CL2021003295A1 - Composiciones de virus adenoasociados para la transferencia del gen de arsa y métodos de uso de las mismas. - Google Patents

Composiciones de virus adenoasociados para la transferencia del gen de arsa y métodos de uso de las mismas.

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Publication number
CL2021003295A1
CL2021003295A1 CL2021003295A CL2021003295A CL2021003295A1 CL 2021003295 A1 CL2021003295 A1 CL 2021003295A1 CL 2021003295 A CL2021003295 A CL 2021003295A CL 2021003295 A CL2021003295 A CL 2021003295A CL 2021003295 A1 CL2021003295 A1 CL 2021003295A1
Authority
CL
Chile
Prior art keywords
adeno
methods
associated virus
gene transfer
virus compositions
Prior art date
Application number
CL2021003295A
Other languages
English (en)
Inventor
Albert Barnes Seymour
Hillard Rubin
Martin Thia Baboval St
Original Assignee
Homology Medicines Inc
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 Homology Medicines Inc filed Critical Homology Medicines Inc
Publication of CL2021003295A1 publication Critical patent/CL2021003295A1/es

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Classifications

    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • 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
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06001—Arylsulfatase (3.1.6.1)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Environmental Sciences (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

En la presente descripción se proporcionan composiciones de virus adenoasociados (AAV) que pueden expresar un polipéptido de arilsulfatasa A (ARSA) en una célula, para restablecer de ese modo la función del gen de ARSA. También se proporcionan métodos para usar las composiciones de AAV y sistemas de empaquetamiento para preparar las composiciones de AAV.
CL2021003295A 2019-06-10 2021-12-10 Composiciones de virus adenoasociados para la transferencia del gen de arsa y métodos de uso de las mismas. CL2021003295A1 (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201962859539P 2019-06-10 2019-06-10
US201962866374P 2019-06-25 2019-06-25
US201962915523P 2019-10-15 2019-10-15
US202062960487P 2020-01-13 2020-01-13
US202062987858P 2020-03-10 2020-03-10
US202063010970P 2020-04-16 2020-04-16

Publications (1)

Publication Number Publication Date
CL2021003295A1 true CL2021003295A1 (es) 2022-09-23

Family

ID=73782242

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2021003295A CL2021003295A1 (es) 2019-06-10 2021-12-10 Composiciones de virus adenoasociados para la transferencia del gen de arsa y métodos de uso de las mismas.

Country Status (15)

Country Link
US (1) US20220204991A1 (es)
EP (1) EP3980447A4 (es)
JP (1) JP2022536338A (es)
KR (1) KR20220035107A (es)
CN (1) CN114502575A (es)
AU (1) AU2020292256B2 (es)
BR (1) BR112021024855A2 (es)
CA (1) CA3142932A1 (es)
CL (1) CL2021003295A1 (es)
CO (1) CO2021016797A2 (es)
IL (1) IL288863A (es)
MX (1) MX2021015076A (es)
PE (1) PE20220233A1 (es)
TW (1) TW202112807A (es)
WO (1) WO2020251954A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230398192A1 (en) * 2022-05-16 2023-12-14 Genzyme Corporation Methods of treating metachromatic leukodystrophy
WO2024026494A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Viral particles retargeted to transferrin receptor 1

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9839696B2 (en) * 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
JP5704361B2 (ja) * 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
ES2879636T3 (es) * 2014-05-02 2021-11-22 Genzyme Corp Vectores AAV para la terapia génica de la retina y el SNC
US20180021364A1 (en) * 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
EP3277819B1 (en) * 2015-03-24 2021-03-03 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
SI3294323T1 (sl) * 2015-05-15 2022-06-30 Regenxbio Inc. Adenoasociacijski virus za terapevtsko dostavo v centralni živčni sistem
EP3746113A4 (en) * 2018-02-01 2022-03-09 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR PAH GENE TRANSFER AND METHODS OF USE
US20220127625A1 (en) * 2019-02-15 2022-04-28 Generation Bio Co. Modulation of rep protein activity in closed-ended dna (cedna) production
CA3135539A1 (en) * 2019-05-03 2020-11-12 Juliette HORDEAUX Compositions useful in treatment of metachromatic leukodystrophy

Also Published As

Publication number Publication date
MX2021015076A (es) 2022-06-02
AU2020292256B2 (en) 2023-01-19
EP3980447A1 (en) 2022-04-13
PE20220233A1 (es) 2022-02-07
US20220204991A1 (en) 2022-06-30
BR112021024855A2 (pt) 2022-05-03
TW202112807A (zh) 2021-04-01
CA3142932A1 (en) 2020-12-17
CO2021016797A2 (es) 2022-01-17
KR20220035107A (ko) 2022-03-21
EP3980447A4 (en) 2023-07-26
JP2022536338A (ja) 2022-08-15
AU2020292256A1 (en) 2022-01-06
IL288863A (en) 2022-02-01
WO2020251954A1 (en) 2020-12-17
CN114502575A (zh) 2022-05-13

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