JP2023542211A - 神経疾患を処置するための方法 - Google Patents

神経疾患を処置するための方法 Download PDF

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JP2023542211A
JP2023542211A JP2023518391A JP2023518391A JP2023542211A JP 2023542211 A JP2023542211 A JP 2023542211A JP 2023518391 A JP2023518391 A JP 2023518391A JP 2023518391 A JP2023518391 A JP 2023518391A JP 2023542211 A JP2023542211 A JP 2023542211A
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disease
composition
disorder
nucleic acid
sequence
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アンナ トレチャコヴァ,
レスター スアレス,
アン ブラーイ,
マイケル エル. ロバーツ,
フィリップ モリエ,
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アスクレピオス バイオファーマシューティカル, インコーポレイテッド
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12Q2600/00Oligonucleotides characterized by their use
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    • C12Y114/00Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/13Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen (1.14.13)
    • C12Y114/13098Cholesterol 24-hydroxylase (1.14.13.98)

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JP2023518391A 2020-09-21 2021-09-21 神経疾患を処置するための方法 Pending JP2023542211A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063080925P 2020-09-21 2020-09-21
US63/080,925 2020-09-21
US202063121152P 2020-12-03 2020-12-03
US63/121,152 2020-12-03
US202163139410P 2021-01-20 2021-01-20
US63/139,410 2021-01-20
US202163140440P 2021-01-22 2021-01-22
US63/140,440 2021-01-22
US202163180407P 2021-04-27 2021-04-27
US63/180,407 2021-04-27
PCT/US2021/071534 WO2022061378A2 (en) 2020-09-21 2021-09-21 Methods for treating neurological disease

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JP2023542211A true JP2023542211A (ja) 2023-10-05
JP2023542211A5 JP2023542211A5 (https=) 2024-08-22

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US (1) US20230365963A1 (https=)
EP (1) EP4213891A4 (https=)
JP (1) JP2023542211A (https=)
AU (1) AU2021344607A1 (https=)
CA (1) CA3193406A1 (https=)
IL (1) IL301525A (https=)
WO (1) WO2022061378A2 (https=)

Families Citing this family (6)

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Publication number Priority date Publication date Assignee Title
EP4314296A2 (en) 2021-03-29 2024-02-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
EP4423272A2 (en) * 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
JP2025503910A (ja) * 2022-01-27 2025-02-06 アスクレピオス バイオファーマシューティカル, インコーポレイテッド 神経疾患を処置するための組成物
WO2023212521A2 (en) * 2022-04-25 2023-11-02 Myrtelle Inc. Treatments of disorders of myelin
EP4601743A1 (en) * 2022-10-14 2025-08-20 Institut National de la Santé et de la Recherche Médicale Methods and pharmaceutical composition for the treatment of alpha-synucleinopathies
WO2024149844A1 (en) * 2023-01-12 2024-07-18 Nantes Université Chemically-modified adeno-associated virus

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US20110034540A1 (en) * 2007-09-12 2011-02-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Use of the CYP46A1 Gene for the Treatment of Alzheimer's Disease
JP2013544236A (ja) * 2010-10-15 2013-12-12 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ハンチントン病の治療法におけるコレステロール−24−ヒドロラーゼのための発現ベクター
US20190298754A1 (en) * 2016-12-02 2019-10-03 Seoul National University R&Db Foundation Use of microrna-188-5p for treating alzheimer's disease
JP2019533439A (ja) * 2016-09-22 2019-11-21 ユニバーシティ オブ マサチューセッツ ハンチントン病のaav処置
US20200071699A1 (en) * 2018-08-31 2020-03-05 Ghassan S. Kassab Treatment of alzheimers disease with micro rna and ghrelin

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US8900627B2 (en) 2008-06-06 2014-12-02 Mirna Therapeutics, Inc. Compositions for the in vivo delivery of RNAi agents
KR101367568B1 (ko) * 2011-05-31 2014-02-27 임우석 Abc 수송체 조절을 통한 퇴행성신경질환에서 이상단백질 조절
BR112014006596A2 (pt) 2011-09-21 2017-03-28 Yissum Res Dev Co nanopartícula que encapsula uma pluralidade de nanocarreadores, composição, uso de uma nanopartícula, kit, sistema de entrega, processo para obter uma nanopartícula, e, processo de nanoaspersão para obter as nanopartículas
ES2661236T3 (es) 2011-12-13 2018-03-28 Henry Ford Health System Procedimientos, sistemas y composiciones para administración de microARN derivado de células/a base de vesículas
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
WO2019177550A1 (en) 2018-03-10 2019-09-19 Koc Universitesi Therapeutic nanoparticles containing argonaute for microrna delivery and compositions and methods using same
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US20110034540A1 (en) * 2007-09-12 2011-02-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Use of the CYP46A1 Gene for the Treatment of Alzheimer's Disease
JP2013544236A (ja) * 2010-10-15 2013-12-12 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ハンチントン病の治療法におけるコレステロール−24−ヒドロラーゼのための発現ベクター
JP2019533439A (ja) * 2016-09-22 2019-11-21 ユニバーシティ オブ マサチューセッツ ハンチントン病のaav処置
US20190298754A1 (en) * 2016-12-02 2019-10-03 Seoul National University R&Db Foundation Use of microrna-188-5p for treating alzheimer's disease
US20200071699A1 (en) * 2018-08-31 2020-03-05 Ghassan S. Kassab Treatment of alzheimers disease with micro rna and ghrelin

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US20230365963A1 (en) 2023-11-16
CA3193406A1 (en) 2022-03-24
AU2021344607A1 (en) 2023-06-01
WO2022061378A2 (en) 2022-03-24
IL301525A (en) 2023-05-01
EP4213891A4 (en) 2025-07-23
WO2022061378A3 (en) 2022-04-28
EP4213891A2 (en) 2023-07-26
AU2021344607A9 (en) 2024-09-12

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