Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

被引：13

作者：

Magrin, Elisa ^{[1
,2
]}

Semeraro, Michaela ^{[1
]}

Magnani, Alessandra ^{[3
,4
]}

Puy, Herve ^{[5
]}

Miccio, Annarita ^{[6
]}

Hebert, Nicolas ^{[7
,8
,9
]}

Diana, Jean-Sebastien

Lefrere, Francois

Suarez, Felipe

Hermine, Olivier

Brousse, Valentine

Poirot, Catherine

Bourget, Philippe

El Nemer, Wassim

Guichard, Isabelle

Moshous, Despina

Neven, Benedicte ^{[6
,10
]}

Monpoux, Fabrice ^{[11
]}

Poiree, Marilyne ^{[11
]}

Bartolucci, Pablo ^{[9
,12
]}

Meritet, Jean-Francois ^{[13
]}

Grevent, David ^{[14
]}

Lefebvre, Thibaud ^{[15
]}

Asmal, Mohammed ^{[16
]}

Whitney, Erin ^{[16
]}

Gayron, Marisa ^{[16
]}

Huang, Wenmei ^{[16
]}

Funck-Brentano, Isabelle ^{[1
]}

de Montalembert, Mariane ^{[17
]}

Joseph, Laure ^{[18
]}

Ribeil, Jean-Antoine ^{[16
]}

Cavazzana, Marina ^{[19
]}

机构：

[1] Necker Childrens Hosp, Assistance Publ Hop Paris, Paris, France

[2] Necker Assistance Publ Hop Paris, Biotherapy Dept, Paris, France

[3] Necker Enfants Malad Univ Hosp, Paris, France

[4] Grp Hosp Univ Ouest, Assistance Publ Hop Paris, Biotherapy Clin Invest Ctr, Paris, France

[5] Univ Paris Diderot, Sorbonne Paris Cite, Paris, France

[6] Univ Paris 05, IMAGINE Inst, Sorbonne Paris Cite, Paris, France

[7] Univ Paris Est Creteil, Creteil, France

[8] Hop Henri Mondor, Etab Francais Sang, Creteil, France

[9] Univ Paris Est Creteil, Inst Mondor, Lab Excellence GR Ex, INSERM U955,EFS,Team Transfus & Malad Globule Rou, Creteil, France

[10] Hop Necker Enfants Malad, Paris, France

[11] Univ Hosp Nice, Nice, France

[12] Henri Mondor Hosp, APHP, Creteil, France

[13] Hop Cochin St Vincent Paul, Paris, France

[14] Necker Childrens Hosp, Dept Pediat Neuroradiol, Paris, France

[15] Univ Paris, Louis Mourier Hosp, Assistance Publ Hop Paris, Paris, France

[16] Bluebird Bio Inc, Cambridge, MA USA

[17] Hop Necker Enfants Malad, APHP, Referral Ctr Sickle Cell Dis, Pediat, Paris, France

[18] Necker Childrens Hosp, Assistance Publ Hop Paris, Biotherapy Dept, Paris, France

[19] Hop Necker Enfants Malad, Grp Hosp Univ Ouest, Biotherapy Clin Invest Ctr, Paris, France

来源：

BLOOD | 2019年 / 134卷

关键词：

D O I：

10.1182/blood-2019-127393

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页数：4

共 50 条

[41] Major Challenges for Gene Therapy of Thalassemia and Sickle Cell Disease
Papanikolaou, Eleni
Anagnou, Nicholas P.
CURRENT GENE THERAPY, 2010, 10 (05) : 404 - 412
[42] Current status and developments in gene therapy for thalassemia and sickle cell disease
Yannaki, Evangelia
Karponi, Garyfalia
THALASSEMIA REPORTS, 2014, 4 (03) : 75 - 80
[43] Gene Therapy for Sickle Cell Disease, β-Thalassemia Enters Regulatory Reviews
Larkin, Howard D.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2022, 328 (18): : 1798 - 1798
[44] Sustained Improvements in Patient-Reported Quality of Life up to 24 Months Post-Treatment with LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy
Walters, Mark C.
Tisdale, John F.
Mapara, Markus Y.
Krishnamurti, Lakshmanan
Kwiatkowski, Janet L.
Aygun, Banu
Kasow, Kimberly A.
Rifkin-Zenenberg, Stacey
Jaroscak, Jennifer
Garbinsky, Diana
Chirila, Costel
Gallagher, Meghan E.
Zhang, Xinyan
Ho, Pei-Ran
Thompson, Alexis A.
Kanter, Julie
BLOOD, 2021, 138
[45] Gene Therapy for Hemoglobinopathies Beta-Thalassemia, Sickle Cell Disease
Leonard, Alexis
Tisdale, John F.
Bonner, Melissa
HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA, 2022, 36 (04) : 769 - 795
[46] Bench to Bedside-Gene Therapy for Thalassemia and Sickle Cell Disease
Leboulch, Philippe
BLOOD, 2015, 126 (23)
[47] Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Outcomes from the Phase 1/2 Northstar and Phase 3 Northstar-2 Studies
Anurathapan, Usanarat
Locatelli, Franco
Kwiatkowski, Janet L.
Rasko, John E. J.
Schiller, Gary J.
Porter, John
Sauer, Martin G.
Thrasher, Adrian J.
Chabannon, Christian
Elliot, Heidi
Deary, Briana
Chen, Ying
Tao, Ge
Asmal, Mohammed
Thompson, Alexis A.
Walters, Mark C.
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2019, 25 (03)
[48] Sickle Cell Disease Gene Therapy Trial Paused
Larkin, Howard D.
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2023, 329 (05): : 364 - 364
[49] EFFICACY AND SAFETY OF LENTIGLOBIN GENE THERAPY IN PATIENTS WITH TRANSFUSION-DEPENDENT β-THALASSEMIA AND NON-β0/β0 GENOTYPES: UPDATED RESULTS FROM THE COMPLETED PHASE 1/2 NORTHSTAR AND ONGOING PHASE 3 NORTHSTAR-2 STUDIES
Rasko, J.
Walters, M.
Kwiatkowski, J.
Hongeng, S.
Porter, J.
Sauer, M.
Thrasher, A.
Thuret, I.
Schiller, G.
Elliot, H.
Deary, B.
Chen, Y.
Tao, G.
Asmal, M.
Locatelli, F.
Thompson, A.
CYTOTHERAPY, 2019, 21 (05) : S14 - S14
[50] A PHASE 3 STUDY TO EVALUATE SAFETY AND EFFICACY OF LENTIGLOBIN GENE THERAPY FOR TRANSFUSION-DEPENDENT B-THALASSEMIA IN PATIENTS WITH NON-B0/B0 GENOTYPES: THE NORTHSTAR-2 (HGB-207) TRIAL
Walters, M.
Thompson, A.
Hongeng, S.
Kwiatkowski, J.
Locatelli, F.
Porter, J.
Sauer, M.
Thrasher, A.
Thuret, I.
Yannaki, E.
Petrusich, A.
Asmal, M.
HAEMATOLOGICA, 2017, 102 : 335 - 335

← 1 2 3 4 5 →