CN103717240A - 用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体 - Google Patents

用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体 Download PDF

Info

Publication number
CN103717240A
CN103717240A CN201280038832.1A CN201280038832A CN103717240A CN 103717240 A CN103717240 A CN 103717240A CN 201280038832 A CN201280038832 A CN 201280038832A CN 103717240 A CN103717240 A CN 103717240A
Authority
CN
China
Prior art keywords
vector
cells
ltr
promoter
cell
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN201280038832.1A
Other languages
English (en)
Chinese (zh)
Inventor
玛利亚·琼恩·德纳罗
米切尔·霍华德·菲尼尔
加博尔·沃莱斯
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Bluebird Bio Inc
Original Assignee
Bluebird Bio Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Bluebird Bio Inc filed Critical Bluebird Bio Inc
Publication of CN103717240A publication Critical patent/CN103717240A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63—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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/867—Retroviral vectors
    • 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
    • A61K35/48—Reproductive organs
    • A61K35/51—Umbilical cord; Umbilical cord blood; Umbilical stem cells
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63—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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0603—Embryonic cells ; Embryoid bodies
    • C12N5/0606—Pluripotent embryonic cells, e.g. embryonic stem cells [ES]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0607—Non-embryonic pluripotent stem cells, e.g. MASC
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0684—Cells of the urinary tract or kidneys
    • C12N5/0686—Kidney cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/24—Vectors characterised by the absence of particular element, e.g. selectable marker, viral origin of replication
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/60—Vector systems having a special element relevant for transcription from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Urology & Nephrology (AREA)
  • Reproductive Health (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Communicable Diseases (AREA)
CN201280038832.1A 2011-06-10 2012-06-08 用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体 Pending CN103717240A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161495857P 2011-06-10 2011-06-10
US61/495,857 2011-06-10
PCT/US2012/041693 WO2012170911A2 (en) 2011-06-10 2012-06-08 Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy

Publications (1)

Publication Number Publication Date
CN103717240A true CN103717240A (zh) 2014-04-09

Family

ID=47296778

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201280038832.1A Pending CN103717240A (zh) 2011-06-10 2012-06-08 用于肾上腺脑白质营养不良症和肾上腺脊髓神经病的基因治疗载体

Country Status (16)

Country Link
US (3) US8858928B2 (cg-RX-API-DMAC7.html)
EP (2) EP3492108A1 (cg-RX-API-DMAC7.html)
JP (2) JP6143231B2 (cg-RX-API-DMAC7.html)
KR (2) KR20150029756A (cg-RX-API-DMAC7.html)
CN (1) CN103717240A (cg-RX-API-DMAC7.html)
AU (1) AU2012267512B2 (cg-RX-API-DMAC7.html)
CA (1) CA2838749A1 (cg-RX-API-DMAC7.html)
DK (1) DK2717922T3 (cg-RX-API-DMAC7.html)
ES (1) ES2710452T3 (cg-RX-API-DMAC7.html)
MX (1) MX2013014544A (cg-RX-API-DMAC7.html)
PL (1) PL2717922T3 (cg-RX-API-DMAC7.html)
PT (1) PT2717922T (cg-RX-API-DMAC7.html)
RU (1) RU2014100160A (cg-RX-API-DMAC7.html)
TR (1) TR201819015T4 (cg-RX-API-DMAC7.html)
WO (1) WO2012170911A2 (cg-RX-API-DMAC7.html)
ZA (1) ZA201309515B (cg-RX-API-DMAC7.html)

