AU2020217708B2 - Adeno-associated virus delivery of CLN3 polynucleotide - Google Patents

Adeno-associated virus delivery of CLN3 polynucleotide

Info

Publication number
AU2020217708B2
AU2020217708B2 AU2020217708A AU2020217708A AU2020217708B2 AU 2020217708 B2 AU2020217708 B2 AU 2020217708B2 AU 2020217708 A AU2020217708 A AU 2020217708A AU 2020217708 A AU2020217708 A AU 2020217708A AU 2020217708 B2 AU2020217708 B2 AU 2020217708B2
Authority
AU
Australia
Prior art keywords
cln3
subc
month
total area
rule
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
AU2020217708A
Other languages
English (en)
Other versions
AU2020217708A1 (en
Inventor
Kevin Foust
Brian K. Kaspar
Kathrin Meyer
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nationwide Childrens Hospital Inc
Ohio State Innovation Foundation
Original Assignee
Nationwide Childrens Hospital Inc
Ohio State Innovation Foundation
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 Nationwide Childrens Hospital Inc, Ohio State Innovation Foundation filed Critical Nationwide Childrens Hospital Inc
Publication of AU2020217708A1 publication Critical patent/AU2020217708A1/en
Application granted granted Critical
Publication of AU2020217708B2 publication Critical patent/AU2020217708B2/en
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)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2020217708A 2019-02-04 2020-02-04 Adeno-associated virus delivery of CLN3 polynucleotide Active AU2020217708B2 (en)

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
AU2026202960A Division AU2026202960A1 (en) 2019-02-04 2026-04-20 Adeno-Associated Virus Delivery Of CLN3 Polynucleotide

Publications (2)

Publication Number Publication Date
AU2020217708A1 AU2020217708A1 (en) 2021-08-19
AU2020217708B2 true AU2020217708B2 (en) 2026-01-22

Family

ID=69771089

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020217708A Active AU2020217708B2 (en) 2019-02-04 2020-02-04 Adeno-associated virus delivery of CLN3 polynucleotide

Country Status (16)

Country Link
US (2) US12404523B2 (https=)
EP (1) EP3921430A1 (https=)
JP (2) JP7691367B2 (https=)
KR (1) KR20210124300A (https=)
CN (1) CN113646436A (https=)
AR (1) AR118005A1 (https=)
AU (1) AU2020217708B2 (https=)
BR (1) BR112021015285A2 (https=)
CA (1) CA3129077A1 (https=)
CL (1) CL2021002049A1 (https=)
EA (1) EA202192160A1 (https=)
IL (1) IL285328A (https=)
MX (1) MX2021009401A (https=)
SG (1) SG11202107986YA (https=)
TW (1) TW202045530A (https=)
WO (1) WO2020163300A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2013296425B2 (en) 2012-08-01 2018-06-07 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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016100575A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease

Family Cites Families (24)

* 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
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
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
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
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released 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
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
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
US9434928B2 (en) 2011-11-23 2016-09-06 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
AU2013296425B2 (en) 2012-08-01 2018-06-07 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
WO2015141521A1 (ja) 2014-03-21 2015-09-24 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記録媒体
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
JP7106534B2 (ja) * 2016-11-17 2022-07-26 ネイションワイド チルドレンズ ホスピタル， インコーポレイテッド メチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルスの髄腔内送達

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016100575A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease

Also Published As

Publication number Publication date
TW202045530A (zh) 2020-12-16
CN113646436A (zh) 2021-11-12
EA202192160A1 (ru) 2021-11-17
US12404523B2 (en) 2025-09-02
KR20210124300A (ko) 2021-10-14
AR118005A1 (es) 2021-09-08
JP2025138648A (ja) 2025-09-25
WO2020163300A1 (en) 2020-08-13
SG11202107986YA (en) 2021-08-30
US20260062713A1 (en) 2026-03-05
EP3921430A1 (en) 2021-12-15
MX2021009401A (es) 2021-11-12
CA3129077A1 (en) 2020-08-13
BR112021015285A2 (pt) 2021-10-05
JP7691367B2 (ja) 2025-06-11
US20220127641A1 (en) 2022-04-28
IL285328A (en) 2021-09-30
AU2020217708A1 (en) 2021-08-19
CL2021002049A1 (es) 2022-04-01
WO2020163300A9 (en) 2021-04-15
JP2022519596A (ja) 2022-03-24

Similar Documents

Publication Publication Date Title
US20260062713A1 (en) Adeno-associated virus delivery of cln3 polynucleotide
MX2013009604A (es) Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia.
AU2020218501B2 (en) Adeno-associated virus delivery of CLN6 polynucleotide
AU2026202960A1 (en) Adeno-Associated Virus Delivery Of CLN3 Polynucleotide
US20250382593A1 (en) Adeno-Associated Virus Delivery of CLN1 Polynucleotide
WO2022170038A1 (en) Adeno-associated virus delivery of cln3 polynucleotide
EA051574B1 (ru) Доставка полинуклеотида cln3 с помощью аденоассоциированного вируса
HK40064267A (en) Adeno-associated virus delivery of cln3 polynucleotide
JP7826334B2 (ja) 髄腔内送達によってピット・ホプキンス症候群を治療するためのメチル－ｃｐｇ結合タンパク質２をコードする組換えアデノ随伴ウイルス
EA050932B1 (ru) Доставка полинуклеотида cln6 с помощью аденоассоциированного вируса
HK40063258A (zh) 腺相关病毒对cln6多核苷酸的递送
JP2024505257A (ja) 神経セロイドリポフスチン症の遺伝子治療
HK40063258B (zh) 腺相关病毒对cln6多核苷酸的递送