CN110582305A - 用于治疗血红蛋白病的载体和组合物 - Google Patents

用于治疗血红蛋白病的载体和组合物 Download PDF

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Publication number
CN110582305A
CN110582305A CN201880027694.4A CN201880027694A CN110582305A CN 110582305 A CN110582305 A CN 110582305A CN 201880027694 A CN201880027694 A CN 201880027694A CN 110582305 A CN110582305 A CN 110582305A
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China
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cell
cells
lentiviral vector
hiv
beta
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CN201880027694.4A
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Chinese (zh)
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加博尔·沃莱斯
大卫·A·威廉姆斯
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Boston Childrens Hospital
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Boston Childrens Hospital
Bluebird Bio Inc
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2740/10011—Retroviridae
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    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
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    • C12N2740/10011—Retroviridae
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201880027694.4A 2017-03-29 2018-03-29 用于治疗血红蛋白病的载体和组合物 Pending CN110582305A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
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

Publications (1)

Publication Number Publication Date
CN110582305A true CN110582305A (zh) 2019-12-17

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CN201880027694.4A Pending CN110582305A (zh) 2017-03-29 2018-03-29 用于治疗血红蛋白病的载体和组合物

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Country Link
US (1) US20220259594A1 (enExample)
EP (1) EP3600448A4 (enExample)
JP (2) JP7228523B2 (enExample)
CN (1) CN110582305A (enExample)
AU (1) AU2018243293A1 (enExample)
BR (1) BR112019020322A2 (enExample)
CA (1) CA3057862A1 (enExample)
IL (1) IL269551A (enExample)
SG (1) SG11201908844YA (enExample)
WO (1) WO2018183692A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113549654A (zh) * 2020-04-24 2021-10-26 康霖生物科技（杭州）有限公司 一种核酸构建体

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WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene
CN110582305A (zh) * 2017-03-29 2019-12-17 蓝鸟生物公司 用于治疗血红蛋白病的载体和组合物
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
CN114450412A (zh) * 2019-06-05 2022-05-06 奥查德疗法(欧洲)有限公司 用于修饰真核细胞的组合物和方法
WO2021067389A1 (en) * 2019-09-30 2021-04-08 Bioverativ Therapeutics Inc. Lentiviral vector formulations
US20240350664A1 (en) * 2020-11-23 2024-10-24 Intas Pharmaceuticals Ltd. Gene therapy based administration of lentivirus vector for treating hemoglobinopathies

Citations (3)

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CN103958667A (zh) * 2011-09-30 2014-07-30 蓝鸟生物公司 用于改善病毒转导的化合物
WO2015164750A2 (en) * 2014-04-25 2015-10-29 Children's Medical Center Corporation Compositions and methods to treating hemoglobinopathies
WO2016094304A2 (en) * 2014-12-12 2016-06-16 Bluebird Bio, Inc. Bcma chimeric antigen receptors

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EP3208339B1 (en) * 2008-09-15 2019-05-01 The Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
US10106816B2 (en) * 2012-12-14 2018-10-23 Case Western Reserve University Genomic RNA packaging enhancer element
SMT202100700T1 (it) 2014-04-25 2022-01-10 2Seventy Bio Inc Recettori antigenici chimerici con promotore mnd
US20170049819A1 (en) * 2014-04-25 2017-02-23 Bluebird Bio, Inc. Kappa/lambda chimeric antigen receptors
CA2960209C (en) 2014-09-04 2023-08-29 Memorial Sloan-Kettering Cancer Center Globin gene therapy for treating hemoglobinopathies
EP3277807B1 (en) 2015-03-31 2019-12-11 Glycotope GmbH Eukaryotic expression vectors comprising regulatory elements of the globin gene clusters
CN108136014A (zh) 2015-08-31 2018-06-08 蓝鸟生物公司 抗唾液酸tn嵌合抗原受体
CN110582305A (zh) * 2017-03-29 2019-12-17 蓝鸟生物公司 用于治疗血红蛋白病的载体和组合物
US11261441B2 (en) * 2017-03-29 2022-03-01 Bluebird Bio, Inc. Vectors and compositions for treating hemoglobinopathies

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103958667A (zh) * 2011-09-30 2014-07-30 蓝鸟生物公司 用于改善病毒转导的化合物
WO2015164750A2 (en) * 2014-04-25 2015-10-29 Children's Medical Center Corporation Compositions and methods to treating hemoglobinopathies
WO2016094304A2 (en) * 2014-12-12 2016-06-16 Bluebird Bio, Inc. Bcma chimeric antigen receptors

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113549654A (zh) * 2020-04-24 2021-10-26 康霖生物科技（杭州）有限公司 一种核酸构建体
CN113549654B (zh) * 2020-04-24 2023-11-07 康霖生物科技（杭州）有限公司 一种核酸构建体

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JP7228523B2 (ja) 2023-02-24
EP3600448A1 (en) 2020-02-05
JP2023033551A (ja) 2023-03-10
IL269551A (en) 2019-11-28
SG11201908844YA (en) 2019-10-30
CA3057862A1 (en) 2018-10-04
AU2018243293A1 (en) 2019-10-31
US20220259594A1 (en) 2022-08-18
EP3600448A4 (en) 2021-01-27
WO2018183692A1 (en) 2018-10-04
BR112019020322A2 (pt) 2020-04-28
JP2020511991A (ja) 2020-04-23
WO2018183692A8 (en) 2019-05-02

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