AU2020215592A1 - AAV vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2 - Google Patents

AAV vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2 Download PDF

Info

Publication number
AU2020215592A1
AU2020215592A1 AU2020215592A AU2020215592A AU2020215592A1 AU 2020215592 A1 AU2020215592 A1 AU 2020215592A1 AU 2020215592 A AU2020215592 A AU 2020215592A AU 2020215592 A AU2020215592 A AU 2020215592A AU 2020215592 A1 AU2020215592 A1 AU 2020215592A1
Authority
AU
Australia
Prior art keywords
aav
dose
vector
tpp1
vector genomes
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.)
Abandoned
Application number
AU2020215592A
Other languages
English (en)
Inventor
David William Anderson
Sue E.I. DASEN
Maryann TOTO
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.)
Spark Therapeutics Inc
Original Assignee
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 Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of AU2020215592A1 publication Critical patent/AU2020215592A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • 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)
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Transplantation (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicinal Preparation (AREA)
AU2020215592A 2019-02-01 2020-01-31 AAV vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2 Abandoned AU2020215592A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962800131P 2019-02-01 2019-02-01
US62/800,131 2019-02-01
PCT/US2020/016235 WO2020160486A1 (en) 2019-02-01 2020-01-31 Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2

Publications (1)

Publication Number Publication Date
AU2020215592A1 true AU2020215592A1 (en) 2021-08-12

Family

ID=71841678

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020215592A Abandoned AU2020215592A1 (en) 2019-02-01 2020-01-31 AAV vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2

Country Status (9)

Country Link
US (1) US20220184188A1 (https=)
EP (1) EP3917522A4 (https=)
JP (2) JP2022518931A (https=)
CN (1) CN113891712A (https=)
AU (1) AU2020215592A1 (https=)
BR (1) BR112021015050A2 (https=)
CA (1) CA3127889A1 (https=)
MX (1) MX2021009250A (https=)
WO (1) WO2020160486A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3132379A1 (en) * 2019-03-22 2020-10-01 Rush University Medical Center Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal storage disorders
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
WO2025231406A1 (en) * 2024-05-02 2025-11-06 The Children's Hospital Of Philadelphia Methods to increase transduction of ependyma cells in brain

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2132309A4 (en) * 2007-02-23 2011-01-05 Univ Florida COMPOSITIONS AND METHODS OF TREATING GLYCOLOGICAL MEMORY DISEASES
WO2012135857A1 (en) * 2011-03-31 2012-10-04 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
US20140271550A1 (en) * 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
EP3024497B1 (en) * 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
US10900053B2 (en) * 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
KR102738860B1 (ko) * 2015-10-23 2024-12-04 유니버시티 오브 아이오와 리써치 파운데이션 리소좀 축적 질환 (lsd)을 갖는 포유동물의 치료 방법에 사용하기 위한 aav 입자를 포함하는 약제학적 조성물
CA3041548A1 (en) * 2016-11-04 2018-05-11 The Children's Hospital Of Philadelphia Gene transfer compositions, methods and uses for treating neurodegenerative diseases

Also Published As

Publication number Publication date
JP2025098018A (ja) 2025-07-01
CN113891712A (zh) 2022-01-04
CA3127889A1 (en) 2020-08-06
JP2022518931A (ja) 2022-03-17
MX2021009250A (es) 2021-11-04
WO2020160486A1 (en) 2020-08-06
US20220184188A1 (en) 2022-06-16
EP3917522A4 (en) 2022-09-28
BR112021015050A2 (pt) 2021-10-05
EP3917522A1 (en) 2021-12-08

Similar Documents

Publication Publication Date Title
EP3364970B1 (en) Gene therapy for use in treating lysosomal storage disease
US20230142320A1 (en) Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs, and tissues
TWI904135B (zh) 用於治療cdkl5缺乏症之基因療法
JP2022528416A (ja) 組換えアデノ随伴ウイルス及びその使用
WO2019210267A2 (en) Engineered aav capsids with increased tropism and aav vectors comprising the engineered capsids and methods of making and using same
JP2022526021A (ja) リソソーム障害のための遺伝子療法
JP2016517278A (ja) スタッファー／フィラーポリヌクレオチド配列を含むベクターおよびその使用方法
JP2023002721A (ja) 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用
TW202305124A (zh) 具有腦特異性靶向模體的新穎構成物及含有其之組成物
JP2016525356A (ja) 変異ａａｖ、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
TW202012425A (zh) 最適化啟動子序列、無內含子之表現構築體及使用方法
JP2025098018A (ja) 遅発型小児性神経セロイドリポフスチン沈着症２型に対するａａｖベクター治療法
CA3061925A1 (en) Sulfamidase (sgsh) variants, vectors, compositions and methods and uses for treating mucopolysaccharidosis type iiia (mps iiia)
US20250011810A1 (en) Recombinant aav formulations
JP2024542950A (ja) Ｃｄｋｌ５欠損症（ｃｄｄ）の治療において有用な組成物
JP2024530050A (ja) 中枢神経系に影響を及ぼす障害の改善された治療のための組成物及び方法
JP2023545384A (ja) 中枢神経系または筋肉送達のための組換えアデノ随伴ウイルス
RU2805606C2 (ru) Композиции, способы и применения переноса генов для лечения нейродегенеративных заболеваний
TW202516019A (zh) 具中樞神經系統靶向模體的突變aav及含有其之組成物
WO2021181096A1 (en) Gene therapy of niemann-pick disease type c
NZ713958B2 (en) Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases

Legal Events

Date Code Title Description
MK5 Application lapsed section 142(2)(e) - patent request and compl. specification not accepted