JP6143231B2 - 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター - Google Patents

副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター Download PDF

Info

Publication number
JP6143231B2
JP6143231B2 JP2014514899A JP2014514899A JP6143231B2 JP 6143231 B2 JP6143231 B2 JP 6143231B2 JP 2014514899 A JP2014514899 A JP 2014514899A JP 2014514899 A JP2014514899 A JP 2014514899A JP 6143231 B2 JP6143231 B2 JP 6143231B2
Authority
JP
Japan
Prior art keywords
vector
cells
ltr
promoter
virus
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.)
Expired - Fee Related
Application number
JP2014514899A
Other languages
English (en)
Japanese (ja)
Other versions
JP2014519335A5 (cg-RX-API-DMAC7.html
JP2014519335A (ja
Inventor
マリア ジョアン ディナーロ,
マリア ジョアン ディナーロ,
ミッチェル ハワード ファイナー,
ミッチェル ハワード ファイナー,
ガボール ベレス,
ガボール ベレス,
Original Assignee
ブルーバード バイオ, インコーポレイテッド
ブルーバード バイオ, インコーポレイテッド
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 ブルーバード バイオ, インコーポレイテッド, ブルーバード バイオ, インコーポレイテッド filed Critical ブルーバード バイオ, インコーポレイテッド
Publication of JP2014519335A publication Critical patent/JP2014519335A/ja
Publication of JP2014519335A5 publication Critical patent/JP2014519335A5/ja
Application granted granted Critical
Publication of JP6143231B2 publication Critical patent/JP6143231B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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)
JP2014514899A 2011-06-10 2012-06-08 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター Expired - Fee Related JP6143231B2 (ja)

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2015042141A Division JP2015107134A (ja) 2011-06-10 2015-03-04 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター

Publications (3)

Publication Number Publication Date
JP2014519335A JP2014519335A (ja) 2014-08-14
JP2014519335A5 JP2014519335A5 (cg-RX-API-DMAC7.html) 2014-12-11
JP6143231B2 true JP6143231B2 (ja) 2017-06-07

Family

ID=47296778

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2014514899A Expired - Fee Related JP6143231B2 (ja) 2011-06-10 2012-06-08 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター
JP2015042141A Pending JP2015107134A (ja) 2011-06-10 2015-03-04 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2015042141A Pending JP2015107134A (ja) 2011-06-10 2015-03-04 副腎脳白質ジストロフィおよび副腎脊髄神経障害のための遺伝子療法ベクター

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)

Families Citing this family (43)

* 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
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
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
PL3370705T3 (pl) * 2015-11-05 2022-08-01 The General Hospital Corporation Dooponowe dostarczanie sekwencji kwasów nukleinowych do kodowania abcd1 w leczeniu adrenomieloneuropatii
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
US20220323612A1 (en) * 2016-06-13 2022-10-13 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
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
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
MX2019006552A (es) * 2016-12-06 2019-10-15 Bluebird Bio Inc Terapia génica para mucopolisacaridosis de tipo i.
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
GB201706121D0 (en) * 2017-04-18 2017-05-31 Glaxosmithkline Ip Dev Ltd Stable cell lines for retroviral production
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
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
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
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
CA3105953A1 (en) 2018-07-09 2020-01-16 Flagship Pioneering Innovations V, Inc. Fusosome compositions and uses thereof
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
EP3880179A2 (en) * 2018-11-14 2021-09-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for cns delivery
KR102163889B1 (ko) * 2019-01-11 2020-10-12 동의대학교 산학협력단 해죽순 추출물을 포함하는 신경병증성 통증 예방 및 치료용 조성물
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
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

Family Cites Families (31)

* 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
US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
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

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
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
CN103717240A (zh) 2014-04-09
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
US20150216903A1 (en) Compounds for improved viral transduction
US20220323612A1 (en) Gene therapy of neuronal ceroid lipofuscinoses
US20200071721A1 (en) Gene therapy for mucopolysaccharidosis, type ii
JP2019536484A (ja) ムコ多糖症i型のための遺伝子治療
AU2014256388A1 (en) Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20141021

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20141021

A871 Explanation of circumstances concerning accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A871

Effective date: 20141021

A975 Report on accelerated examination

Free format text: JAPANESE INTERMEDIATE CODE: A971005

Effective date: 20141114

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20141204

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20150304

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20150601

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20151001

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20151014

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20151113

A912 Re-examination (zenchi) completed and case transferred to appeal board

Free format text: JAPANESE INTERMEDIATE CODE: A912

Effective date: 20151225

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20170324

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20170428

R150 Certificate of patent or registration of utility model

Ref document number: 6143231

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

S531 Written request for registration of change of domicile

Free format text: JAPANESE INTERMEDIATE CODE: R313531

R154 Certificate of patent or utility model (reissue)

Free format text: JAPANESE INTERMEDIATE CODE: R154

R150 Certificate of patent or registration of utility model

Ref document number: 6143231

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R350 Written notification of registration of transfer

Free format text: JAPANESE INTERMEDIATE CODE: R350

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

LAPS Cancellation because of no payment of annual fees