AU2017318717B2 - Methods and vectors for treating CNS disorders - Google Patents

Methods and vectors for treating CNS disorders Download PDF

Info

Publication number
AU2017318717B2
AU2017318717B2 AU2017318717A AU2017318717A AU2017318717B2 AU 2017318717 B2 AU2017318717 B2 AU 2017318717B2 AU 2017318717 A AU2017318717 A AU 2017318717A AU 2017318717 A AU2017318717 A AU 2017318717A AU 2017318717 B2 AU2017318717 B2 AU 2017318717B2
Authority
AU
Australia
Prior art keywords
aav
mammal
apoe
raav
human
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
AU2017318717A
Other languages
English (en)
Other versions
AU2017318717A1 (en
Inventor
Beverly L. Davidson
Katherine A. High
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.)
Childrens Hospital of Philadelphia CHOP
Spark Therapeutics Inc
Original Assignee
Childrens Hospital of Philadelphia CHOP
Spark Therapeutics 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 Childrens Hospital of Philadelphia CHOP, Spark Therapeutics Inc filed Critical Childrens Hospital of Philadelphia CHOP
Publication of AU2017318717A1 publication Critical patent/AU2017318717A1/en
Priority to AU2024227138A priority Critical patent/AU2024227138A1/en
Application granted granted Critical
Publication of AU2017318717B2 publication Critical patent/AU2017318717B2/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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C07K14/775—Apolipopeptides
    • 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
    • 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
    • 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)
    • 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)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • 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)
    • C12Y301/06004—N-Acetylgalactosamine-6-sulfatase (3.1.6.4)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01031—Beta-glucuronidase (3.2.1.31)
    • 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
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • 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
    • A01K2267/0312—Animal model for Alzheimer's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2017318717A 2016-09-02 2017-09-01 Methods and vectors for treating CNS disorders Active AU2017318717B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024227138A AU2024227138A1 (en) 2016-09-02 2024-10-06 Methods and vectors for treating cns disorders

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662383274P 2016-09-02 2016-09-02
US62/383,274 2016-09-02
PCT/US2017/049959 WO2018045347A1 (en) 2016-09-02 2017-09-01 Methods and vectors for treating cns disorders

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2024227138A Division AU2024227138A1 (en) 2016-09-02 2024-10-06 Methods and vectors for treating cns disorders

Publications (2)

Publication Number Publication Date
AU2017318717A1 AU2017318717A1 (en) 2019-03-21
AU2017318717B2 true AU2017318717B2 (en) 2024-10-24

Family

ID=61309371

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2017318717A Active AU2017318717B2 (en) 2016-09-02 2017-09-01 Methods and vectors for treating CNS disorders
AU2024227138A Abandoned AU2024227138A1 (en) 2016-09-02 2024-10-06 Methods and vectors for treating cns disorders

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2024227138A Abandoned AU2024227138A1 (en) 2016-09-02 2024-10-06 Methods and vectors for treating cns disorders

Country Status (9)

