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RS64499B1 - Modifikovani protein kapsida raav za gensku terapiju - Google Patents

Modifikovani protein kapsida raav za gensku terapiju

Info

Publication number
RS64499B1
RS64499B1 RS20230730A RSP20230730A RS64499B1 RS 64499 B1 RS64499 B1 RS 64499B1 RS 20230730 A RS20230730 A RS 20230730A RS P20230730 A RSP20230730 A RS P20230730A RS 64499 B1 RS64499 B1 RS 64499B1
Authority
RS
Serbia
Prior art keywords
gene therapy
capsid protein
raav capsid
modified raav
modified
Prior art date
Application number
RS20230730A
Other languages
English (en)
Inventor
Jonathan Douglas Finn
Dirk Grimm
Kathleen Börner
Susanne Anna Snoek
Niels Broekstra
Der Sanden Sabine Maria Gertrude Van
Original Assignee
Meiragtx Uk Ii Ltd
Univ 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 Ltd, Univ Heidelberg filed Critical Meiragtx Uk Ii Ltd
Publication of RS64499B1 publication Critical patent/RS64499B1/sr

Links

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    • 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
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • 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
    • 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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dermatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
RS20230730A 2018-01-17 2019-01-17 Modifikovani protein kapsida raav za gensku terapiju RS64499B1 (sr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18152133 2018-01-17
EP19701080.4A EP3740222B1 (en) 2018-01-17 2019-01-17 A modified raav capsid protein for gene therapy
PCT/EP2019/051128 WO2019141765A1 (en) 2018-01-17 2019-01-17 A modified raav capsid protein for gene therapy

Publications (1)

Publication Number Publication Date
RS64499B1 true RS64499B1 (sr) 2023-09-29

Family

ID=61022137

Family Applications (1)

Application Number Title Priority Date Filing Date
RS20230730A RS64499B1 (sr) 2018-01-17 2019-01-17 Modifikovani protein kapsida raav za gensku terapiju

Country Status (25)

Country Link
US (1) US20200354744A1 (sr)
EP (1) EP3740222B1 (sr)
JP (1) JP7389040B2 (sr)
KR (1) KR20200135768A (sr)
CN (1) CN112004544B (sr)
AU (1) AU2019209595B2 (sr)
BR (1) BR112020014625A2 (sr)
CA (1) CA3087910A1 (sr)
DK (1) DK3740222T5 (sr)
EA (1) EA202091712A1 (sr)
ES (1) ES2957622T3 (sr)
FI (1) FI3740222T3 (sr)
HR (1) HRP20231126T1 (sr)
HU (1) HUE062774T2 (sr)
IL (1) IL275838B2 (sr)
LT (1) LT3740222T (sr)
MX (1) MX2020006764A (sr)
PH (1) PH12020551096A1 (sr)
PL (1) PL3740222T3 (sr)
PT (1) PT3740222T (sr)
RS (1) RS64499B1 (sr)
SG (1) SG11202006056RA (sr)
SI (1) SI3740222T1 (sr)
SM (1) SMT202300284T1 (sr)
WO (1) WO2019141765A1 (sr)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3356390T3 (pl) 2015-09-28 2021-07-05 The University Of North Carolina At Chapel Hill Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał
CN111511375A (zh) * 2017-06-30 2020-08-07 因提玛生物科学公司 用于基因治疗的腺相关病毒载体
CA3094311A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ,インコーポレイテッド 抗体を回避するウイルスベクター
CN110437317B (zh) * 2019-01-30 2023-05-02 上海科技大学 具有变异衣壳蛋白的腺相关病毒及其用途
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
SMT202400486T1 (it) * 2019-07-15 2025-01-14 Meiragtx Uk Ii Ltd Proteine di capside avv modificate per il trattamento della malattia artritica
MX2022004352A (es) 2019-10-17 2022-07-19 Stridebio Inc Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c.
CN110950934B (zh) * 2019-12-31 2022-11-04 复旦大学 一种腺相关病毒衣壳蛋白、载体及其构建方法与应用
WO2021168509A1 (en) * 2020-02-25 2021-09-02 Children's Medical Research Institute Adeno-associated virus capsid polypeptides and vectors
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
EP4413147A1 (en) 2021-10-08 2024-08-14 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
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
WO2025049993A1 (en) 2023-08-31 2025-03-06 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (21)

