JP2021521833A - 筋疾患の治療のための改変ａａｖカプシドポリペプチド - Google Patents

筋疾患の治療のための改変ａａｖカプシドポリペプチド Download PDF

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JP2021521833A
JP2021521833A JP2020560127A JP2020560127A JP2021521833A JP 2021521833 A JP2021521833 A JP 2021521833A JP 2020560127 A JP2020560127 A JP 2020560127A JP 2020560127 A JP2020560127 A JP 2020560127A JP 2021521833 A JP2021521833 A JP 2021521833A
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aav capsid
capsid polypeptide
muscle
aav
amino acid
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JP2021521833A5 (enExample
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グリム，ディルク
ヴァインマン，ヨーナス
アンダリ，ヨーゼフ エル
アンダリ，ヨーゼフ エル
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ウニベルジテート ハイデルベルク
ウニベルジテート ハイデルベルク
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Publication of JP2021521833A publication Critical patent/JP2021521833A/ja
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    • 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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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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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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    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/035—Fusion polypeptide containing a localisation/targetting motif containing a signal for targeting to the external surface of a cell, e.g. to the outer membrane of Gram negative bacteria, GPI- anchored eukaryote proteins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011—Parvoviridae
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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
    • C12N2750/14145—Special targeting system for viral vectors

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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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JP2020560127A 2018-04-27 2019-04-26 筋疾患の治療のための改変ａａｖカプシドポリペプチド Pending JP2021521833A (ja)

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JP2023191983A JP7707261B2 (ja) 2018-04-27 2023-11-10 筋疾患の治療のための改変ａａｖカプシドポリペプチド

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EP18169822.6 2018-04-27
EP18169822 2018-04-27
PCT/EP2019/060790 WO2019207132A1 (en) 2018-04-27 2019-04-26 Modified aav capsid polypeptides for treatment of muscular diseases

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JP2021521833A5 JP2021521833A5 (enExample) 2022-05-09

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JP2023191983A Active JP7707261B2 (ja) 2018-04-27 2023-11-10 筋疾患の治療のための改変ａａｖカプシドポリペプチド

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US (1) US20210363193A1 (enExample)
EP (2) EP3784288B1 (enExample)
JP (2) JP2021521833A (enExample)
CN (1) CN112040988A (enExample)
AU (2) AU2019258830C1 (enExample)
CA (1) CA3097375A1 (enExample)
WO (1) WO2019207132A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022551487A (ja) * 2019-10-10 2022-12-09 ソリッド・バイオサイエンシーズ・インコーポレーテッド 改変されたａａｖカプシドおよびその使用
JP2022551986A (ja) * 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物

Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL278213B2 (en) 2018-04-27 2026-04-01 Rocket Pharmaceuticals Ltd A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
MX2021005720A (es) 2018-11-16 2021-07-21 Astellas Pharma Inc Metodo para tratar la distrofia muscular mediante el direccionamiento del gen de utrofina.
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
US20220325296A1 (en) * 2019-09-12 2022-10-13 The Broad Institute, Inc. Engineered adeno-associated virus capsids
EP4031673A1 (en) * 2019-09-19 2022-07-27 Genethon Gene therapy expression system alleviating cardiac toxicity of fkrp
TWI897882B (zh) * 2019-09-20 2025-09-21 英商Ｕｃｌ商業有限責任公司 右心室心律失常性心肌病之基因療法組合物及治療
CN115379863A (zh) 2020-04-14 2022-11-22 吉尼松公司 用于治疗酸性神经酰胺酶缺乏症的载体
AU2021259422A1 (en) 2020-04-20 2022-12-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
US20230173102A1 (en) * 2020-04-28 2023-06-08 Genethon Use of a Synthetic AAV Capsid for Gene Therapy of Muscle and Central Nervous System Disorders
AU2021267885A1 (en) * 2020-05-05 2023-01-19 Duke University Compositions and methods for formation and secretion of extracellular vesicles and AAV particles
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
US12668814B2 (en) * 2020-07-22 2026-06-30 The Broad Institute, Inc. Engineered muscle targeting compositions
WO2022020616A1 (en) * 2020-07-22 2022-01-27 The Broad Institute, Inc. Engineered muscle targeting compositions
IL300187A (en) * 2020-08-05 2023-03-01 Spacecraft Seven Llc Gene therapy CSRP3 (protein 3 rich in cysteine and glycine)
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
CN116096431A (zh) * 2020-09-10 2023-05-09 吉尼松公司 肽修饰的aav衣壳
CN112194706B (zh) * 2020-09-30 2022-03-08 广州派真生物技术有限公司 腺相关病毒突变体及其应用
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
MX2023005446A (es) 2020-11-11 2023-07-18 European Molecular Biology Laboratory Particulas virales modificadas para terapia genica.
WO2022114243A1 (en) * 2020-11-25 2022-06-02 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting dmpk gene
CA3210955A1 (en) * 2021-02-09 2022-08-18 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
CA3216419A1 (en) * 2021-04-23 2022-10-27 Greg NACHTRAB Tissue-targeted modified aav capsids and methods of use thereof
CN113480615B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
EP4453013A1 (en) * 2021-12-21 2024-10-30 Research Institute at Nationwide Children's Hospital Materials and methods for the treatment of limb girdle muscular dystrophy
CN114516901B (zh) * 2022-03-11 2023-01-17 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
US20250195694A1 (en) * 2022-03-18 2025-06-19 University Of Florida Research Foundation, Incorporated Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector
KR20240169111A (ko) 2022-04-06 2024-12-02 젠자임 코포레이션 Dm-1 근긴장성 이영양증에 대한 표적화된 유전자 요법
EP4507719A1 (en) 2022-04-13 2025-02-19 Universitat Autònoma de Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
EP4269426A1 (en) * 2022-04-29 2023-11-01 Ospedale San Raffaele S.r.l. Gene therapy
FI4536686T3 (fi) 2022-06-10 2026-01-13 Genethon Lihaksen parannetun transduktiotehokkuuden omaava peptidimodifioitu aav-kapsidi
JP2026509271A (ja) 2023-03-10 2026-03-17 ダイノ セラピューティクス，インコーポレーテッド カプシドポリペプチドおよびその使用方法
WO2024188913A1 (en) 2023-03-10 2024-09-19 Genethon Induction of immune tolerance by aav vector comprising the combination of a liver detargeted capsid and a tandem liver-muscle specific promoter
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024211480A2 (en) 2023-04-05 2024-10-10 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
AU2024271851A1 (en) 2023-05-16 2025-12-18 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
CN121712809A (zh) 2023-07-03 2026-03-20 Ucb生物制药有限责任公司 工程化的aav衣壳蛋白
CN117964715B (zh) * 2023-07-21 2025-01-24 广州派真生物技术有限公司 腺相关病毒突变体及其应用
KR20260082361A (ko) 2023-08-31 2026-06-04 디노 테라퓨틱스, 인코포레이티드 캡시드 폴리펩타이드 및 이의 사용 방법
WO2025133247A1 (en) 2023-12-21 2025-06-26 Genethon Methods for aav vector re-administration
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
WO2025232917A1 (en) * 2024-05-10 2025-11-13 Hangzhou Jiayin Biotech Ltd. Novel liver detargeted aav variants and uses thereof
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2026017727A1 (en) 2024-07-16 2026-01-22 Genethon Alternative barcoding method to assess transduction efficiency of aav vectors
WO2026064444A1 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064428A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026094009A1 (en) 2024-11-04 2026-05-07 Insmed Incorporated Compositions and methods for treating duchenne muscular dystrophy
WO2026122963A1 (en) 2024-12-06 2026-06-11 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005512569A (ja) * 2001-12-21 2005-05-12 メディジーン・アクチェンゲゼルシャフト 目的とする細胞指向性をもつウイルスクローンの同定に有用な修飾した構造遺伝子またはキャプシド修飾した粒子のライブラリー
JP2015517308A (ja) * 2012-05-09 2015-06-22 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
JP2021520203A (ja) * 2018-04-05 2021-08-19 ジェネトン 減少した肝臓向性を有するＡＡＶ９とＡＡＶｒｈ７４とのハイブリッド組換えアデノ随伴ウイルス血清型

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6833447B1 (en) * 2000-07-10 2004-12-21 Monsanto Technology, Llc Myxococcus xanthus genome sequences and uses thereof
WO2005017101A2 (en) * 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
US7467102B2 (en) * 2003-09-11 2008-12-16 International Business Machines Corporation Request type grid computing
US20130305398A1 (en) * 2012-02-16 2013-11-14 Marie Coffin Genes and uses for plant enhacement
EP2970995A1 (en) * 2013-03-14 2016-01-20 Pioneer Hi-Bred International, Inc. Compositions having dicamba decarboxylase activity and methods of use
WO2017096164A1 (en) * 2015-12-02 2017-06-08 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
WO2017189963A1 (en) * 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2018129203A2 (en) * 2017-01-06 2018-07-12 The Regents Of The University Of California Method for temporal and tissue-specific drug delivery and induced nucleic acid recombination
JP7403165B2 (ja) * 2017-12-19 2023-12-22 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 血液脳関門を通過してウイルスベクターを送達するための方法および組成物

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005512569A (ja) * 2001-12-21 2005-05-12 メディジーン・アクチェンゲゼルシャフト 目的とする細胞指向性をもつウイルスクローンの同定に有用な修飾した構造遺伝子またはキャプシド修飾した粒子のライブラリー
JP2015517308A (ja) * 2012-05-09 2015-06-22 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
JP2021520203A (ja) * 2018-04-05 2021-08-19 ジェネトン 減少した肝臓向性を有するＡＡＶ９とＡＡＶｒｈ７４とのハイブリッド組換えアデノ随伴ウイルス血清型

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
DOBRUCKI LW. ET AL.: "Analysis of angiogenesis induced by local IGF-1 expression after myocardial infarction using microSP", J MOL CELL CARDIOL, 2010, VOL. 48, P. 1071-1079, JPN6023010374, ISSN: 0005104372 *
VARADI K. ET AL.: "Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directe", GENE THERAPY, 2012, VOL. 19, P. 800-809, JPN6023010375, ISSN: 0005104373 *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022551487A (ja) * 2019-10-10 2022-12-09 ソリッド・バイオサイエンシーズ・インコーポレーテッド 改変されたａａｖカプシドおよびその使用
US12653909B2 (en) 2019-10-10 2026-06-16 Solid Biosciences Inc. Modified AAV capsids and uses thereof
JP2022551986A (ja) * 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物

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