KR20220081971A - 관절염 질환 치료용 변형된 aav 캡시드 단백질 - Google Patents

관절염 질환 치료용 변형된 aav 캡시드 단백질 Download PDF

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KR20220081971A
KR20220081971A KR1020227004437A KR20227004437A KR20220081971A KR 20220081971 A KR20220081971 A KR 20220081971A KR 1020227004437 A KR1020227004437 A KR 1020227004437A KR 20227004437 A KR20227004437 A KR 20227004437A KR 20220081971 A KR20220081971 A KR 20220081971A
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South Korea
Prior art keywords
seq
capsid protein
amino acid
transgene
modified capsid
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KR1020227004437A
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Korean (ko)
Inventor
사빈 마리아 거트루드 반 더 샌딘
수잔느 안나 스노크
닐스 브록스트라
조나단 더글라스 핀
더크 그림
캐슬린 뵈르너
Original Assignee
메이라지티엑스 유케이 Ii 리미티드
우니페르시테트 하이델베르크
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Publication of KR20220081971A publication Critical patent/KR20220081971A/ko
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    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • 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
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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
    • 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
    • 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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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
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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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  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Rheumatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (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)
KR1020227004437A 2019-07-15 2020-07-15 관절염 질환 치료용 변형된 aav 캡시드 단백질 Pending KR20220081971A (ko)

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

Publications (1)

Publication Number Publication Date
KR20220081971A true KR20220081971A (ko) 2022-06-16

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ID=68425203

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227004437A Pending KR20220081971A (ko) 2019-07-15 2020-07-15 관절염 질환 치료용 변형된 aav 캡시드 단백질

Country Status (23)

Country Link
US (1) US20220267797A1 (https=)
EP (1) EP3999119B1 (https=)
JP (1) JP7681277B2 (https=)
KR (1) KR20220081971A (https=)
CN (1) CN114502737B (https=)
AU (1) AU2020314883A1 (https=)
BR (1) BR112022000724A2 (https=)
CA (1) CA3146791A1 (https=)
DK (1) DK3999119T3 (https=)
ES (1) ES2998884T3 (https=)
FI (1) FI3999119T3 (https=)
HR (1) HRP20241647T1 (https=)
HU (1) HUE069369T2 (https=)
IL (1) IL289826A (https=)
LT (1) LT3999119T (https=)
MX (1) MX2022000551A (https=)
PH (1) PH12022550109A1 (https=)
PL (1) PL3999119T3 (https=)
PT (1) PT3999119T (https=)
RS (1) RS66255B1 (https=)
SI (1) SI3999119T1 (https=)
SM (1) SMT202400486T1 (https=)
WO (1) WO2021009684A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202006056RA (en) * 2018-01-17 2020-07-29 Meiragtx Uk Ii Ltd A modified raav capsid protein for gene therapy
MX2024004217A (es) 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
IL313832A (en) * 2021-12-28 2024-08-01 Chengdu Origen Biotechnology Co Ltd Modified AAV capsid protein and its use
TW202502803A (zh) 2023-03-10 2025-01-16 美商戴諾治療公司 衣殼多肽及其使用方法
CN121311235A (zh) 2023-04-13 2026-01-09 梅里特斯基因调控有限公司 炎症诱导型启动子
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)
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

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Publication number Priority date Publication date Assignee Title
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2006110689A2 (en) * 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
EP2311967B1 (en) 2005-10-20 2017-09-20 UniQure IP B.V. Improved AAV vectors produced in insect cells
KR101589259B1 (ko) 2006-06-21 2016-02-01 유니큐어 아이피 비.브이. 곤충세포 내 ａａｖ의 생산에 유용한 ａａｖ-ｒｅｐ78의 번역을 위한 변형된 개시 코돈을 갖는 벡터
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
EP2012122A1 (en) * 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
SI3093345T1 (sl) 2007-07-26 2019-08-30 Uniqure Ip B.V. Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami
EP3257937B1 (en) 2008-02-19 2022-08-03 uniQure IP B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
WO2009154452A1 (en) 2008-06-17 2009-12-23 Amsterdam Molecular Therapeutics B.V. Parvoviral capsid with incorporated Gly-Ala repeat region
WO2011112089A2 (en) 2010-03-11 2011-09-15 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Mutated rep encoding sequences for use in aav production
PT2744895E (pt) 2011-09-08 2016-02-08 Uniqure Ip Bv Remoção de vírus contaminantes de preparações de aav
WO2013174760A1 (en) * 2012-05-25 2013-11-28 Deutsches Krebsforschungszentrum Optimized aav-vectors for high transduction rates in dendritic cells
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
IL248102B (en) * 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
CN107002096A (zh) 2014-10-06 2017-08-01 阿罗根有限公司 基于aav的基因疗法
FI3445773T3 (fi) * 2016-05-13 2023-03-30 4D Molecular Therapeutics Inc Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä
CN109843306A (zh) 2016-08-19 2019-06-04 卡琳缪恩股份有限公司 使用自身互补型重组腺相关病毒治疗病症的方法和组合物
KR20250121159A (ko) 2016-12-07 2025-08-11 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 인터류킨-1 수용체 길항제(IL-1RA) cDNA
US20200370039A1 (en) * 2017-04-11 2020-11-26 Ruprecht-Karls-Universitat Heidelberg Adeno-associated virus library
SG11202006056RA (en) * 2018-01-17 2020-07-29 Meiragtx Uk Ii Ltd A modified raav capsid protein for gene therapy

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Publication number Publication date
HRP20241647T1 (hr) 2025-02-14
DK3999119T3 (da) 2024-10-28
EP3999119A1 (en) 2022-05-25
ES2998884T3 (en) 2025-02-24
CN114502737A (zh) 2022-05-13
FI3999119T3 (fi) 2024-11-01
AU2020314883A1 (en) 2022-03-03
IL289826A (en) 2022-03-01
US20220267797A1 (en) 2022-08-25
JP2022541520A (ja) 2022-09-26
CN114502737B (zh) 2025-05-09
WO2021009684A1 (en) 2021-01-21
CA3146791A1 (en) 2021-01-21
SMT202400486T1 (it) 2025-01-14
SI3999119T1 (sl) 2025-03-31
MX2022000551A (es) 2022-05-18
PH12022550109A1 (en) 2022-12-12
PT3999119T (pt) 2024-11-25
JP7681277B2 (ja) 2025-05-22
HUE069369T2 (hu) 2025-02-28
LT3999119T (lt) 2024-12-10
BR112022000724A2 (pt) 2022-06-14
RS66255B1 (sr) 2024-12-31
PL3999119T3 (pl) 2025-02-10
EP3999119B1 (en) 2024-09-11

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