Cited By (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104857014A (zh) * 2015-04-21 2015-08-26 武汉大学 2-羟丙基-β-环化糊精在制备治疗X-连锁肾上腺脑白质退化症的药物中的应用
CN108456697A (zh) * 2018-03-29 2018-08-28 成都优娃生物科技有限公司 应用慢病毒载体EF1α启动子优化表达ABCD1基因治疗肾上腺脑白质营养不良症
CN108472264A (zh) * 2015-11-05 2018-08-31 通用医疗公司 编码用于治疗肾上腺脊髓神经病的abcd1的核酸序列的鞘内递送
CN109475619A (zh) * 2016-06-13 2019-03-15 蓝鸟生物公司 神经元蜡样脂褐质沉积症的基因治疗
CN110199028A (zh) * 2016-12-06 2019-09-03 蓝鸟生物公司 用于ii型黏多糖贮积症的基因治疗
CN110214182A (zh) * 2016-12-06 2019-09-06 蓝鸟生物公司 用于i型黏多糖贮积症的基因治疗
CN110520535A (zh) * 2017-04-18 2019-11-29 葛兰素史克知识产权开发有限公司 用于逆转录病毒产生的稳定细胞系
CN112367973A (zh) * 2018-05-15 2021-02-12 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
CN112955174A (zh) * 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
CN113613633A (zh) * 2018-11-14 2021-11-05 旗舰先锋创新V股份有限公司 用于cns递送的融合剂脂质体组合物
CN116096904A (zh) * 2019-12-31 2023-05-09 天鹅生物疗法有限公司 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2717922T3 (en) 2011-06-10 2019-01-07 Bluebird Bio Inc GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE
ES2708948T3 (es) 2012-11-27 2019-04-12 Childrens Medical Ct Corp Elementos reguladores distales de bcl11a como dianas para la reinducción de la hemoglobina fetal
GB201318804D0 (en) † 2013-10-24 2013-12-11 Adaptimmune Ltd Vectors for transgene expression
CN106794260B (zh) 2014-04-25 2021-02-23 儿童医疗中心有限公司 治疗血红蛋白病的组合物和方法
WO2015164759A2 (en) * 2014-04-25 2015-10-29 Bluebird Bio, Inc. Mnd promoter chimeric antigen receptors
CN106456670B (zh) 2014-04-25 2020-01-07 蓝鸟生物公司 制备过继性细胞疗法的改善方法
RS61406B1 (sr) 2014-06-06 2021-03-31 Bluebird Bio Inc Poboljšane kompozicije t ćelija
EP3172231B1 (en) 2014-07-24 2021-05-05 Bluebird Bio, Inc. Bcma chimeric antigen receptors
US10383929B2 (en) 2014-12-12 2019-08-20 Bluebird Bio, Inc. BCMA chimeric antigen receptors
EP3270960A4 (en) * 2015-03-20 2018-08-08 Bluebird Bio, Inc. Vector formulations
EP3763814A1 (en) 2015-05-08 2021-01-13 The Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
IL246634B (en) * 2015-07-23 2020-08-31 Brett Buno A method for creating a high-titer hepatitis e virus stock and detecting calibration for hepatitis e virus
WO2017099712A1 (en) 2015-12-07 2017-06-15 Bluebird Bio, Inc. Improved t cell compositions
CN108883136A (zh) 2016-02-12 2018-11-23 蓝鸟生物公司 Vcn增强子组合物及其使用方法
KR20250134722A (ko) 2016-02-12 2025-09-11 블루버드 바이오, 인코포레이티드. Vcn 인핸서 조성물 및 이의 사용 방법
JP7215905B2 (ja) * 2016-03-18 2023-01-31 プレシゲン，インコーポレイテッド Ｖｉｉ型コラーゲン欠損症の治療のための組成物及び方法
US20190345450A1 (en) * 2016-06-17 2019-11-14 Fred Hutchinson Cancer Research Center Strategies to assess and/or produce cell populations with predictive engraftment potential
MX2019005277A (es) 2016-11-04 2019-09-27 Bluebird Bio Inc Composiciones de celulas t con car anti-bcma.
US20180193270A1 (en) 2016-11-29 2018-07-12 PureTech Health LLC Exosomes for delivery of therapeutic agents
SG11201908844YA (en) 2017-03-29 2019-10-30 Bluebird Bio Inc Vectors and compositions for treating hemoglobinopathies
US11261441B2 (en) * 2017-03-29 2022-03-01 Bluebird Bio, Inc. Vectors and compositions for treating hemoglobinopathies
US11788087B2 (en) 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery
WO2019070740A2 (en) 2017-10-02 2019-04-11 Fred Hutchinson Cancer Research Center LUTENING HORMONE RECEPTOR BINDING AGENTS AND LUTINISING HORMONE AGONISTS FOR IDENTIFICATION, MULTIPLICATION, REMOVAL AND MODIFICATION OF PRIMITIVE STEM CELL POPULATIONS
US12522811B2 (en) 2018-05-01 2026-01-13 The Children's Medical Center Corporation Enhanced BCL11A RNP / CRISPR delivery and editing using a 3XNLS-CAS9
US12350284B2 (en) 2018-05-02 2025-07-08 The Children's Medical Center Corporation BCL11A microRNAs for treating hemoglobinopathies
MY204234A (en) 2018-08-27 2024-08-16 Regeneron Pharma Use of raman spectroscopy in downstream purification
KR20210131991A (ko) 2018-11-14 2021-11-03 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 구획-특이적 카고 전달을 위한 조성물 및 방법
KR102163889B1 (ko) * 2019-01-11 2020-10-12 동의대학교 산학협력단 해죽순 추출물을 포함하는 신경병증성 통증 예방 및 치료용 조성물
JP2024532963A (ja) 2021-04-08 2024-09-11 サナ バイオテクノロジー，インコーポレイテッド Ｃｄ８特異的抗体コンストラクト及びその組成物
CN115838765A (zh) * 2021-09-18 2023-03-24 济南赛尔生物科技股份有限公司 一种治疗异染性脑白质营养不良的慢病毒载体
CN114478713B (zh) * 2022-02-21 2023-03-28 江苏蒙彼利生物科技有限公司 一种cmv包膜蛋白包装慢病毒载体及其应用
CN121620592A (zh) * 2023-05-01 2026-03-06 西雅图儿童医院d/b/a西雅图儿童研究所 表达突变体白细胞介素15的免疫细胞

