JP2022536338A - Ａｒｓａ遺伝子移入のためのアデノ随伴ウイルス組成物およびその使用方法 - Google Patents

Ａｒｓａ遺伝子移入のためのアデノ随伴ウイルス組成物およびその使用方法 Download PDF

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JP2022536338A
JP2022536338A JP2021573242A JP2021573242A JP2022536338A JP 2022536338 A JP2022536338 A JP 2022536338A JP 2021573242 A JP2021573242 A JP 2021573242A JP 2021573242 A JP2021573242 A JP 2021573242A JP 2022536338 A JP2022536338 A JP 2022536338A
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amino acid
seq
capsid protein
protein corresponding
sequence
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JPWO2020251954A5 (https=
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ティア・バボヴァル・セント・マーティン
アルバート・バーンズ・シーモア
ヒラード・ルビン
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ホモロジー・メディシンズ・インコーポレイテッド
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    • 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
    • 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
    • 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
    • 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
    • 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)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Neurosurgery (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
JP2021573242A 2019-06-10 2020-06-09 Ａｒｓａ遺伝子移入のためのアデノ随伴ウイルス組成物およびその使用方法 Pending JP2022536338A (ja)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201962859539P 2019-06-10 2019-06-10
US62/859,539 2019-06-10
US201962866374P 2019-06-25 2019-06-25
US62/866,374 2019-06-25
US201962915523P 2019-10-15 2019-10-15
US62/915,523 2019-10-15
US202062960487P 2020-01-13 2020-01-13
US62/960,487 2020-01-13
US202062987858P 2020-03-10 2020-03-10
US62/987,858 2020-03-10
US202063010970P 2020-04-16 2020-04-16
US63/010,970 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 (3)

Publication Number Publication Date
JP2022536338A true JP2022536338A (ja) 2022-08-15
JPWO2020251954A5 JPWO2020251954A5 (https=) 2023-06-05
JP2022536338A5 JP2022536338A5 (https=) 2023-06-05

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JP2021573242A Pending JP2022536338A (ja) 2019-06-10 2020-06-09 Ａｒｓａ遺伝子移入のためのアデノ随伴ウイルス組成物およびその使用方法

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

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2769577C1 (ru) * 2021-06-01 2022-04-04 федеральное государственное автономное образовательное учреждение высшего образования "Казанский (Приволжский) федеральный университет" (ФГАОУ ВО КФУ) Препарат для лечения метахроматической лейкодистрофии и способ ее лечения
AU2023273809A1 (en) * 2022-05-16 2025-01-02 Genzyme Corporation Methods of treating metachromatic leukodystrophy
JP2025525792A (ja) 2022-07-29 2025-08-07 リジェネロン・ファーマシューティカルズ・インコーポレイテッド トランスフェリン受容体１に対して再標的化されるウイルス粒子
WO2024036250A2 (en) * 2022-08-10 2024-02-15 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20180021364A1 (en) * 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US20180071373A1 (en) * 2015-05-15 2018-03-15 Regents Of The University Of Minnesota Adeno-associated virus for therapeutic delivery to central nervous system
US20180163227A1 (en) * 2010-04-30 2018-06-14 City Of Hope Recombinant adeno-associated vectors for targeted treatment
JP2018522546A (ja) * 2015-06-18 2018-08-16 ザ・ブロード・インスティテュート・インコーポレイテッド オフターゲット効果を低下させるｃｒｉｓｐｒ酵素突然変異

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Publication number Priority date Publication date Assignee Title
EP2634253B1 (en) * 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
IL248102B (en) * 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
JP6836999B2 (ja) * 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
KR102528641B1 (ko) * 2018-02-01 2023-05-03 호몰로지 메디슨, 인크. Pah 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
US20220127625A1 (en) * 2019-02-15 2022-04-28 Generation Bio Co. Modulation of rep protein activity in closed-ended dna (cedna) production
BR112021021908A2 (pt) * 2019-05-03 2022-02-01 Univ Pennsylvania Composições úteis no tratamento da leucodistrofia metacromática

Patent Citations (4)

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US20180163227A1 (en) * 2010-04-30 2018-06-14 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US20180021364A1 (en) * 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US20180071373A1 (en) * 2015-05-15 2018-03-15 Regents Of The University Of Minnesota Adeno-associated virus for therapeutic delivery to central nervous system
JP2018522546A (ja) * 2015-06-18 2018-08-16 ザ・ブロード・インスティテュート・インコーポレイテッド オフターゲット効果を低下させるｃｒｉｓｐｒ酵素突然変異

Non-Patent Citations (2)

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KURAI, T., ET AL.: ""AAV1 mediated co-expression of formylglycine-generating enzyme and arylsulfatase A efficiently corr", MOLECULAR THERAPY, vol. 15, no. 1, JPN6024018398, January 2007 (2007-01-01), pages 38 - 43, ISSN: 0005484297 *
YAMAZAKI, Y., ET AL.: ""Targeted gene transfer into ependymal cells through intraventricular injection of AAV1 vector and l", SCIENTIFIC REPORTS, vol. 4, JPN6024018397, 1 July 2014 (2014-07-01), pages 1 - 7, ISSN: 0005484296 *

Also Published As

Publication number Publication date
MX2021015076A (es) 2022-06-02
WO2020251954A1 (en) 2020-12-17
EP3980447A1 (en) 2022-04-13
TW202112807A (zh) 2021-04-01
AU2020292256B2 (en) 2023-01-19
IL288863A (en) 2022-02-01
EP3980447A4 (en) 2023-07-26
AU2020292256A1 (en) 2022-01-06
PE20220233A1 (es) 2022-02-07
BR112021024855A2 (pt) 2022-05-03
KR20220035107A (ko) 2022-03-21
CL2021003295A1 (es) 2022-09-23
US20220204991A1 (en) 2022-06-30
CO2021016797A2 (es) 2022-01-17
CN114502575A (zh) 2022-05-13
CA3142932A1 (en) 2020-12-17

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