JP7691367B2 - Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達 - Google Patents

Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達 Download PDF

Info

Publication number
JP7691367B2
JP7691367B2 JP2021545401A JP2021545401A JP7691367B2 JP 7691367 B2 JP7691367 B2 JP 7691367B2 JP 2021545401 A JP2021545401 A JP 2021545401A JP 2021545401 A JP2021545401 A JP 2021545401A JP 7691367 B2 JP7691367 B2 JP 7691367B2
Authority
JP
Japan
Prior art keywords
cln3
scaav9
mice
months
composition
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2021545401A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022519596A (ja
JPWO2020163300A5 (enExample
Inventor
キャスリン マイヤー，
ブライアン ケー． カスパー，
ケビン ファウスト，
Original Assignee
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
オハイオ ステート イノベーション ファウンデーション
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 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, オハイオ ステート イノベーション ファウンデーション filed Critical 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
Publication of JP2022519596A publication Critical patent/JP2022519596A/ja
Publication of JPWO2020163300A5 publication Critical patent/JPWO2020163300A5/ja
Priority to JP2025090921A priority Critical patent/JP2025138648A/ja
Application granted granted Critical
Publication of JP7691367B2 publication Critical patent/JP7691367B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2021545401A 2019-02-04 2020-02-04 Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達 Active JP7691367B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025090921A JP2025138648A (ja) 2019-02-04 2025-05-30 Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962800911P 2019-02-04 2019-02-04
US62/800,911 2019-02-04
PCT/US2020/016542 WO2020163300A1 (en) 2019-02-04 2020-02-04 Adeno-associated virus delivery of cln3 polynucleotide

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025090921A Division JP2025138648A (ja) 2019-02-04 2025-05-30 Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達

Publications (3)

Publication Number Publication Date
JP2022519596A JP2022519596A (ja) 2022-03-24
JPWO2020163300A5 JPWO2020163300A5 (enExample) 2023-02-10
JP7691367B2 true JP7691367B2 (ja) 2025-06-11

Family

ID=69771089

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021545401A Active JP7691367B2 (ja) 2019-02-04 2020-02-04 Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達
JP2025090921A Pending JP2025138648A (ja) 2019-02-04 2025-05-30 Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025090921A Pending JP2025138648A (ja) 2019-02-04 2025-05-30 Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達

Country Status (16)

Country Link
US (1) US12404523B2 (enExample)
EP (1) EP3921430A1 (enExample)
JP (2) JP7691367B2 (enExample)
KR (1) KR20210124300A (enExample)
CN (1) CN113646436A (enExample)
AR (1) AR118005A1 (enExample)
AU (1) AU2020217708A1 (enExample)
BR (1) BR112021015285A2 (enExample)
CA (1) CA3129077A1 (enExample)
CL (1) CL2021002049A1 (enExample)
EA (1) EA202192160A1 (enExample)
IL (1) IL285328A (enExample)
MX (1) MX2021009401A (enExample)
SG (1) SG11202107986YA (enExample)
TW (1) TW202045530A (enExample)
WO (1) WO2020163300A1 (enExample)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2879719B1 (en) 2012-08-01 2018-07-04 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
WO2022170038A1 (en) * 2021-02-05 2022-08-11 Amicus Therapeutics, Inc. Adeno-associated virus delivery of cln3 polynucleotide
JP2025524569A (ja) * 2022-07-06 2025-07-30 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｃｌｎ１ポリヌクレオチドのアデノ随伴ウイルス送達

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2018094251A1 (en) 2016-11-17 2018-05-24 Kaspar Brian K Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
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
CA2230758A1 (en) 1995-09-08 1997-03-13 Genzyme Corporation Improved 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
CA2264483C (en) 1996-09-06 2011-03-22 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP1944362B1 (en) 1997-09-05 2015-10-21 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant AAV vectors
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・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 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
EP2879719B1 (en) * 2012-08-01 2018-07-04 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2018094251A1 (en) 2016-11-17 2018-05-24 Kaspar Brian K Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2

Also Published As

Publication number Publication date
EP3921430A1 (en) 2021-12-15
EA202192160A1 (ru) 2021-11-17
US12404523B2 (en) 2025-09-02
CL2021002049A1 (es) 2022-04-01
US20220127641A1 (en) 2022-04-28
MX2021009401A (es) 2021-11-12
SG11202107986YA (en) 2021-08-30
KR20210124300A (ko) 2021-10-14
JP2025138648A (ja) 2025-09-25
WO2020163300A9 (en) 2021-04-15
AR118005A1 (es) 2021-09-08
WO2020163300A1 (en) 2020-08-13
IL285328A (en) 2021-09-30
JP2022519596A (ja) 2022-03-24
CA3129077A1 (en) 2020-08-13
CN113646436A (zh) 2021-11-12
AU2020217708A1 (en) 2021-08-19
BR112021015285A2 (pt) 2021-10-05
TW202045530A (zh) 2020-12-16

Similar Documents

Publication Publication Date Title
JP2025138648A (ja) Ｃｌｎ３ポリヌクレオチドのアデノ随伴ウイルス送達
WO2019227219A1 (en) Adeno-associated viral vector-mediated gene therapy for treating fragile x-associated disorders
JP2025093922A (ja) Ｃｌｎ６ポリヌクレオチドのアデノ随伴ウイルス送達
EP3541429A1 (en) Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2
JP2024543253A (ja) Ｓｌｃ６ａ１遺伝子治療のための材料及び方法
AU2020278960A1 (en) Optimized gene therapy targeting retinal cells
US20250382593A1 (en) Adeno-Associated Virus Delivery of CLN1 Polynucleotide
WO2022170038A1 (en) Adeno-associated virus delivery of cln3 polynucleotide
US20240189452A1 (en) Recombinant Adeno-Associated Virus Encoding Methyl-CPG Binding Protein 2 for Treating PITT Hopkins Syndrome VIA Intrathecal Delivery
HK40064267A (en) Adeno-associated virus delivery of cln3 polynucleotide
HK40063258A (en) Adeno-associated virus delivery of cln6 polynucleotide

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230202

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230202

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240123

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240422

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20240621

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240723

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20240723

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20241112

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250210

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20250507

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250530

R150 Certificate of patent or registration of utility model

Ref document number: 7691367

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150