PL3999119T3 - Modyfikowane białka kapsydu aav do leczenia choroby zapalnej stawów - Google Patents

Modyfikowane białka kapsydu aav do leczenia choroby zapalnej stawów

Info

Publication number
PL3999119T3
PL3999119T3 PL20740418.7T PL20740418T PL3999119T3 PL 3999119 T3 PL3999119 T3 PL 3999119T3 PL 20740418 T PL20740418 T PL 20740418T PL 3999119 T3 PL3999119 T3 PL 3999119T3
Authority
PL
Poland
Prior art keywords
treatment
capsid proteins
aav capsid
inflammatory arthritis
modified aav
Prior art date
Application number
PL20740418.7T
Other languages
English (en)
Inventor
Sabine Maria Gertrude Van Der Sanden
Susanne Anna Snoek
Niels Broekstra
Jonathan Douglas FINN
Dirk Grimm
Kathleen BÖRNER
Original Assignee
Meiragtx Uk Ii Limited
University Of Heidelberg
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Meiragtx Uk Ii Limited, University Of Heidelberg filed Critical Meiragtx Uk Ii Limited
Publication of PL3999119T3 publication Critical patent/PL3999119T3/pl

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/24Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
    • C07K16/244Interleukins [IL]
    • C07K16/245IL-1
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/24Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
    • C07K16/244Interleukins [IL]
    • C07K16/248IL-6
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2866Immunoglobulins [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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • 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)
PL20740418.7T 2019-07-15 2020-07-15 Modyfikowane białka kapsydu aav do leczenia choroby zapalnej stawów PL3999119T3 (pl)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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
PL3999119T3 true PL3999119T3 (pl) 2025-02-10

Family

ID=68425203

Family Applications (1)

Application Number Title Priority Date Filing Date
PL20740418.7T PL3999119T3 (pl) 2019-07-15 2020-07-15 Modyfikowane białka kapsydu aav do leczenia choroby zapalnej stawów

Country Status (23)

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

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019209595B2 (en) * 2018-01-17 2023-04-27 Meiragtx Uk Ii Limited 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.
AU2022423978A1 (en) * 2021-12-28 2024-07-18 Chengdu Origen Biotechnology Co., Ltd. Modified aav capsid protein and use thereof
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
KR20260013513A (ko) 2023-04-13 2026-01-28 메이라지티엑스 진 레귤레이션 리미티드 염증 유도성 프로모터
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
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

Family Cites Families (21)

* Cited by examiner, † Cited by third party
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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
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
US8999678B2 (en) * 2005-04-07 2015-04-07 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
DK3093345T3 (da) 2007-07-26 2019-06-24 Uniqure Ip Bv Baculovirusvektorer omfattende gentagne kodende sekvenser med differentielle foretrukne kodoner
CA2715924C (en) 2008-02-19 2021-01-12 Andrew Christian BAKKER Optimisation of expression of parvoviral rep and cap proteins in insect cells
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker Parvoviral capsid with incorporated gly-ala repeat region
EP2545165B1 (en) 2010-03-11 2020-07-29 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
EP3425044A1 (en) 2011-09-08 2019-01-09 uniQure IP B.V. Removal of contaminating viruses from aav preparations
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
WO2015168666A2 (en) * 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
CA2963168A1 (en) 2014-10-06 2016-04-14 Arthrogen B.V. Aav-based gene therapy
CN116333057A (zh) * 2016-05-13 2023-06-27 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
KR20230130155A (ko) * 2016-08-19 2023-09-11 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 재조합 자기-상보적인 아데노-부속 바이러스를 이용한 병태의 치료 방법 및 조성물
WO2018106956A2 (en) * 2016-12-07 2018-06-14 University Of Florida Research Foundation, Incorporated IL-1RA CDNAs
WO2018189244A1 (en) * 2017-04-11 2018-10-18 Ruprecht-Karls-Universität Heidelberg Adeno-associated virus library
AU2019209595B2 (en) * 2018-01-17 2023-04-27 Meiragtx Uk Ii Limited A modified rAAV capsid protein for gene therapy

Also Published As

Publication number Publication date
JP2022541520A (ja) 2022-09-26
CA3146791A1 (en) 2021-01-21
JP7681277B2 (ja) 2025-05-22
EP3999119A1 (en) 2022-05-25
RS66255B1 (sr) 2024-12-31
US20220267797A1 (en) 2022-08-25
WO2021009684A1 (en) 2021-01-21
CN114502737B (zh) 2025-05-09
PH12022550109A1 (en) 2022-12-12
SMT202400486T1 (it) 2025-01-14
MX2022000551A (es) 2022-05-18
SI3999119T1 (sl) 2025-03-31
CN114502737A (zh) 2022-05-13
HRP20241647T1 (hr) 2025-02-14
BR112022000724A2 (pt) 2022-06-14
LT3999119T (lt) 2024-12-10
ES2998884T3 (en) 2025-02-24
DK3999119T3 (da) 2024-10-28
IL289826A (en) 2022-03-01
AU2020314883A1 (en) 2022-03-03
KR20220081971A (ko) 2022-06-16
FI3999119T3 (fi) 2024-11-01
HUE069369T2 (hu) 2025-02-28
EP3999119B1 (en) 2024-09-11
PT3999119T (pt) 2024-11-25

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