AU2019389047A1 - AAV viral vectors and uses thereof - Google Patents

AAV viral vectors and uses thereof Download PDF

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Publication number
AU2019389047A1
AU2019389047A1 AU2019389047A AU2019389047A AU2019389047A1 AU 2019389047 A1 AU2019389047 A1 AU 2019389047A1 AU 2019389047 A AU2019389047 A AU 2019389047A AU 2019389047 A AU2019389047 A AU 2019389047A AU 2019389047 A1 AU2019389047 A1 AU 2019389047A1
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AU
Australia
Prior art keywords
patient
administration
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per
months
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Pending
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AU2019389047A
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English (en)
Inventor
Joseph BALLEYDIER
Douglas FELTNER
James Michael HATFIELD
Robert Emil Hodge
Allan Arman KASPAR
Brian K. Kaspar
Matthew MERIGGIOLI
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Novartis AG
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Novartis AG
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 Novartis AG filed Critical Novartis AG
Publication of AU2019389047A1 publication Critical patent/AU2019389047A1/en
Pending legal-status Critical Current

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    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
AU2019389047A 2018-11-30 2019-11-27 AAV viral vectors and uses thereof Pending AU2019389047A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862773894P 2018-11-30 2018-11-30
US62/773,894 2018-11-30
US201962835242P 2019-04-17 2019-04-17
US62/835,242 2019-04-17
PCT/US2019/063649 WO2020113034A1 (en) 2018-11-30 2019-11-27 Aav viral vectors and uses thereof

Publications (1)

Publication Number Publication Date
AU2019389047A1 true AU2019389047A1 (en) 2021-05-20

Family

ID=69167903

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2019389047A Pending AU2019389047A1 (en) 2018-11-30 2019-11-27 AAV viral vectors and uses thereof

Country Status (12)

Country Link
US (1) US20220001028A1 (https=)
EP (1) EP3886919A1 (https=)
JP (2) JP7677887B2 (https=)
KR (1) KR20210099025A (https=)
CN (2) CN120860258A (https=)
AU (1) AU2019389047A1 (https=)
BR (1) BR112021009739A2 (https=)
CA (1) CA3116630A1 (https=)
IL (1) IL282885A (https=)
MX (1) MX2021006359A (https=)
TW (2) TWI897418B (https=)
WO (1) WO2020113034A1 (https=)

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TW202140791A (zh) * 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
CN112121179A (zh) * 2020-09-15 2020-12-25 山东兴瑞生物科技有限公司 一种组合物及其在治疗脊髓性肌萎缩症中的应用
US20240084323A1 (en) * 2021-01-13 2024-03-14 Dignity Health Modulation of chitinase protein expression
EP4377459A2 (en) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Compositions and methods for modulating expression of frataxin (fxn)
WO2023010135A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4380602A4 (en) * 2021-08-03 2025-06-11 The Regents Of The University Of California ADENO-ASSOCIATED VIRAL (AAV)-MEDIATED EXPRESSION OF SPHINGOSINE 1-PHOSPHATE LYASE (SPL) FOR THE TREATMENT OF PULMONARY FIBROSIS
CN114276419B (zh) * 2021-12-30 2023-11-17 上海勉亦生物科技有限公司 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
KR20230152503A (ko) * 2022-04-27 2023-11-03 주식회사 헬릭스미스 척수강 내 투여에 최적화 된 간세포 성장인자 유전자가 도입된 aav 벡터
KR102717399B1 (ko) * 2022-05-10 2024-10-16 서울대학교산학협력단 인간 smn1 단백질 변이체 및 이의 용도
WO2023246734A1 (en) 2022-06-21 2023-12-28 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of sma disease
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024238958A1 (en) * 2023-05-18 2024-11-21 Ultragenyx Pharmaceutical Inc. Formulations comprising recombinant aav and methods of administering the same
WO2026028089A1 (en) 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)

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Also Published As

Publication number Publication date
WO2020113034A1 (en) 2020-06-04
TW202509229A (zh) 2025-03-01
US20220001028A1 (en) 2022-01-06
CN113226380A (zh) 2021-08-06
MX2021006359A (es) 2021-08-11
CN120860258A (zh) 2025-10-31
EP3886919A1 (en) 2021-10-06
JP2025060641A (ja) 2025-04-10
JP7677887B2 (ja) 2025-05-15
BR112021009739A2 (pt) 2021-10-19
JP2022511776A (ja) 2022-02-01
TW202039859A (zh) 2020-11-01
TWI844587B (zh) 2024-06-11
CA3116630A1 (en) 2020-06-04
JP7808674B2 (ja) 2026-01-29
KR20210099025A (ko) 2021-08-11
TWI897418B (zh) 2025-09-11
IL282885A (en) 2021-06-30

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