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BR112021024855A2 - Composições de vírus adenoassociados para transferência de gene arsa e métodos de uso das mesmas - Google Patents

Composições de vírus adenoassociados para transferência de gene arsa e métodos de uso das mesmas

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Publication number
BR112021024855A2
BR112021024855A2 BR112021024855A BR112021024855A BR112021024855A2 BR 112021024855 A2 BR112021024855 A2 BR 112021024855A2 BR 112021024855 A BR112021024855 A BR 112021024855A BR 112021024855 A BR112021024855 A BR 112021024855A BR 112021024855 A2 BR112021024855 A2 BR 112021024855A2
Authority
BR
Brazil
Prior art keywords
compositions
adeno
methods
gene transfer
arsa
Prior art date
Application number
BR112021024855A
Other languages
English (en)
Inventor
Barnes Seymour Albert
Hillard Rubin
Baboval St Martin Thia
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 BR112021024855A2 publication Critical patent/BR112021024855A2/pt

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    • 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
    • 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
    • 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)

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  • 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)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Neurosurgery (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Psychiatry (AREA)
  • Environmental Sciences (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (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

composições de vírus adenoassociados para transferência de gene arsa e métodos de uso das mesmas. a presente invenção refere-se a composições de vírus adenoassociados (aav) que podem expressar um polipeptídeo arilsulfatase a (arsa) em uma célula, restaurando assim a função do gene arsa. também são fornecidos métodos de uso das composições de aav e sistemas de empacotamento para produzir as composições de aav.
BR112021024855A 2019-06-10 2020-06-09 Composições de vírus adenoassociados para transferência de gene arsa e métodos de uso das mesmas BR112021024855A2 (pt)

Applications Claiming Priority (7)

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
PCT/US2020/036846 WO2020251954A1 (en) 2019-06-10 2020-06-09 Adeno-associated virus compositions for arsa gene transfer and methods of use thereof

Publications (1)

Publication Number Publication Date
BR112021024855A2 true BR112021024855A2 (pt) 2022-05-03

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021024855A BR112021024855A2 (pt) 2019-06-10 2020-06-09 Composições de vírus adenoassociados para transferência de gene arsa e métodos de uso das mesmas

Country Status (15)

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

Families Citing this family (3)

* 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
WO2024036250A2 (en) * 2022-08-10 2024-02-15 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof

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
CN103189507A (zh) * 2010-10-27 2013-07-03 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
EA034355B1 (ru) * 2014-05-02 2020-01-30 Джензим Корпорейшн Векторы на основе aav для генной терапии в сетчатке и cns
WO2016115503A1 (en) * 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
AU2016235163B2 (en) * 2015-03-24 2022-03-24 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
RU2751952C2 (ru) * 2015-05-15 2021-07-21 Реджентс Оф Зэ Юниверсити Оф Миннесота Вектор на основе аденоассоциированного вируса для терапевтической доставки в центральную нервную систему
EA202091844A1 (ru) * 2018-02-01 2021-02-03 Хомолоджи Медисинс, Инк. Композиции аденоассоциированных вирусов для переноса гена pah и способы их применения
CN113454232B (zh) * 2019-02-15 2024-10-29 世代生物公司 封闭端dna（cedna）产生中对rep蛋白活性的调节
PE20220930A1 (es) * 2019-05-03 2022-05-31 Univ Pennsylvania Composiciones utiles en el tratamiento de leucodistrofia metacromatica

Also Published As

Publication number Publication date
AU2020292256B2 (en) 2023-01-19
KR20220035107A (ko) 2022-03-21
MX2021015076A (es) 2022-06-02
CO2021016797A2 (es) 2022-01-17
TW202112807A (zh) 2021-04-01
IL288863A (en) 2022-02-01
CA3142932A1 (en) 2020-12-17
CN114502575A (zh) 2022-05-13
PE20220233A1 (es) 2022-02-07
AU2020292256A1 (en) 2022-01-06
CL2021003295A1 (es) 2022-09-23
EP3980447A1 (en) 2022-04-13
US20220204991A1 (en) 2022-06-30
WO2020251954A1 (en) 2020-12-17
JP2022536338A (ja) 2022-08-15
EP3980447A4 (en) 2023-07-26

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