JP7623299B2 - 乳児性悪性大理石骨病に対する遺伝子療法ベクター - Google Patents

乳児性悪性大理石骨病に対する遺伝子療法ベクター Download PDF

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JP7623299B2
JP7623299B2 JP2021569298A JP2021569298A JP7623299B2 JP 7623299 B2 JP7623299 B2 JP 7623299B2 JP 2021569298 A JP2021569298 A JP 2021569298A JP 2021569298 A JP2021569298 A JP 2021569298A JP 7623299 B2 JP7623299 B2 JP 7623299B2
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ブライアン ビアード
デイビッド リックス
ラジ プラバカール
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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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C—CHEMISTRY; METALLURGY
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    • 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
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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
    • C12N15/63—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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0387—Animal model for diseases of the immune system
    • 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

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • 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)
JP2021569298A 2019-05-23 2020-05-22 乳児性悪性大理石骨病に対する遺伝子療法ベクター Active JP7623299B2 (ja)

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JP2025005848A JP2025063200A (ja) 2019-05-23 2025-01-16 乳児性悪性大理石骨病に対する遺伝子療法ベクター

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US201962852216P 2019-05-23 2019-05-23
US62/852,216 2019-05-23
PCT/US2020/034394 WO2020237219A1 (en) 2019-05-23 2020-05-22 Gene therapy vectors for infantile malignant osteopetrosis

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JP2022532802A JP2022532802A (ja) 2022-07-19
JP2022532802A5 JP2022532802A5 (https=) 2023-05-25
JP7623299B2 true JP7623299B2 (ja) 2025-01-28

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JP2025005848A Pending JP2025063200A (ja) 2019-05-23 2025-01-16 乳児性悪性大理石骨病に対する遺伝子療法ベクター

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US (1) US20220218844A1 (https=)
EP (1) EP3973066A4 (https=)
JP (2) JP7623299B2 (https=)
KR (1) KR20220012266A (https=)
CN (1) CN113994008A (https=)
AU (1) AU2020278046A1 (https=)
BR (1) BR112021023473A2 (https=)
CA (1) CA3137700A1 (https=)
IL (1) IL288193A (https=)
MX (1) MX2021014337A (https=)
SG (1) SG11202112347RA (https=)
WO (1) WO2020237219A1 (https=)

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HUE055862T2 (hu) 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
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
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
CN116042719B (zh) * 2022-12-28 2025-11-11 蓝图生物医药(广州)有限公司 一种重组腺相关病毒载体及其应用

Citations (2)

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JP2002508184A (ja) 1997-12-12 2002-03-19 セル ジェネシス，インコーポレイティド 高力価で安全な組換えレンチウイルスベクター作成の方法及び手段
WO2018049273A1 (en) 2016-09-08 2018-03-15 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Gene therapy for patients with fanconi anemia

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GB0202771D0 (en) * 2002-02-06 2002-03-27 Univ Aberdeen Genetic markers for bone mass
CA2650507A1 (en) * 2006-04-27 2007-11-08 Universite De Montreal Assessment and reduction of risk of graft-versus-host disease
AU2013309488A1 (en) * 2012-08-29 2015-03-05 Nature Technology Corporation DNA plasmids with improved expression
WO2014077863A1 (en) * 2012-11-19 2014-05-22 Nature Technology Corporation Replicative minicircle vectors with improved expression
MX374532B (es) * 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
CN116064678A (zh) * 2016-01-15 2023-05-05 美国基因技术国际有限公司 用于活化γ-δT细胞的方法和组合物
CN121102467A (zh) * 2016-04-22 2025-12-12 克莱格医学有限公司 用于细胞免疫疗法的组合物和方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002508184A (ja) 1997-12-12 2002-03-19 セル ジェネシス，インコーポレイティド 高力価で安全な組換えレンチウイルスベクター作成の方法及び手段
WO2018049273A1 (en) 2016-09-08 2018-03-15 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Gene therapy for patients with fanconi anemia

Non-Patent Citations (2)

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Title
LUKE, J., et al.，Improved antibiotic-free DNA vaccine vectors utilizing a novel RNA based plasmid selection system.，Vaccine，2009年，Vol.27, No.46，pp.6454-6459
MOSCATELLI, I., et al.，Targeting NSG mice engrafting cells with a clinically applicable lentiviral vector corrects osteoclasts in infantile malignant osteopetrosis.，Human Gene Therapy，2017年，pp.1-12, Supplementary Data

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EP3973066A1 (en) 2022-03-30
SG11202112347RA (en) 2021-12-30
MX2021014337A (es) 2022-03-17
EP3973066A4 (en) 2023-07-26
JP2025063200A (ja) 2025-04-15
WO2020237219A1 (en) 2020-11-26
KR20220012266A (ko) 2022-02-03
JP2022532802A (ja) 2022-07-19
CA3137700A1 (en) 2020-11-26
CN113994008A (zh) 2022-01-28
AU2020278046A1 (en) 2022-01-27
US20220218844A1 (en) 2022-07-14
IL288193A (en) 2022-01-01
BR112021023473A2 (pt) 2022-02-01

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