AU2020218501B2 - Adeno-associated virus delivery of CLN6 polynucleotide - Google Patents

Adeno-associated virus delivery of CLN6 polynucleotide

Info

Publication number
AU2020218501B2
AU2020218501B2 AU2020218501A AU2020218501A AU2020218501B2 AU 2020218501 B2 AU2020218501 B2 AU 2020218501B2 AU 2020218501 A AU2020218501 A AU 2020218501A AU 2020218501 A AU2020218501 A AU 2020218501A AU 2020218501 B2 AU2020218501 B2 AU 2020218501B2
Authority
AU
Australia
Prior art keywords
cln6
months
composition
nucleic acid
scaav9
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
AU2020218501A
Other languages
English (en)
Other versions
AU2020218501A1 (en
Inventor
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
Original Assignee
Nationwide Childrens Hospital 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 Nationwide Childrens Hospital Inc filed Critical Nationwide Childrens Hospital Inc
Publication of AU2020218501A1 publication Critical patent/AU2020218501A1/en
Application granted granted Critical
Publication of AU2020218501B2 publication Critical patent/AU2020218501B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • 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

Landscapes

  • 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)
AU2020218501A 2019-02-04 2020-02-04 Adeno-associated virus delivery of CLN6 polynucleotide Active AU2020218501B2 (en)

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

Publications (2)

Publication Number Publication Date
AU2020218501A1 AU2020218501A1 (en) 2021-08-19
AU2020218501B2 true AU2020218501B2 (en) 2026-01-29

Family

ID=69771088

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020218501A Active AU2020218501B2 (en) 2019-02-04 2020-02-04 Adeno-associated virus delivery of CLN6 polynucleotide

Country Status (14)

Country Link
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=)

Families Citing this family (4)

* 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
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

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 (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU613316B2 (en) * 1986-09-12 1991-08-01 Genentech Inc. Improved recombinant expression
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
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
SE9403613D0 (sv) * 1994-10-21 1994-10-21 Pharmacia Ab Improvement of an expression vector for production of recombinant proteins
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
JP3765574B2 (ja) * 2001-02-22 2006-04-12 三菱化学株式会社 逆向き反復配列を含む組み換え遺伝子及びその利用
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
US9943611B2 (en) * 2012-11-01 2018-04-17 California Institute Of Technology Reversible gene expression
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
CA3011939A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
EP3411484B1 (en) * 2016-02-05 2023-10-04 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid

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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
MICHAëL HOCQUEMILLER, LAURA GIERSCH, MICKAEL AUDRAIN, SAMANTHA PARKER, NATHALIE CARTIER: "Adeno-Associated Virus-Based Gene Therapy for CNS Diseases", HUMAN GENE THERAPY, MARY ANN LIEBERT, INC. PUBLISHERS, GB, vol. 27, no. 7, 1 July 2016 (2016-07-01), GB, pages 478 - 496, XP055579779, ISSN: 1043-0342, DOI: 10.1089/hum.2016.087 *
MOLE SARA E ET AL: "Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis", LANCET NEUROLOGY, vol. 18, no. 1, 20 November 2018 (2018-11-20), pages 107 - 116, *

Also Published As

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
CL2021002051A1 (es) 2022-04-08
SG11202107983TA (en) 2021-08-30
CN113574176B (zh) 2025-06-06
JP2022519597A (ja) 2022-03-24
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

Similar Documents

Publication Publication Date Title
AU2020218501B2 (en) Adeno-associated virus delivery of CLN6 polynucleotide
US20260062713A1 (en) Adeno-associated virus delivery of cln3 polynucleotide
JP2022523766A (ja) Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス
AU2020278960A1 (en) Optimized gene therapy targeting retinal cells
JP2024543253A (ja) Ｓｌｃ６ａ１遺伝子治療のための材料及び方法
EA050932B1 (ru) Доставка полинуклеотида cln6 с помощью аденоассоциированного вируса
US20250382593A1 (en) Adeno-Associated Virus Delivery of CLN1 Polynucleotide
HK40063258A (zh) 腺相关病毒对cln6多核苷酸的递送
HK40063258B (zh) 腺相关病毒对cln6多核苷酸的递送
AU2026202960A1 (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
TW202246513A (zh) Ｃｌｎ３多核苷酸的腺相關病毒遞送
HK40064267A (en) Adeno-associated virus delivery of cln3 polynucleotide
EA051574B1 (ru) Доставка полинуклеотида cln3 с помощью аденоассоциированного вируса
JP2024505257A (ja) 神経セロイドリポフスチン症の遺伝子治療
CN121335978A (zh) 一种人源MeCp2启动子及其用途