* Cited by examiner, † Cited by third party
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
US7271002B2 (en) 2001-11-09 2007-09-18 United States Of America, Represented By The Secretary, Department Of Health And Human Services Production of adeno-associated virus in insect cells
JP4863874B2 (ja) * 2003-09-01 2012-01-25 アカデミッシュ メディッシュ セントラム 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
AU2010201278B2 (en) * 2003-09-01 2012-11-15 Academisch Medisch Centrum AAV vectors for in vivo gene therapy of rheumatoid arthritis
NZ545656A (en) * 2003-09-01 2009-02-28 Amc Amsterdam AAV vectors for in vivo gene therapy of rheumatoid arthritis
PL1945779T3 (pl) 2005-10-20 2013-08-30 Uniqure Ip Bv Ulepszone wektory AAV produkowane w komórkach owadzich
WO2007148971A2 (en) 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
WO2008024998A2 (en) 2006-08-24 2008-02-28 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
CN101868547B (zh) 2007-07-26 2016-03-16 尤尼克尔Ip股份有限公司 包含具有差别密码子偏性的重复编码序列的杆状病毒载体
AU2009215987B2 (en) 2008-02-19 2015-01-22 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
EP3792348A3 (en) 2010-03-11 2021-06-23 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
KR101961347B1 (ko) 2011-09-08 2019-03-25 유니큐어 아이피 비.브이. Aav 제조물로부터 오염 바이러스의 제거
WO2013174760A1 (en) * 2012-05-25 2013-11-28 Deutsches Krebsforschungszentrum Optimized aav-vectors for high transduction rates in dendritic cells
SG11201510757PA (en) * 2013-05-21 2016-01-28 Univ Florida Capsid-modified, raav3 vector compositions and uses in gene therapy of human liver cancer
DE102014207498A1 (de) * 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
CN104107438A (zh) * 2014-05-29 2014-10-22 中国人民解放军军事医学科学院基础医学研究所 Spry2在制备预防和治疗类风湿性关节炎药物中的应用
AU2015330092A1 (en) 2014-10-06 2017-04-13 Arthrogen B.V. AAV-based gene therapy
EP3233131A1 (en) * 2014-12-16 2017-10-25 Board of Regents of the University of Nebraska Gene therapy for juvenile batten disease
AU2018323049B2 (en) * 2017-08-31 2025-03-13 Benitec IP Holdings Inc. Adeno-associated virus (AAV) with modified phospholipase domain
SMT202400486T1 (it) * 2019-07-15 2025-01-14 Meiragtx Uk Ii Ltd Proteine di capside avv modificate per il trattamento della malattia artritica

Also Published As

Publication number Publication date
DK3740222T3 (da) 2023-07-31
CN112004544B (zh) 2024-08-06
PT3740222T (pt) 2023-07-11
SI3740222T1 (sl) 2023-11-30
CA3087910A1 (en) 2019-07-25
HRP20231126T1 (hr) 2024-01-05
EP3740222B1 (en) 2023-06-28
KR20200135768A (ko) 2020-12-03
WO2019141765A1 (en) 2019-07-25
PL3740222T3 (pl) 2023-12-04
LT3740222T (lt) 2023-09-11
JP7389040B2 (ja) 2023-11-29
NZ767106A (en) 2023-10-27
EP3740222A1 (en) 2020-11-25
IL275838A (en) 2020-08-31
FI3740222T3 (fi) 2023-07-26
SG11202006056RA (en) 2020-07-29
AU2019209595B2 (en) 2023-04-27
AU2019209595A1 (en) 2020-09-03
BR112020014625A2 (pt) 2020-12-08
CN112004544A (zh) 2020-11-27
HUE062774T2 (hu) 2023-12-28
MX2020006764A (es) 2021-01-08
IL275838B2 (en) 2024-11-01
IL275838B1 (en) 2024-07-01
DK3740222T5 (da) 2024-08-26
US20200354744A1 (en) 2020-11-12
PH12020551096A1 (en) 2021-06-21
SMT202300284T1 (it) 2023-11-13
ES2957622T3 (es) 2024-01-23
JP2021510528A (ja) 2021-04-30
EA202091712A1 (ru) 2021-02-16

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