Country Link
US (1) US20190192693A1 (enExample)
EP (1) EP3506930A4 (enExample)
JP (2) JP7610923B2 (enExample)
CN (1) CN110121356A (enExample)
AU (2) AU2017318717B2 (enExample)
BR (1) BR112019004353A2 (enExample)
CA (1) CA3035628A1 (enExample)
MX (1) MX2019002518A (enExample)
WO (1) WO2018045347A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
MX2020000713A (es) * 2017-07-17 2020-08-31 Spark Therapeutics Inc Metodos de aferesis y sus usos.
US12275948B2 (en) 2018-06-28 2025-04-15 The University Of North Carolina At Chapel Hill Optimized CLN5 genes and expression cassettes and their use
JP7624724B2 (ja) 2018-09-26 2025-01-31 カリフォルニア インスティテュート オブ テクノロジー 標的遺伝子療法のためのアデノ随伴ウイルス組成物
JP2022513034A (ja) * 2018-11-14 2022-02-07 レジェンクスバイオ インコーポレーテッド 神経セロイドリポフスチン症の遺伝子治療
JP7616995B2 (ja) * 2018-11-28 2025-01-17 プリベイル セラピューティクス，インコーポレーテッド 神経変性疾患のための遺伝子治療
JP7637058B2 (ja) 2019-02-22 2025-02-27 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 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
US20220348638A1 (en) * 2019-09-24 2022-11-03 The J. David Gladstone Institutes, a testamentary trust estabilished under the Will of J. David Glad Method of targeting neuronal apoe to treat a neurocognitive disorder
MX2022004524A (es) * 2019-10-16 2022-07-21 Univ Cornell Tratamiento génico para la enfermedad de alzheimer.
AU2021351720A1 (en) * 2020-10-01 2023-05-11 Sangamo Therapeutics, Inc. Methods of detecting anti-aav antibodies
CN112852881B (zh) * 2021-02-09 2023-08-15 南京贝思奥生物科技有限公司 利用细胞穿膜肽增强腺相关病毒在中枢神经系统转导效率的方法
TW202426479A (zh) * 2022-10-10 2024-07-01 美商星火治療公司 Apoe基因療法

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015077473A1 (en) * 2013-11-20 2015-05-28 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7135339B2 (en) * 2003-11-20 2006-11-14 University Of Iowa Research Foundation Methods for producing and using in vivo pseudotyped retroviruses using envelope glycoproteins from lymphocytic choriomeningitis virus (LCMV)
JP5704361B2 (ja) * 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
IN2014KN02672A (enExample) * 2012-05-18 2015-05-08 Univ Iowa Res Found
RU2664471C2 (ru) * 2013-07-26 2018-08-17 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения болезней мозга
AU2016219789B2 (en) * 2015-02-20 2021-03-25 Fondazione Telethon Methods and compositions for treating genetic eye diseases

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015077473A1 (en) * 2013-11-20 2015-05-28 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
ALEXANDRA SHARLAND等: "Liver-directed gene expression using recombinant AAV 2/8 vectors -- a tolerogenic strategy for gene delivery?", 《DISCOVERY MEDECINE》 *

Also Published As

Publication number Publication date
JP2022050574A (ja) 2022-03-30
AU2017318717A1 (en) 2019-03-21
MX2019002518A (es) 2019-07-18
JP7610923B2 (ja) 2025-01-09
CA3035628A1 (en) 2018-03-08
WO2018045347A1 (en) 2018-03-08
EP3506930A4 (en) 2020-09-23
CN110121356A (zh) 2019-08-13
US20190192693A1 (en) 2019-06-27
JP2019529385A (ja) 2019-10-17
EP3506930A1 (en) 2019-07-10
BR112019004353A2 (pt) 2019-05-28
AU2024227138A1 (en) 2024-10-31

Similar Documents

Publication Publication Date Title
AU2017318717B2 (en) Methods and vectors for treating CNS disorders
CA2873890C (en) Methods and compositions for treating amyloid deposits
JP7190352B2 (ja) 認知保護を提供しつつ疾患の発症及び進行を遅らせるための遺伝子治療を用いた神経変性疾患の治療方法
US20230295657A1 (en) Gene therapy using nucleic acid constructs comprising methyl cpg binding protein 2 (mecp2) promoter sequences
IL294535A (en) Zinc finger protein transcription factors for repressing tau expression
EP3071240B1 (en) Method for monitoring efficacy of treatment of alzheimer's disease with an apoe isoform
KR20250112089A (ko) 퇴행성 뇌 질환의 예방 또는 치료를 위한 뇌혈관 특이적 유전자 치료용 조성물
WO2025231406A1 (en) Methods to increase transduction of ependyma cells in brain
WO2024160761A1 (en) An aav2-vector variant for targeted transfer of genes
HK1229258B (en) Method for monitoring efficacy of treatment of alzheimer's disease with an apoe isoform
HK1229258A1 (en) Method for monitoring efficacy of treatment of alzheimer's disease with an apoe isoform
BR112016011258B1 (pt) Métodos e composiçôes para o tratamento de depósitos de amiloide

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)