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999014310A1 (en) * 1997-09-18 1999-03-25 The Salk Institute For Biological Studies Rna export element and methods of use

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
US5061620A (en) 1990-03-30 1991-10-29 Systemix, Inc. Human hematopoietic stem cell
US5466468A (en) 1990-04-03 1995-11-14 Ciba-Geigy Corporation Parenterally administrable liposome formulation comprising synthetic lipids
US5635387A (en) 1990-04-23 1997-06-03 Cellpro, Inc. Methods and device for culturing human hematopoietic cells and their precursors
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
US5460964A (en) 1992-04-03 1995-10-24 Regents Of The University Of Minnesota Method for culturing hematopoietic cells
ATE155681T1 (de) 1992-05-18 1997-08-15 Minnesota Mining & Mfg Einrichtung zur transmucosalen wirkstoffabgabe
ES2249760T3 (es) * 1993-01-26 2006-04-01 The Trustees Of The University Of Pennsylvania (A Corporation Of Pennsylvania) Composiciones y procedimientos de administracion de materias geneticas.
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5409813A (en) 1993-09-30 1995-04-25 Systemix, Inc. Method for mammalian cell separation from a mixture of cell populations
US5658784A (en) * 1994-04-14 1997-08-19 Dana-Farber Cancer Institute, Inc. Nucleic acid encoding transcription factor p300 and uses of p300
US5677136A (en) 1994-11-14 1997-10-14 Systemix, Inc. Methods of obtaining compositions enriched for hematopoietic stem cells, compositions derived therefrom and methods of use thereof
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
FR2765578B1 (fr) 1997-07-03 1999-09-10 Adir Procede de preparation d'un perhydroisoindole substitue
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
FR2777909B1 (fr) 1998-04-24 2002-08-02 Pasteur Institut Utilisation de sequences d'adn de structure triplex pour le tranfert de sequences de nucleotides dans des cellules, vecteurs recombinants contenant ces sequences triplex
FR2778670B1 (fr) * 1998-05-18 2002-12-13 Rhone Poulenc Rorer Sa Methodes et compositions pour la production de particules virales
US20030039636A1 (en) 2001-05-01 2003-02-27 Genetix Pharmaceuticals, Inc. Novel self-inactivating (SIN) lentiviral vectors
WO2002088346A2 (en) 2001-05-01 2002-11-07 National Research Council Of Canada A system for inducible expression in eukaryotic cells
ES2348868T3 (es) 2002-12-13 2010-12-16 Genetix Pharmaceuticals Inc. Vectores retrovirales terapeuticos para terapia genica.
EP2573185A3 (en) * 2005-02-16 2013-06-05 Lentigen Corporation Lentiviral vectors and their use
JP2007054069A (ja) 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
EP1835032A1 (en) * 2006-03-14 2007-09-19 Université de Liège A self-inactivating recombinant lentiviral vector for the inhibition of HIV replication
WO2008134878A1 (en) * 2007-05-04 2008-11-13 University Health Network Compositions and methods for cancer treatment
EP2020444B1 (en) 2007-08-03 2017-05-31 Institut Pasteur Defective non-integrative lentiviral transfer vectors for vaccines
CN102438658A (zh) 2009-03-13 2012-05-02 莱蒂恩公司 非整合型逆转录病毒载体疫苗
DK2717922T3 (en) 2011-06-10 2019-01-07 Bluebird Bio Inc GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999014310A1 (en) * 1997-09-18 1999-03-25 The Salk Institute For Biological Studies Rna export element and methods of use

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
ALESSANDRA BIFFI ET AL: "Gene therapy for leukodystrophies", 《HUMAN MOLECULAR GENETICS》, 31 March 2011 (2011-03-31) *
NATHALIE CARTIER ET AL: "Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy", 《SCIENCE》, 31 December 2009 (2009-12-31) *
STEPHANIE BALCAITIS ET AL: "Lentiviral Transduction of Microglial Cells", 《GLIA》, 31 December 2005 (2005-12-31), pages 48 - 55 *
姚德生 等: "携带OPCML基因的慢病毒表达质粒构建", 《现代妇产科进展》, 31 July 2006 (2006-07-31), pages 520 - 1 *
孙克宁 等: "慢病毒载体的构建及其应用于转基因动物的研究进展", 《遗传繁育》, 31 December 2010 (2010-12-31), pages 116 - 120 *

