CN117561336A - 治疗原发性高草酸尿症1型的体内慢病毒基因疗法 - Google Patents

治疗原发性高草酸尿症1型的体内慢病毒基因疗法 Download PDF

Info

Publication number
CN117561336A
CN117561336A CN202280045601.7A CN202280045601A CN117561336A CN 117561336 A CN117561336 A CN 117561336A CN 202280045601 A CN202280045601 A CN 202280045601A CN 117561336 A CN117561336 A CN 117561336A
Authority
CN
China
Prior art keywords
seq
lentiviral vector
sequence
mice
full length
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.)
Pending
Application number
CN202280045601.7A
Other languages
English (en)
Chinese (zh)
Inventor
J·C·塞戈比亚·桑斯
M·加西亚·布拉沃
A·莫利诺·维森特
A·加西亚·托拉尔巴
V·尼托·罗梅罗
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.)
Biomedical Network Research Consortium Center
Himenez Diaz Health Research Foundation Institute
Energy Environment And Technology Research Center Oamp
Original Assignee
Biomedical Network Research Consortium Center
Himenez Diaz Health Research Foundation Institute
Energy Environment And Technology Research Center Oamp
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 Biomedical Network Research Consortium Center, Himenez Diaz Health Research Foundation Institute, Energy Environment And Technology Research Center Oamp filed Critical Biomedical Network Research Consortium Center
Publication of CN117561336A publication Critical patent/CN117561336A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1096—Transferases (2.) transferring nitrogenous groups (2.6)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y206/00—Transferases transferring nitrogenous groups (2.6)
    • C12Y206/01—Transaminases (2.6.1)
    • C12Y206/01044—Alanine--glyoxylate transaminase (2.6.1.44)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y206/00—Transferases transferring nitrogenous groups (2.6)
    • C12Y206/01—Transaminases (2.6.1)
    • C12Y206/01051—Serine--pyruvate transaminase (2.6.1.51)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202280045601.7A 2021-04-26 2022-04-26 治疗原发性高草酸尿症1型的体内慢病毒基因疗法 Pending CN117561336A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21382363.6 2021-04-26
EP21382363.6A EP4083217A1 (en) 2021-04-26 2021-04-26 In vivo lentiviral gene therapy for the treatment of primary hyperoxaluria type 1
PCT/EP2022/061107 WO2022229223A1 (en) 2021-04-26 2022-04-26 In vivo lentiviral gene therapy for the treatment of primary hyperoxaluria type 1

Publications (1)

Publication Number Publication Date
CN117561336A true CN117561336A (zh) 2024-02-13

Family

ID=75746556

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202280045601.7A Pending CN117561336A (zh) 2021-04-26 2022-04-26 治疗原发性高草酸尿症1型的体内慢病毒基因疗法

Country Status (8)

Country Link
EP (2) EP4083217A1 (enExample)
JP (1) JP2024516655A (enExample)
KR (1) KR20240004627A (enExample)
CN (1) CN117561336A (enExample)
AU (1) AU2022266057A1 (enExample)
CA (1) CA3218031A1 (enExample)
MX (1) MX2023012684A (enExample)
WO (1) WO2022229223A1 (enExample)

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2384200B1 (en) * 2009-01-30 2013-07-10 UniQure IP B.V. Alanine-glyoxylate aminotransferase therapeutics
WO2015059701A1 (en) * 2013-10-24 2015-04-30 Medgenics Medical Israel Ltd. Micro-organs providing sustained delivery of a therapeutic polypeptide and methods of use thereof
US11008561B2 (en) * 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
US11976293B2 (en) * 2016-12-05 2024-05-07 The Regents Of The University Of California Optimized lentiviral vector for stem cell gene therapy of hemoglobinopathies
US20210038744A1 (en) * 2018-02-01 2021-02-11 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor viii

Also Published As

Publication number Publication date
EP4330415A1 (en) 2024-03-06
WO2022229223A1 (en) 2022-11-03
JP2024516655A (ja) 2024-04-16
EP4083217A1 (en) 2022-11-02
CA3218031A1 (en) 2022-11-03
KR20240004627A (ko) 2024-01-11
MX2023012684A (es) 2024-02-15
AU2022266057A1 (en) 2023-11-16

Similar Documents

Publication Publication Date Title
US20240141381A1 (en) Compositions and methods for treating phenylketonuria
US20220162643A1 (en) Optimized phenylananine hydroxylase expression
JP7838779B2 (ja) フェニルケトン尿症を処置するための、プロモーターとエンハンサーの組み合わせを有するベクター
Laughrea et al. Mutations in the kissing-loop hairpin of human immunodeficiency virus type 1 reduce viral infectivity as well as genomic RNA packaging and dimerization
Aronoff et al. Specificity of retroviral RNA packaging
McBride et al. Position dependence of functional hairpins important for human immunodeficiency virus type 1 RNA encapsidation in vivo
JP7176756B2 (ja) パーキンソン病を処置するためのウイルスベクター
CA3217247A1 (en) Lentiviral vectors useful for the treatment of disease
Hajjar et al. A model system for nonhomologous recombination between retroviral and cellular RNA
Mautino et al. Modified human immunodeficiency virus-based lentiviral vectors display decreased sensitivity to trans-dominant Rev
KR20240004627A (ko) 1형 원발성 고옥살산뇨증의 치료를 위한 생체내 렌티바이러스 유전자 치료
Anderson et al. Comparison of the transcriptional activity of the long terminal repeats of simian immunodeficiency viruses SIVmac251 and SIVmac239 in T-cell lines and macrophage cell lines
KR101390967B1 (ko) 안전성이 향상된 레트로바이러스 벡터
US20060233757A1 (en) Vectors with viral insulators
CN121241146A (zh) 逆转录病毒载体
TW202409289A (zh) 用於治療肌強直性營養不良之組合物及方法
CA2208946A1 (en) Attenuated human immunodeficiency virus vaccine
EP4132590A1 (en) Modified vectors for production of retrovirus
WO2005032561A1 (ja) ＨＩＶ−Ｖｐｒの機能に関する発明
Element 827. Integration and Biodistribution Studies of Retroviral Vector in Mouse Gonad Tissues
JP2024516655A5 (enExample)

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