTHSTAR-3 STUDIES FOR TRANSFUSION-DEPENDENT β-THALASSEMIA (TDT)
    Locatelli, F.
    Kwiatkowski, J. L.
    Thompson, A. A.
    Yannaki, E.
    Kulozik, A. E.
    Sauer, M. G.
    Porter, J. B.
    Thuret, I.
    Hongeng, S.
    Lal, A.
    Thrasher, A. J.
    Tao, G.
    Liu, W.
    Colvin, R. A.
    Walters, M. C.
    HAEMATOLOGICA, 2020, 105 : S36 - S36
  • [8] Interim results from the phase 3 Northstar-2 and Northstar-3 studies of LentiGlobin gene therapy for the treatment of transfusion-dependent β-thalassemia
    Kunz, J.
    Locatelli, F.
    Kulozik, A.
    Sauer, M.
    Kwiatkowski, J.
    Porter, J.
    Thuret, I.
    Hongeng, S.
    Lal, A.
    Thrasher, A.
    Yannaki, E.
    Elliot, H.
    Tao, G.
    Chen, Y.
    Colvin, R.
    Walters, M.
    Thompson, A.
    ONCOLOGY RESEARCH AND TREATMENT, 2019, 42 : 301 - 301
  • [9] Clinical outcomes following autologous hematopoietic stem cell transplantation with betibeglogene autotemcel (beti-cel, LentiGlobin for β-thalassemia) gene therapy in the phase 3 Northstar-2 and Northstar-3 studies for transfusion-dependent β-thalassemia (TDT)
    Kulozik, A. E.
    Locatelli, F.
    Kunz, J.
    Kwiatkowski, J. L.
    Thompson, A. A.
    Yannaki, E.
    Sauer, M. G.
    Schambach, A.
    Schmidt, M.
    Porter, J. B.
    Thuret, I.
    Hongeng, S.
    Lal, A.
    Thrasher, A. J.
    Tao, G.
    Liu, W.
    Colvin, R. A.
    Walters, M. C.
    ONCOLOGY RESEARCH AND TREATMENT, 2020, 43 (SUPPL 4) : 33 - 33
  • [10] Interim results from the phase 3 Northstar-2 and Northstar-3 studies of LentiGlobin gene therapy for the treatment of transfusion-dependent β-thalassaemia
    Yannaki, E.
    Thompson, A. A.
    Kulozik, A.
    Sauer, M.
    Kwiatkowski, J. L.
    Porter, J.
    Thuret, I.
    Hongeng, S.
    Lal, A.
    Thrasher, A. J.
    Elliot, H.
    Tao, G.
    Chen, Y.
    Colvin, R. A.
    Walters, M. C.
    Locatelli, F.
    HUMAN GENE THERAPY, 2019, 30 (11) : A10 - A11
123