Cited By (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104857014B (zh) * 2015-04-21 2017-12-08 武汉大学 2‑羟丙基‑β‑环化糊精在制备治疗X‑连锁肾上腺脑白质退化症的药物中的应用
CN104857014A (zh) * 2015-04-21 2015-08-26 武汉大学 2-羟丙基-β-环化糊精在制备治疗X-连锁肾上腺脑白质退化症的药物中的应用
CN108472264A (zh) * 2015-11-05 2018-08-31 通用医疗公司 编码用于治疗肾上腺脊髓神经病的abcd1的核酸序列的鞘内递送
CN109475619A (zh) * 2016-06-13 2019-03-15 蓝鸟生物公司 神经元蜡样脂褐质沉积症的基因治疗
CN110214182A (zh) * 2016-12-06 2019-09-06 蓝鸟生物公司 用于i型黏多糖贮积症的基因治疗
CN110199028A (zh) * 2016-12-06 2019-09-03 蓝鸟生物公司 用于ii型黏多糖贮积症的基因治疗
CN110520535A (zh) * 2017-04-18 2019-11-29 葛兰素史克知识产权开发有限公司 用于逆转录病毒产生的稳定细胞系
CN110520535B (zh) * 2017-04-18 2024-01-09 葛兰素史克知识产权开发有限公司 用于逆转录病毒产生的稳定细胞系
WO2019185067A1 (zh) * 2018-03-29 2019-10-03 成都优娃生物科技有限公司 应用慢病毒载体EF1α启动子优化表达ABCD1基因治疗肾上腺脑白质营养不良症
CN108456697A (zh) * 2018-03-29 2018-08-28 成都优娃生物科技有限公司 应用慢病毒载体EF1α启动子优化表达ABCD1基因治疗肾上腺脑白质营养不良症
CN108456697B (zh) * 2018-03-29 2022-05-27 成都优娃生物科技有限公司 应用慢病毒载体EF1α启动子优化表达ABCD1基因治疗肾上腺脑白质营养不良症
US12065662B2 (en) 2018-03-29 2024-08-20 Chengdu Youwa Biotechnology Co., Ltd. Application of lentiviral vector EF1α promoter for optimising ABCD1 gene expression to treat adrenoleukodystrophy
CN112367973A (zh) * 2018-05-15 2021-02-12 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
CN112955174A (zh) * 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
US12378578B2 (en) 2018-07-09 2025-08-05 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
CN113613633A (zh) * 2018-11-14 2021-11-05 旗舰先锋创新V股份有限公司 用于cns递送的融合剂脂质体组合物
CN116096904A (zh) * 2019-12-31 2023-05-09 天鹅生物疗法有限公司 改进的aav-abcd1构建体和用于治疗或预防肾上腺脑白质营养不良(ald)和/或肾上腺脊髓神经病(amn)的用途

Also Published As

Publication number Publication date
EP3492108A1 (en) 2019-06-05
DK2717922T3 (en) 2019-01-07
MX2013014544A (es) 2014-04-30
KR20140039035A (ko) 2014-03-31
WO2012170911A2 (en) 2012-12-13
US9061031B2 (en) 2015-06-23
EP2717922B1 (en) 2018-09-12
PL2717922T3 (pl) 2019-03-29
JP6143231B2 (ja) 2017-06-07
ZA201309515B (en) 2016-01-27
RU2014100160A (ru) 2015-07-20
US20130004471A1 (en) 2013-01-03
ES2710452T3 (es) 2019-04-25
KR20150029756A (ko) 2015-03-18
JP2015107134A (ja) 2015-06-11
US20150037296A1 (en) 2015-02-05
EP2717922A4 (en) 2015-01-07
CA2838749A1 (en) 2012-12-13
TR201819015T4 (tr) 2019-01-21
AU2012267512B2 (en) 2014-11-20
EP2717922A2 (en) 2014-04-16
US9789139B2 (en) 2017-10-17
US8858928B2 (en) 2014-10-14
JP2014519335A (ja) 2014-08-14
KR101528440B1 (ko) 2015-06-26
US20150064150A1 (en) 2015-03-05
WO2012170911A3 (en) 2013-04-11
PT2717922T (pt) 2018-12-18

Similar Documents

Publication Publication Date Title
US9061031B2 (en) Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
US10280435B2 (en) Gene therapy methods
CN108220246B (zh) 用于改善病毒转导的化合物
US20150216903A1 (en) Compounds for improved viral transduction
US20200071721A1 (en) Gene therapy for mucopolysaccharidosis, type ii
AU2014256388A1 (en) Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
C12 Rejection of a patent application after its publication
RJ01 Rejection of invention patent application after publication

Application publication date: 20140409