CN114502737B - 用于治疗关节炎疾病的修饰的aav衣壳蛋白 - Google Patents

用于治疗关节炎疾病的修饰的aav衣壳蛋白 Download PDF

Info

Publication number
CN114502737B
CN114502737B CN202080064732.0A CN202080064732A CN114502737B CN 114502737 B CN114502737 B CN 114502737B CN 202080064732 A CN202080064732 A CN 202080064732A CN 114502737 B CN114502737 B CN 114502737B
Authority
CN
China
Prior art keywords
seq
capsid protein
transgene
modified capsid
amino acid
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
CN202080064732.0A
Other languages
English (en)
Chinese (zh)
Other versions
CN114502737A (zh
Inventor
S·M·G·范德桑登
S·A·斯内克
N·布雷克斯特拉
J·D·芬
D·格林
K·贝尔纳
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Meritus Gene Regulation Ltd
Fraunhofer Gesellschaft zur Foerderung der Angewandten Forschung eV
Original Assignee
Fraunhofer Gesellschaft zur Foerderung der Angewandten Forschung eV
MeiraGTx UK II Ltd
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 Fraunhofer Gesellschaft zur Foerderung der Angewandten Forschung eV, MeiraGTx UK II Ltd filed Critical Fraunhofer Gesellschaft zur Foerderung der Angewandten Forschung eV
Publication of CN114502737A publication Critical patent/CN114502737A/zh
Application granted granted Critical
Publication of CN114502737B publication Critical patent/CN114502737B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14171—Demonstrated 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)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • 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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
CN202080064732.0A 2019-07-15 2020-07-15 用于治疗关节炎疾病的修饰的aav衣壳蛋白 Active CN114502737B (zh)

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 (2)

Publication Number Publication Date
CN114502737A CN114502737A (zh) 2022-05-13
CN114502737B true CN114502737B (zh) 2025-05-09

Family

ID=68425203

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080064732.0A Active CN114502737B (zh) 2019-07-15 2020-07-15 用于治疗关节炎疾病的修饰的aav衣壳蛋白

Country Status (23)

Country Link
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)
SI (1) SI3999119T1 (enExample)
SM (1) SMT202400486T1 (enExample)
WO (1) WO2021009684A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RS64499B1 (sr) * 2018-01-17 2023-09-29 Meiragtx Uk Ii Ltd Modifikovani protein kapsida raav za gensku terapiju
EP4413147A4 (en) 2021-10-08 2025-07-30 Dyno Therapeutics Inc CAPSID VARIANTS AND METHODS OF USE THEREOF
CN116444626A (zh) * 2021-12-28 2023-07-18 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
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2024213660A1 (en) 2023-04-13 2024-10-17 Meiragtx Uk Ii Limited Inflammation-inducible promoters
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
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112029737A (zh) * 2005-04-07 2020-12-04 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2002360355B2 (en) 2001-11-09 2005-07-07 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
AU2006304997B2 (en) 2005-10-20 2012-03-01 Uniqure Ip B.V. Improved AAV vectors produced in insect cells
ES2785223T3 (es) 2006-06-21 2020-10-06 Uniqure Ip Bv Células de insecto para la producción de vectores de AAV
EP2061891B1 (en) 2006-08-24 2012-04-11 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
EP2012122A1 (en) * 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
SI2173888T1 (sl) 2007-07-26 2017-01-31 Uniqure Ip B.V. Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami
ES2644880T3 (es) 2008-02-19 2017-11-30 Uniqure Ip B.V. Optimización de expresión de proteínas Rep y Cap parvovirales en las células de insecto
EP2297185A1 (en) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. 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
AU2012304993B2 (en) 2011-09-08 2017-09-14 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
EP3137497B2 (en) * 2014-05-02 2025-03-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2016055437A1 (en) 2014-10-06 2016-04-14 Arthrogen B.V. Aav-based gene therapy
WO2017197355A2 (en) * 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
US11207382B2 (en) * 2016-08-19 2021-12-28 University Of Florida Research Foundation, Incorporated Compositions for treating conditions using recombinant self-complementary adeno-associated virus
KR20250121159A (ko) * 2016-12-07 2025-08-11 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 인터류킨-1 수용체 길항제(IL-1RA) cDNA
WO2018189244A1 (en) * 2017-04-11 2018-10-18 Ruprecht-Karls-Universität Heidelberg Adeno-associated virus library
RS64499B1 (sr) * 2018-01-17 2023-09-29 Meiragtx Uk Ii Ltd Modifikovani protein kapsida raav za gensku terapiju

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112029737A (zh) * 2005-04-07 2020-12-04 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法

Also Published As

Publication number Publication date
BR112022000724A2 (pt) 2022-06-14
IL289826A (en) 2022-03-01
HRP20241647T1 (hr) 2025-02-14
US20220267797A1 (en) 2022-08-25
SMT202400486T1 (it) 2025-01-14
EP3999119A1 (en) 2022-05-25
PH12022550109A1 (en) 2022-12-12
SI3999119T1 (sl) 2025-03-31
CN114502737A (zh) 2022-05-13
DK3999119T3 (da) 2024-10-28
AU2020314883A1 (en) 2022-03-03
RS66255B1 (sr) 2024-12-31
KR20220081971A (ko) 2022-06-16
HUE069369T2 (hu) 2025-02-28
PT3999119T (pt) 2024-11-25
JP7681277B2 (ja) 2025-05-22
ES2998884T3 (en) 2025-02-24
MX2022000551A (es) 2022-05-18
LT3999119T (lt) 2024-12-10
PL3999119T3 (pl) 2025-02-10
FI3999119T3 (fi) 2024-11-01
WO2021009684A1 (en) 2021-01-21
JP2022541520A (ja) 2022-09-26
CA3146791A1 (en) 2021-01-21
EP3999119B1 (en) 2024-09-11

Similar Documents

Publication Publication Date Title
CN112004544B (zh) 用于基因疗法的修饰的rAAV衣壳蛋白
JP7681277B2 (ja) 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
US20170304466A1 (en) AAV-Based Gene Therapy
JP2018515096A (ja) 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むａａｖ分離株及び融合タンパク質
JP4863874B2 (ja) 関節リウマチのインビボ遺伝子治療のためのａａｖベクター
EA048424B1 (ru) Модифицированные капсидные белки aav для лечения заболевания суставов
HK40072785B (en) Modified aav capsid proteins for treatment of arthritic disease
HK40072785A (en) Modified aav capsid proteins for treatment of arthritic disease
HK40039645B (en) A modified raav capsid protein for gene therapy
HK40039645A (en) A modified raav capsid protein for gene therapy
EA045763B1 (ru) Модифицированный капсидный белок raav для генной терапии
NZ767106B2 (en) A modified raav capsid protein for gene therapy
AU2010201278A1 (en) AAV vectors for in vivo gene therapy of rheumatoid arthritis

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant
TR01 Transfer of patent right

Effective date of registration: 20260109

Address after: London, England

Patentee after: Meritus Gene Regulation Ltd.

Country or region after: United Kingdom

Patentee after: FRAUNHOFER-GESELLSCHAFT ZUR FORDERUNG DER ANGEWANDTEN FORSCHUNG E.V.

Country or region after: Germany

Address before: London, England

Patentee before: MEIRAGTX UK II LTD.

Country or region before: United Kingdom

Patentee before: FRAUNHOFER-GESELLSCHAFT ZUR FORDERUNG DER ANGEWANDTEN FORSCHUNG E.V.

Country or region before: Germany

TR01 Transfer of patent right