JP2023540756A - 向上した形質導入効率及び脳内における広範な分布を有する組換えアデノ随伴ウイルスベクターの新規の操作されたキャプシド血清型 - Google Patents

向上した形質導入効率及び脳内における広範な分布を有する組換えアデノ随伴ウイルスベクターの新規の操作されたキャプシド血清型 Download PDF

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JP2023540756A
JP2023540756A JP2023514976A JP2023514976A JP2023540756A JP 2023540756 A JP2023540756 A JP 2023540756A JP 2023514976 A JP2023514976 A JP 2023514976A JP 2023514976 A JP2023514976 A JP 2023514976A JP 2023540756 A JP2023540756 A JP 2023540756A
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growth factor
gene
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brain
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JPWO2022051633A5 (https=
JP2023540756A5 (https=
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ツァオ，レイ
ホアン，ウェイ
バンキエヴィチ，クシシュトフ
ハダチェク，ピョートル
サマランチ，ルイス
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オハイオ・ステイト・イノベーション・ファウンデーション
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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
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K38/18—Growth factors; Growth regulators
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    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1825—Fibroblast growth factor [FGF]
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    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1841—Transforming growth factor [TGF]
    • A—HUMAN NECESSITIES
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    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1858—Platelet-derived growth factor [PDGF]
    • A—HUMAN NECESSITIES
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    • A61K38/00—Medicinal preparations containing peptides
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1858—Platelet-derived growth factor [PDGF]
    • A61K38/1866—Vascular endothelial growth factor [VEGF]
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1875—Bone morphogenic factor; Osteogenins; Osteogenic factor; Bone-inducing factor
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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/20—Interleukins [IL]
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    • A61K38/22—Hormones
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    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
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JP2023514976A 2020-09-04 2021-09-03 向上した形質導入効率及び脳内における広範な分布を有する組換えアデノ随伴ウイルスベクターの新規の操作されたキャプシド血清型 Pending JP2023540756A (ja)

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US202063074548P 2020-09-04 2020-09-04
US63/074,548 2020-09-04
PCT/US2021/049081 WO2022051633A2 (en) 2020-09-04 2021-09-03 Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain

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JP2023540756A true JP2023540756A (ja) 2023-09-26
JPWO2022051633A5 JPWO2022051633A5 (https=) 2024-09-11
JP2023540756A5 JP2023540756A5 (https=) 2024-09-11

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US (1) US20230330267A1 (https=)
EP (1) EP4208557A4 (https=)
JP (1) JP2023540756A (https=)
CN (1) CN116249541A (https=)
AU (1) AU2021335601A1 (https=)
CA (1) CA3191543A1 (https=)
WO (1) WO2022051633A2 (https=)

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US20240158861A1 (en) * 2021-04-23 2024-05-16 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and compositions for treating cell senescence accumulation related disease
WO2025059678A1 (en) * 2023-09-15 2025-03-20 Ohio State Innovation Foundation Novel engineered capsid serotypes of recombinant adeno-associated viral vectors with reduced liver tropism

Citations (3)

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Publication number Priority date Publication date Assignee Title
JP2017518271A (ja) * 2014-05-02 2017-07-06 ジェンザイム・コーポレーション 網膜およびｃｎｓ遺伝子治療用ａａｖベクター
WO2019040586A1 (en) * 2017-08-25 2019-02-28 Ovid Therapeutics Inc. RECOMBINANT ADENO-ASSOCIATE VECTORS
WO2019222444A2 (en) * 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution

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EP1486567A1 (en) * 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
WO2009120978A2 (en) * 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor

Patent Citations (3)

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Publication number Priority date Publication date Assignee Title
JP2017518271A (ja) * 2014-05-02 2017-07-06 ジェンザイム・コーポレーション 網膜およびｃｎｓ遺伝子治療用ａａｖベクター
WO2019040586A1 (en) * 2017-08-25 2019-02-28 Ovid Therapeutics Inc. RECOMBINANT ADENO-ASSOCIATE VECTORS
WO2019222444A2 (en) * 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution

Non-Patent Citations (3)

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Title
JASON J. SIU、他: "Improved Gene Delivery to Adult Mouse Spinal Cord Through the Use of Engineered Hybrid Adeno-Associa", GENE THER., vol. 24(6), JPN7025003947, June 2017 (2017-06-01), pages 361 - 369, ISSN: 0005770443 *
JASON J. SIU: "Hypothalamic Gene Therapy by an Autoregulatory BDNF Vector to Prevent Melanocortin-4-Receptor-Defici", DISSERTATION; 2018, DOCTOR OF PHILOSOPHY, OHIO STATE UNIVERSITY, NEUROSCIENCE GRADUATE STUDIES PROGR, JPN7025003945, 2018, ISSN: 0005770441 *
P. C. ISSA、他: "Assessment of Tropism and Effectiveness of New Primate-Derived Hybrid Recombinant AAV Serotypes in t", PLOS ONE, vol. 8(4), JPN7025003946, 9 April 2013 (2013-04-09), pages 60361, ISSN: 0005770442 *

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WO2022051633A3 (en) 2022-04-14
CN116249541A (zh) 2023-06-09
WO2022051633A2 (en) 2022-03-10
AU2021335601A1 (en) 2023-04-06
EP4208557A4 (en) 2025-05-14
CA3191543A1 (en) 2022-03-10
EP4208557A2 (en) 2023-07-12
US20230330267A1 (en) 2023-10-19

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