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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English (en)
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
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    • 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
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    • C12N2740/10011—Retroviridae
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    • 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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    • C12N2740/16071—Demonstrated in vivo effect
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    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
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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 (https=)
EP (1) EP3600448A4 (https=)
JP (2) JP7228523B2 (https=)
CN (1) CN110582305A (https=)
AU (1) AU2018243293A1 (https=)
BR (1) BR112019020322A2 (https=)
CA (1) CA3057862A1 (https=)
IL (1) IL269551A (https=)
SG (1) SG11201908844YA (https=)
WO (1) WO2018183692A1 (https=)

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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US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
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
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
EP3980549A4 (en) * 2019-06-05 2023-07-26 Orchard Therapeutics (Europe) Limited COMPOSITIONS AND METHODS FOR MODIFYING EUKARYOT CELLS
KR20220097891A (ko) * 2019-09-30 2022-07-08 바이오버라티브 테라퓨틱스 인크. 렌티바이러스 벡터 제형
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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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

Family Cites Families (9)

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US8383604B2 (en) * 2008-09-15 2013-02-26 Children's Medical Center Corporation Modulation of BCL11A for treatment of hemoglobinopathies
WO2014093965A1 (en) * 2012-12-14 2014-06-19 Case Western Reserve University Genomic rna packaging enhancer element
PL3134432T3 (pl) * 2014-04-25 2020-10-19 Bluebird Bio, Inc. Promotor mnd chimeryczne receptory antygenowe
US20170049819A1 (en) * 2014-04-25 2017-02-23 Bluebird Bio, Inc. Kappa/lambda chimeric antigen receptors
EP3620521B1 (en) 2014-09-04 2024-05-29 Memorial Sloan-Kettering Cancer Center Globin gene therapy for treating hemoglobinopathies
LT3277807T (lt) 2015-03-31 2020-01-10 Glycotope Gmbh Eukariotiniai ekspresijos vektoriai, apimantys globino genų klasterių reguliavimo elementus
WO2017040529A1 (en) 2015-08-31 2017-03-09 Bluebird Bio, Inc. Anti-sialyl tn chimeric antigen receptors
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
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

Also Published As

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

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