JP7681277B2 - 関節炎疾患の治療のための修飾ａａｖキャプシドタンパク質 - Google Patents

関節炎疾患の治療のための修飾ａａｖキャプシドタンパク質 Download PDF

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JP7681277B2
JP7681277B2 JP2022502928A JP2022502928A JP7681277B2 JP 7681277 B2 JP7681277 B2 JP 7681277B2 JP 2022502928 A JP2022502928 A JP 2022502928A JP 2022502928 A JP2022502928 A JP 2022502928A JP 7681277 B2 JP7681277 B2 JP 7681277B2
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seq
amino acid
capsid protein
acid sequence
transgene
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JP2022541520A5 (enExample
JP2022541520A (ja
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マリア ヘルトルーデ ファン デル サンデン サビーネ
アンナ スノーク スサンネ
ブルクストラ ニールス
ダグラス フィン ジョナサン
グリム ディルク
ベルナー カトリーン
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メイラグティーエックス ユーケー アイアイ リミティド
ユニバーシティ オブ ハイデルベルク
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/24—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
    • C07K16/244—Interleukins [IL]
    • C07K16/245—IL-1
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/24—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
    • C07K16/244—Interleukins [IL]
    • C07K16/248—IL-6
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2866—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against receptors for cytokines, lymphokines, interferons
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/86—Viral vectors
    • 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
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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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    • C12N2750/00011—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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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
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  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2022502928A 2019-07-15 2020-07-15 関節炎疾患の治療のための修飾ａａｖキャプシドタンパク質 Active JP7681277B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
NL2023505 2019-07-15
NL2023505 2019-07-15
PCT/IB2020/056635 WO2021009684A1 (en) 2019-07-15 2020-07-15 Modified aav capsid proteins for treatment of arthritic disease

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Publication Number Publication Date
JP2022541520A JP2022541520A (ja) 2022-09-26
JP2022541520A5 JP2022541520A5 (enExample) 2023-07-21
JP7681277B2 true JP7681277B2 (ja) 2025-05-22

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US (1) US20220267797A1 (enExample)
EP (1) EP3999119B1 (enExample)
JP (1) JP7681277B2 (enExample)
KR (1) KR20220081971A (enExample)
CN (1) CN114502737B (enExample)
AU (1) AU2020314883A1 (enExample)
BR (1) BR112022000724A2 (enExample)
CA (1) CA3146791A1 (enExample)
DK (1) DK3999119T3 (enExample)
ES (1) ES2998884T3 (enExample)
FI (1) FI3999119T3 (enExample)
HR (1) HRP20241647T1 (enExample)
HU (1) HUE069369T2 (enExample)
IL (1) IL289826A (enExample)
LT (1) LT3999119T (enExample)
MX (1) MX2022000551A (enExample)
PH (1) PH12022550109A1 (enExample)
PL (1) PL3999119T3 (enExample)
PT (1) PT3999119T (enExample)
RS (1) RS66255B1 (enExample)
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SM (1) SMT202400486T1 (enExample)
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JP7389040B2 (ja) * 2018-01-17 2023-11-29 メイラジーティーエックス・ユーケー・ザ・セカンド・リミテッド 遺伝子治療のための修飾ｒＡＡＶキャプシドタンパク質
WO2022198126A1 (en) * 2021-03-19 2022-09-22 Washington University Methods for generating mechanically-responsive cells and uses thereof
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
WO2023125481A1 (zh) * 2021-12-28 2023-07-06 成都弘基生物科技有限公司 经过修饰的aav衣壳蛋白及其用途
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
EP4694906A1 (en) 2023-04-13 2026-02-18 MeiraGTx Gene Regulation Limited Inflammation-inducible promoters
KR20260082361A (ko) 2023-08-31 2026-06-04 디노 테라퓨틱스, 인코포레이티드 캡시드 폴리펩타이드 및 이의 사용 방법
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
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026122963A1 (en) 2024-12-06 2026-06-11 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

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WO2018106956A2 (en) 2016-12-07 2018-06-14 University Of Florida Research Foundation, Incorporated IL-1RA CDNAs

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EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
DE23193606T1 (de) * 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
PT3272872T (pt) 2005-10-20 2020-06-26 Uniqure Ip Bv Vetores aav melhorados produzidos em células de insetos
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WO2013174760A1 (en) 2012-05-25 2013-11-28 Deutsches Krebsforschungszentrum Optimized aav-vectors for high transduction rates in dendritic cells
WO2018035451A1 (en) 2016-08-19 2018-02-22 Calimmune, Inc. Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus
WO2018106956A2 (en) 2016-12-07 2018-06-14 University Of Florida Research Foundation, Incorporated IL-1RA CDNAs

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MX2022000551A (es) 2022-05-18
RS66255B1 (sr) 2024-12-31
KR20220081971A (ko) 2022-06-16
HRP20241647T1 (hr) 2025-02-14
PH12022550109A1 (en) 2022-12-12
EP3999119A1 (en) 2022-05-25
DK3999119T3 (da) 2024-10-28
SMT202400486T1 (it) 2025-01-14
HUE069369T2 (hu) 2025-02-28
BR112022000724A2 (pt) 2022-06-14
PL3999119T3 (pl) 2025-02-10
CN114502737A (zh) 2022-05-13
US20220267797A1 (en) 2022-08-25
PT3999119T (pt) 2024-11-25
CA3146791A1 (en) 2021-01-21
ES2998884T3 (en) 2025-02-24
AU2020314883A1 (en) 2022-03-03
WO2021009684A1 (en) 2021-01-21
IL289826A (en) 2022-03-01
CN114502737B (zh) 2025-05-09
FI3999119T3 (fi) 2024-11-01
EP3999119B1 (en) 2024-09-11
LT3999119T (lt) 2024-12-10
JP2022541520A (ja) 2022-09-26
SI3999119T1 (sl) 2025-03-31

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