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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JPWO2022099007A5 (https=
JP2023549124A5 (https=
Inventor
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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    • 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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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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)

Applications Claiming Priority (5)

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
JPWO2022099007A5 JPWO2022099007A5 (https=) 2024-10-31
JP2023549124A5 JP2023549124A5 (https=) 2024-10-31

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

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JP7807380B2 (ja) 2020-02-21 2026-01-27 アコーオス インコーポレイテッド ヒト対象において非加齢関連聴力障害を処置するための組成物および方法
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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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
US12054724B2 (en) * 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
CN118497276A (zh) * 2018-11-07 2024-08-16 阿库斯股份有限公司 腺相关病毒载体用于在内耳中的毛细胞和支持细胞中校正基因缺陷/表达蛋白质的用途
CA3153133A1 (en) * 2019-09-30 2021-04-08 Chris Bartolome Adeno-associated virus (aav) systems for treatment of genetic hearing loss

Patent Citations (2)

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

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