JP7228523B2 - ヘモグロビン異常症を治療するためのベクターおよび組成物 - Google Patents

ヘモグロビン異常症を治療するためのベクターおよび組成物 Download PDF

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JP7228523B2
JP7228523B2 JP2019553350A JP2019553350A JP7228523B2 JP 7228523 B2 JP7228523 B2 JP 7228523B2 JP 2019553350 A JP2019553350 A JP 2019553350A JP 2019553350 A JP2019553350 A JP 2019553350A JP 7228523 B2 JP7228523 B2 JP 7228523B2
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JP2020511991A5 (enExample
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ガボール ベレス，
デイビッド エー． ウィリアムズ，
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Boston Childrens Hospital
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JP2019553350A 2017-03-29 2018-03-29 ヘモグロビン異常症を治療するためのベクターおよび組成物 Active JP7228523B2 (ja)

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US201762478375P 2017-03-29 2017-03-29
US62/478,375 2017-03-29
US201762489149P 2017-04-24 2017-04-24
US62/489,149 2017-04-24
PCT/US2018/025165 WO2018183692A1 (en) 2017-03-29 2018-03-29 Vectors and compositions for treating hemoglobinopathies

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JP2023033551A (ja) * 2017-03-29 2023-03-10 ブルーバード バイオ， インコーポレイテッド ヘモグロビン異常症を治療するためのベクターおよび組成物

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EP3160478A4 (en) 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
US20220160788A1 (en) * 2019-03-22 2022-05-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Bifunctional vectors allowing bcl11a silencing and expression of an anti-sickling hbb and uses thereof for gene therapy of b-hemoglobinopathies
JP2022534798A (ja) * 2019-06-05 2022-08-03 オーチャード セラピューティクス（ヨーロッパ）リミテッド 真核細胞を修飾するための組成物及び方法
CN115279896A (zh) * 2019-09-30 2022-11-01 比奥维拉迪维治疗股份有限公司 慢病毒载体配制品
CN117384967A (zh) * 2020-04-24 2024-01-12 康霖生物科技（杭州）有限公司 一种核酸构建体
EP4222271A4 (en) * 2020-11-23 2025-07-30 Intas Pharmaceuticals Ltd LENTIVIRUS VECTOR GENE THERAPY-BASED DELIVERY FOR THE TREATMENT OF HEMOGLOBINOPATHIES

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WO2015164750A2 (en) 2014-04-25 2015-10-29 Children's Medical Center Corporation Compositions and methods to treating hemoglobinopathies
WO2015164759A2 (en) 2014-04-25 2015-10-29 Bluebird Bio, Inc. Mnd promoter chimeric antigen receptors
WO2016037138A1 (en) 2014-09-04 2016-03-10 Memorial Sloan-Kettering Cancer Center Global gene therapy for treating hemoglobinopathies
WO2016094304A2 (en) 2014-12-12 2016-06-16 Bluebird Bio, Inc. Bcma chimeric antigen receptors
WO2016156404A1 (en) 2015-03-31 2016-10-06 Glycotope Gmbh Eukaryotic expression vectors comprising regulatory elements of the globin gene clusters
WO2017040529A1 (en) 2015-08-31 2017-03-09 Bluebird Bio, Inc. Anti-sialyl tn chimeric antigen receptors

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UA114796C2 (uk) * 2011-09-30 2017-08-10 Блубьод Байо, Інк. Спосіб підвищення ефективності трансдукції
WO2014093965A1 (en) * 2012-12-14 2014-06-19 Case Western Reserve University Genomic rna packaging enhancer element
EP3134434A4 (en) * 2014-04-25 2017-10-25 Bluebird Bio, Inc. Kappa/lambda chimeric antigen receptors
US11261441B2 (en) * 2017-03-29 2022-03-01 Bluebird Bio, Inc. Vectors and compositions for treating hemoglobinopathies
JP7228523B2 (ja) * 2017-03-29 2023-02-24 ブルーバード バイオ， インコーポレイテッド ヘモグロビン異常症を治療するためのベクターおよび組成物

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WO2015164750A2 (en) 2014-04-25 2015-10-29 Children's Medical Center Corporation Compositions and methods to treating hemoglobinopathies
WO2015164759A2 (en) 2014-04-25 2015-10-29 Bluebird Bio, Inc. Mnd promoter chimeric antigen receptors
WO2016037138A1 (en) 2014-09-04 2016-03-10 Memorial Sloan-Kettering Cancer Center Global gene therapy for treating hemoglobinopathies
WO2016094304A2 (en) 2014-12-12 2016-06-16 Bluebird Bio, Inc. Bcma chimeric antigen receptors
WO2016156404A1 (en) 2015-03-31 2016-10-06 Glycotope Gmbh Eukaryotic expression vectors comprising regulatory elements of the globin gene clusters
WO2017040529A1 (en) 2015-08-31 2017-03-09 Bluebird Bio, Inc. Anti-sialyl tn chimeric antigen receptors

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Publication number Priority date Publication date Assignee Title
JP2023033551A (ja) * 2017-03-29 2023-03-10 ブルーバード バイオ， インコーポレイテッド ヘモグロビン異常症を治療するためのベクターおよび組成物

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WO2018183692A1 (en) 2018-10-04
BR112019020322A2 (pt) 2020-04-28
AU2018243293A1 (en) 2019-10-31
SG11201908844YA (en) 2019-10-30
IL269551A (en) 2019-11-28
EP3600448A4 (en) 2021-01-27
US20220259594A1 (en) 2022-08-18
CA3057862A1 (en) 2018-10-04
EP3600448A1 (en) 2020-02-05
JP2023033551A (ja) 2023-03-10
WO2018183692A8 (en) 2019-05-02
CN110582305A (zh) 2019-12-17
JP2020511991A (ja) 2020-04-23

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