JP2022519597A - Ｃｌｎ６ポリヌクレオチドのアデノ随伴ウイルス送達 - Google Patents

Ｃｌｎ６ポリヌクレオチドのアデノ随伴ウイルス送達 Download PDF

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JP2022519597A
JP2022519597A JP2021545402A JP2021545402A JP2022519597A JP 2022519597 A JP2022519597 A JP 2022519597A JP 2021545402 A JP2021545402 A JP 2021545402A JP 2021545402 A JP2021545402 A JP 2021545402A JP 2022519597 A JP2022519597 A JP 2022519597A
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nucleic acid
cln6
seq
acid sequence
scaav9
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JP2022519597A5 (https=
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キャスリン マイヤー，
ブライアン ケー． カスパー，
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リサーチ・インスティチュート・アット・ネーションワイド・チルドレンズ・ホスピタル
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Publication of JP2022519597A publication Critical patent/JP2022519597A/ja
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    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (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)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2021545402A 2019-02-04 2020-02-04 Ｃｌｎ６ポリヌクレオチドのアデノ随伴ウイルス送達 Pending JP2022519597A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025023029A JP2025093922A (ja) 2019-02-04 2025-02-17 Ｃｌｎ６ポリヌクレオチドのアデノ随伴ウイルス送達

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201962800915P 2019-02-04 2019-02-04
US62/800,915 2019-02-04
US201962880641P 2019-07-30 2019-07-30
US62/880,641 2019-07-30
US201962881151P 2019-07-31 2019-07-31
US62/881,151 2019-07-31
US201962912977P 2019-10-09 2019-10-09
US62/912,977 2019-10-09
US201962923125P 2019-10-18 2019-10-18
US62/923,125 2019-10-18
PCT/US2020/016541 WO2020163299A1 (en) 2019-02-04 2020-02-04 Adeno-associated virus delivery of cln6 polynucleotide

Related Child Applications (1)

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JP2025023029A Division JP2025093922A (ja) 2019-02-04 2025-02-17 Ｃｌｎ６ポリヌクレオチドのアデノ随伴ウイルス送達

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JP2022519597A true JP2022519597A (ja) 2022-03-24
JP2022519597A5 JP2022519597A5 (https=) 2023-02-10

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JP2025023029A Pending JP2025093922A (ja) 2019-02-04 2025-02-17 Ｃｌｎ６ポリヌクレオチドのアデノ随伴ウイルス送達

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US (1) US20220202956A1 (https=)
EP (1) EP3921429A1 (https=)
JP (2) JP2022519597A (https=)
KR (1) KR20210124299A (https=)
CN (2) CN113574176B (https=)
AU (1) AU2020218501B2 (https=)
BR (1) BR112021015150A2 (https=)
CA (1) CA3127801A1 (https=)
CL (1) CL2021002051A1 (https=)
IL (1) IL285329A (https=)
MX (1) MX2021009404A (https=)
SG (1) SG11202107983TA (https=)
TW (1) TW202033768A (https=)
WO (1) WO2020163299A1 (https=)

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* Cited by examiner, † Cited by third party
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AU2013296425B2 (en) 2012-08-01 2018-06-07 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
CA3137080A1 (en) * 2019-04-15 2020-10-22 Sanford Research Gene therapy for treating or preventing visual effects in batten disease
WO2023018674A1 (en) * 2021-08-09 2023-02-16 Amicus Therapeutics, Inc. Determination of gene transduction potency in neuron-like cells
WO2026039311A1 (en) 2024-08-10 2026-02-19 Vectorgen Lentiviral vector for treating human immunodeficiency virus infection

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JPH09103296A (ja) * 1986-09-12 1997-04-22 Genentech Inc 安定化配列を有するベクターおよび真核宿主細胞
JPH10507633A (ja) * 1994-10-21 1998-07-28 フアーマシア・アンド・アツプジヨン・アー・ベー 組換え蛋白質生産用発現ベクターの改良
WO2002066638A1 (fr) * 2001-02-22 2002-08-29 Gencom Corporation Gene recombinant contenant une sequence de repetition inversee et utilisation correspondante
JP2015525783A (ja) * 2012-08-01 2015-09-07 ネイションワイド チルドレンズ ホスピタル 組換えアデノ随伴ウイルス９の髄腔内送達
WO2017136202A1 (en) * 2016-02-05 2017-08-10 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
JP2018500311A (ja) * 2014-12-16 2018-01-11 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法

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WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
JP2001500497A (ja) 1996-09-06 2001-01-16 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法
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DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
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JPH10507633A (ja) * 1994-10-21 1998-07-28 フアーマシア・アンド・アツプジヨン・アー・ベー 組換え蛋白質生産用発現ベクターの改良
WO2002066638A1 (fr) * 2001-02-22 2002-08-29 Gencom Corporation Gene recombinant contenant une sequence de repetition inversee et utilisation correspondante
JP2015525783A (ja) * 2012-08-01 2015-09-07 ネイションワイド チルドレンズ ホスピタル 組換えアデノ随伴ウイルス９の髄腔内送達
JP2018500311A (ja) * 2014-12-16 2018-01-11 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
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Publication number Publication date
CA3127801A1 (en) 2020-08-13
IL285329A (en) 2021-09-30
TW202033768A (zh) 2020-09-16
EP3921429A1 (en) 2021-12-15
US20220202956A1 (en) 2022-06-30
AU2020218501A1 (en) 2021-08-19
AU2020218501B2 (en) 2026-01-29
CL2021002051A1 (es) 2022-04-08
SG11202107983TA (en) 2021-08-30
CN113574176B (zh) 2025-06-06
MX2021009404A (es) 2021-11-12
JP2025093922A (ja) 2025-06-24
BR112021015150A2 (pt) 2021-09-28
CN113574176A (zh) 2021-10-29
CN120505372A (zh) 2025-08-19
WO2020163299A1 (en) 2020-08-13
KR20210124299A (ko) 2021-10-14

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