JP2023549124A - 難聴の治療のためのバリアントアデノ随伴ウイルス（ａａｖ）カプシドポリペプチド、及びその遺伝子治療薬 - Google Patents

難聴の治療のためのバリアントアデノ随伴ウイルス（ａａｖ）カプシドポリペプチド、及びその遺伝子治療薬 Download PDF

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JP2023549124A
JP2023549124A JP2023527351A JP2023527351A JP2023549124A JP 2023549124 A JP2023549124 A JP 2023549124A JP 2023527351 A JP2023527351 A JP 2023527351A JP 2023527351 A JP2023527351 A JP 2023527351A JP 2023549124 A JP2023549124 A JP 2023549124A
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gjb2
cells
variant
acid sequence
aav
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JP2023549124A5 (enExample
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ペノック，スティーブン
ティマース，アドリアン，エム．
シアマン，マーク
バルトロメ，クリストファー
ワン，シャオボー
メイサー，プラナフ，ディネシュ
エム． ウリベ，フィリップ
スゾボタ，ステファニー
ジャックス，ボニー，イー．
フォスター，アラン，シー．
ピウ，ファブリス
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イーライ リリー アンド カンパニー
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • 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
    • 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
    • A61K48/0008—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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    • 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
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    • 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
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    • 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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    • 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
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • 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

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JP2023527351A 2020-11-06 2021-11-05 難聴の治療のためのバリアントアデノ随伴ウイルス（ａａｖ）カプシドポリペプチド、及びその遺伝子治療薬 Pending JP2023549124A (ja)

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Application Number Priority Date Filing Date Title
US202063110697P 2020-11-06 2020-11-06
US63/110,697 2020-11-06
US202163146269P 2021-02-05 2021-02-05
US63/146,269 2021-02-05
PCT/US2021/058255 WO2022099007A1 (en) 2020-11-06 2021-11-05 Variant adeno-associated virus (aav)capsid polypeptides and gene therapeutics thereof for treatment of hearing loss

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JP2023549124A true JP2023549124A (ja) 2023-11-22
JP2023549124A5 JP2023549124A5 (enExample) 2024-10-31

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US (1) US20240226330A9 (enExample)
JP (1) JP2023549124A (enExample)
KR (1) KR20230117731A (enExample)
AU (1) AU2021376225A1 (enExample)
CA (1) CA3197592A1 (enExample)
IL (1) IL302653A (enExample)
MX (1) MX2023005344A (enExample)
WO (1) WO2022099007A1 (enExample)

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KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
WO2024164318A1 (zh) * 2023-02-10 2024-08-15 苏州星奥拓维生物技术有限公司 Gch1抑制剂在制备用于预防和治疗听力损伤的药物中的用途
CN116925239B (zh) * 2023-07-17 2024-10-18 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法

Citations (2)

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Publication number Priority date Publication date Assignee Title
CN111349148A (zh) * 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 一种腺相关病毒载体及其用途
WO2020148458A1 (en) * 2019-01-18 2020-07-23 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene

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US10294281B2 (en) * 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
WO2019200016A1 (en) * 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
EP4063510A1 (en) * 2018-11-07 2022-09-28 Akouos, Inc. Use of adeno-associated viral vectors to correct gene defects/ express proteins in hair cells and supporting cells in the inner ear
WO2021067448A1 (en) * 2019-09-30 2021-04-08 Applied Genetic Technologies Corporation Adeno-associated virus (aav) systems for treatment of genetic hearing loss

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020148458A1 (en) * 2019-01-18 2020-07-23 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
CN111349148A (zh) * 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 一种腺相关病毒载体及其用途

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
"Perinatal Gjb2 gene transfer rescues hearing in a mouse model of hereditary deafness", HUMAN MOLECULAR GENETICS, vol. pp. 1-11, JPN7025003350, 2015, ISSN: 0005651809 *
GENE THERAPY, vol. Vol.21, pp.71-80, JPN6025030670, 2014, ISSN: 0005651810 *

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US20240226330A9 (en) 2024-07-11
WO2022099007A1 (en) 2022-05-12
CA3197592A1 (en) 2022-05-12
IL302653A (en) 2023-07-01
US20240131189A1 (en) 2024-04-25
AU2021376225A1 (en) 2023-06-22
MX2023005344A (es) 2023-12-07
KR20230117731A (ko) 2023-08-09

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