CA3058735A1 - Gene therapy of neuronal ceroid lipofuscinoses - Google Patents

Gene therapy of neuronal ceroid lipofuscinoses Download PDF

Info

Publication number
CA3058735A1
CA3058735A1 CA3058735A CA3058735A CA3058735A1 CA 3058735 A1 CA3058735 A1 CA 3058735A1 CA 3058735 A CA3058735 A CA 3058735A CA 3058735 A CA3058735 A CA 3058735A CA 3058735 A1 CA3058735 A1 CA 3058735A1
Authority
CA
Canada
Prior art keywords
polynucleotide
cells
cell
promoter
tpp1
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.)
Abandoned
Application number
CA3058735A
Other languages
English (en)
French (fr)
Inventor
Kendrick A. GOSS
Geoffrey B. PARSONS
Asiya GINIATULLINA
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 CA3058735A1 publication Critical patent/CA3058735A1/en
Abandoned 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)
CA3058735A 2016-06-13 2017-06-13 Gene therapy of neuronal ceroid lipofuscinoses Abandoned CA3058735A1 (en)

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 (1)

Publication Number Publication Date
CA3058735A1 true CA3058735A1 (en) 2017-12-21

Family

ID=60663744

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3058735A Abandoned CA3058735A1 (en) 2016-06-13 2017-06-13 Gene therapy of neuronal ceroid lipofuscinoses

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

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2012267512B2 (en) * 2011-06-10 2014-11-20 Bluebird Bio, Inc. Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
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

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
EP3468594A4 (en) 2020-01-22
US20220323612A1 (en) 2022-10-13
JP2019517281A (ja) 2019-06-24

Similar Documents

Publication Publication Date Title
US20220323612A1 (en) Gene therapy of neuronal ceroid lipofuscinoses
AU2012267512B2 (en) Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
US20220339296A1 (en) Gene therapy for mucopolysaccharidosis, type i
EP3414321B1 (en) Vcn enhancer compositions and methods of using the same
US20190184035A1 (en) Bcl11a homing endonuclease variants, compositions, and methods of use
US20200071721A1 (en) Gene therapy for mucopolysaccharidosis, type ii
JP2021523724A (ja) フソソーム組成物およびその使用
AU2021207810A1 (en) Modification of blood type antigens
CA2849720A1 (en) Improved gene therapy methods
KR20230044420A (ko) 바이러스 푸소좀을 생산하기 위한 방법 및 조성물
JP2026081307A (ja) フソソーム組成物およびその使用
WO2002094989A2 (en) Retroviral vectors and methods of using same
AU2014256388A1 (en) Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy

Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20230912