MX2023012684A - Terapia con gen lentiviral in vivo para el tratamiento de hiperoxaluria primaria tipo 1. - Google Patents

Terapia con gen lentiviral in vivo para el tratamiento de hiperoxaluria primaria tipo 1.

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Publication number
MX2023012684A
MX2023012684A MX2023012684A MX2023012684A MX2023012684A MX 2023012684 A MX2023012684 A MX 2023012684A MX 2023012684 A MX2023012684 A MX 2023012684A MX 2023012684 A MX2023012684 A MX 2023012684A MX 2023012684 A MX2023012684 A MX 2023012684A
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MX
Mexico
Prior art keywords
ltr
nucleic acid
terminal repeat
long terminal
wpre
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MX2023012684A
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English (en)
Inventor
Sanz José Carlos Segovia
Bravo María García
Vicente Andrea Molinos
Torralba Aida García
Romero Virginia Nieto
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Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P
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Publication of MX2023012684A publication Critical patent/MX2023012684A/es

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    • 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
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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)
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    • 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)
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    • 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
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    • A01K2267/0306—Animal model for genetic diseases
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    • 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
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    • 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
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    • 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
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    • 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)
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  • Biochemistry (AREA)
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  • 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)
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  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente invención se refiere a un vector de lentivirus (LV) que comprende un ácido nucleico, en el cual el ácido nucleico comprende una unidad de transcripción la cual comprende, de 5' a 3', un promotor específico de hepatocito, de preferencia un promotor ET, una secuencia de nucleótido que codifica para la proteína de alanina-glioxilato y serina-piruvato aminotransferasa (AGXT-RHEAM), un elemento regulador post-transcripción del virus de la hepatitis de la marmota (Marmota monax) (WPRE), de preferencia un WPRE mutado, y por lo menos una copia de por lo menos una secuencia objetivo de miRNA, de preferencia miRNA-142. El vector lentiviral de la presente invención comprende también los elementos de estructura base necesarios para que el LV lleve a cabo su función, los cuales son por lo menos uno de los siguientes: una repetición terminal larga 5' (5' LTR), un sitio de unión a indiciador (PBS), una señal de empaquetamiento psi, la región tallo-asa 4 (SL4) del virus de VIH de tipo silvestre, un elemento de responsivo a Rev (RRE), y un tracto de polipurina central (cPPT) de solapa de ADN, y una repetición terminal larga 3' (3' LTR), o cualquier combinación de los mismos.
MX2023012684A 2021-04-26 2022-04-26 Terapia con gen lentiviral in vivo para el tratamiento de hiperoxaluria primaria tipo 1. MX2023012684A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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
MX2023012684A true MX2023012684A (es) 2024-02-15

Family

ID=75746556

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023012684A MX2023012684A (es) 2021-04-26 2022-04-26 Terapia con gen lentiviral in vivo para el tratamiento de hiperoxaluria primaria tipo 1.

Country Status (8)

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

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2750811C (en) * 2009-01-30 2017-08-29 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Alanine-glyoxylate aminotransferase therapeutics
EP3548617A4 (en) * 2016-12-05 2020-06-17 The Regents of The University of California LENTIVIRAL VECTOR OPTIMIZED FOR HEMOGLOBINOPATHY STEM CELL THERAPY
AU2019215063A1 (en) * 2018-02-01 2020-09-03 Bioverativ Therapeutics, Inc. Use of lentiviral vectors expressing Factor VIII

Also Published As

Publication number Publication date
WO2022229223A1 (en) 2022-11-03
JP2024516655A (ja) 2024-04-16
CA3218031A1 (en) 2022-11-03
KR20240004627A (ko) 2024-01-11
CN117561336A (zh) 2024-02-13
EP4083217A1 (en) 2022-11-02
EP4330415A1 (en) 2024-03-06
AU2022266057A1 (en) 2023-11-16

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