JP2019517281A - 神経セロイドリポフスチン症の遺伝子治療 - Google Patents

神経セロイドリポフスチン症の遺伝子治療 Download PDF

Info

Publication number
JP2019517281A
JP2019517281A JP2019517200A JP2019517200A JP2019517281A JP 2019517281 A JP2019517281 A JP 2019517281A JP 2019517200 A JP2019517200 A JP 2019517200A JP 2019517200 A JP2019517200 A JP 2019517200A JP 2019517281 A JP2019517281 A JP 2019517281A
Authority
JP
Japan
Prior art keywords
cells
polynucleotide
promoter
cell
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.)
Pending
Application number
JP2019517200A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019517281A5 (enExample
Inventor
ケンドリック エー． ゴス，
ケンドリック エー． ゴス，
ジェフリー ビー． パーソンズ，
ジェフリー ビー． パーソンズ，
アシヤ ジニアトゥリナ，
アシヤ ジニアトゥリナ，
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 JP2019517281A publication Critical patent/JP2019517281A/ja
Publication of JP2019517281A5 publication Critical patent/JP2019517281A5/ja
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Communicable Diseases (AREA)
  • Neurosurgery (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Obesity (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (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)
JP2019517200A 2016-06-13 2017-06-13 神経セロイドリポフスチン症の遺伝子治療 Pending JP2019517281A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662349505P 2016-06-13 2016-06-13
US62/349,505 2016-06-13
US201762457498P 2017-02-10 2017-02-10
US62/457,498 2017-02-10
PCT/US2017/037230 WO2017218519A1 (en) 2016-06-13 2017-06-13 Gene therapy of neuronal ceroid lipofuscinoses

Publications (2)

Publication Number Publication Date
JP2019517281A true JP2019517281A (ja) 2019-06-24
JP2019517281A5 JP2019517281A5 (enExample) 2020-07-09

Family

ID=60663744

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019517200A Pending JP2019517281A (ja) 2016-06-13 2017-06-13 神経セロイドリポフスチン症の遺伝子治療

Country Status (7)

Country Link
US (1) US20220323612A1 (enExample)
EP (1) EP3468594A4 (enExample)
JP (1) JP2019517281A (enExample)
CN (1) CN109475619A (enExample)
AU (1) AU2017283474A1 (enExample)
CA (1) CA3058735A1 (enExample)
WO (1) WO2017218519A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
CA2935960C (en) 2014-01-08 2023-01-10 Bart Lipkens Acoustophoresis device with dual acoustophoretic chamber
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
CN109641063B (zh) 2016-04-20 2022-11-15 能源环境和技术研究中心O.A., M.P. 用于增强pklr的基因表达的组合物和方法
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
SG11202003907WA (en) 2017-12-14 2020-05-28 Flodesign Sonics Inc Acoustic transducer drive and controller
JP7576754B2 (ja) 2018-04-11 2024-11-01 スペースクラフト セブン リミテッド ライアビリティ カンパニー 幹細胞移植のための組成物および方法
MX2021001292A (es) * 2018-07-30 2021-07-15 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Metodos para la modificacion genica de celulas hematopoyeticas.
AU2020245415B2 (en) * 2019-03-22 2025-06-26 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal stoarge disorders
CN112522321B (zh) * 2020-12-16 2022-04-08 北京艺妙神州医药科技有限公司 一种基因治疗载体及其用途

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014519335A (ja) * 2011-06-10 2014-08-14 ブルーバード バイオ， インコーポレイテッド 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 Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014089449A1 (en) * 2012-12-07 2014-06-12 Rush University Nedical Center Composition and method for treating neuronal ceroid lipofuscinosis
WO2016028879A1 (en) * 2014-08-19 2016-02-25 The Children's Hospital Of Philadelphia Compositions and methods for modulating immune response

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014519335A (ja) * 2011-06-10 2014-08-14 ブルーバード バイオ， インコーポレイテッド 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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
GENE THER, 2003, VOL. 10, NO. 1, PP. 34-42, JPN6021024602, ISSN: 0004693512 *
PLOS ONE, 2012, VOL. 7, NO. 7, ARTICLE NO. E40509 (PP. 1-7), JPN6021024601, ISSN: 0004536363 *

Also Published As

Publication number Publication date
EP3468594A1 (en) 2019-04-17
CN109475619A (zh) 2019-03-15
WO2017218519A1 (en) 2017-12-21
AU2017283474A1 (en) 2019-01-03
CA3058735A1 (en) 2017-12-21
EP3468594A4 (en) 2020-01-22
US20220323612A1 (en) 2022-10-13

Similar Documents

Publication Publication Date Title
EP3414321B1 (en) Vcn enhancer compositions and methods of using the same
US20220323612A1 (en) Gene therapy of neuronal ceroid lipofuscinoses
AU2012267512B2 (en) Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
US20190184035A1 (en) Bcl11a homing endonuclease variants, compositions, and methods of use
US20220339296A1 (en) Gene therapy for mucopolysaccharidosis, type i
US20200071721A1 (en) Gene therapy for mucopolysaccharidosis, type ii
CA3164591A1 (en) Modification of blood type antigens
EP4255502A2 (en) Compositions and methods for the targeting of bcl11a
CN108883136A (zh) Vcn增强子组合物及其使用方法
JP2025016450A (ja) 人工トランス活性化因子による選択
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: 20200521

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200521

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210625

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20220128