KR20240004627A - 1형 원발성 고옥살산뇨증의 치료를 위한 생체내 렌티바이러스 유전자 치료 - Google Patents

1형 원발성 고옥살산뇨증의 치료를 위한 생체내 렌티바이러스 유전자 치료 Download PDF

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Publication number
KR20240004627A
KR20240004627A KR1020237040673A KR20237040673A KR20240004627A KR 20240004627 A KR20240004627 A KR 20240004627A KR 1020237040673 A KR1020237040673 A KR 1020237040673A KR 20237040673 A KR20237040673 A KR 20237040673A KR 20240004627 A KR20240004627 A KR 20240004627A
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South Korea
Prior art keywords
seq
lentiviral vector
sequence
agxt
full length
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Pending
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KR1020237040673A
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English (en)
Korean (ko)
Inventor
산츠 호세 카를로스 세고비아
브라보 마리아 가르시아
비센테 안드레아 몰리노스
토랄바 아이다 가르시아
로메로 비르히니아 니에토
Original Assignee
센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피.
콘소르시오 센트로 데 인베스티카시온 비오메디카 엔 레드
푼다시안 인스티튜토 데 인베스티카시온 사니타리아 푼다시안 시메네스 디아즈
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Application filed by 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피., 콘소르시오 센트로 데 인베스티카시온 비오메디카 엔 레드, 푼다시안 인스티튜토 데 인베스티카시온 사니타리아 푼다시안 시메네스 디아즈 filed Critical 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피.
Publication of KR20240004627A publication Critical patent/KR20240004627A/ko
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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

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  • 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)
KR1020237040673A 2021-04-26 2022-04-26 1형 원발성 고옥살산뇨증의 치료를 위한 생체내 렌티바이러스 유전자 치료 Pending KR20240004627A (ko)

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
KR20240004627A true KR20240004627A (ko) 2024-01-11

Family

ID=75746556

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237040673A Pending KR20240004627A (ko) 2021-04-26 2022-04-26 1형 원발성 고옥살산뇨증의 치료를 위한 생체내 렌티바이러스 유전자 치료

Country Status (8)

Country Link
EP (2) EP4083217A1 (cg-RX-API-DMAC7.html)
JP (1) JP2024516655A (cg-RX-API-DMAC7.html)
KR (1) KR20240004627A (cg-RX-API-DMAC7.html)
CN (1) CN117561336A (cg-RX-API-DMAC7.html)
AU (1) AU2022266057A1 (cg-RX-API-DMAC7.html)
CA (1) CA3218031A1 (cg-RX-API-DMAC7.html)
MX (1) MX2023012684A (cg-RX-API-DMAC7.html)
WO (1) WO2022229223A1 (cg-RX-API-DMAC7.html)

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
CN117561336A (zh) 2024-02-13
MX2023012684A (es) 2024-02-15
AU2022266057A1 (en) 2023-11-16

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Patent event date: 20231124

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Comment text: International Patent Application

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Patent event code: PA02012R01D

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