CN115175706A - 用于治疗年龄相关性黄斑变性及其它眼部疾病和病症的腺伴随病毒(aav)载体 - Google Patents

用于治疗年龄相关性黄斑变性及其它眼部疾病和病症的腺伴随病毒(aav)载体 Download PDF

Info

Publication number
CN115175706A
CN115175706A CN202080089391.2A CN202080089391A CN115175706A CN 115175706 A CN115175706 A CN 115175706A CN 202080089391 A CN202080089391 A CN 202080089391A CN 115175706 A CN115175706 A CN 115175706A
Authority
CN
China
Prior art keywords
nucleic acid
cfh
vector
seq
aav
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.)
Pending
Application number
CN202080089391.2A
Other languages
English (en)
Chinese (zh)
Inventor
M·希尔曼
A·蒂默斯
J·纽马克
S·彭诺克
R·穆雷利
C·宋
L·克耶斯
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.)
Applied Genetic Technologies Corp
Original Assignee
Applied Genetic Technologies Corp
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 Applied Genetic Technologies Corp filed Critical Applied Genetic Technologies Corp
Publication of CN115175706A publication Critical patent/CN115175706A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C07K14/472—Complement proteins, e.g. anaphylatoxin, C3a, C5a
    • 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
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • 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
    • 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)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (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)
CN202080089391.2A 2019-10-22 2020-10-22 用于治疗年龄相关性黄斑变性及其它眼部疾病和病症的腺伴随病毒(aav)载体 Pending CN115175706A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962924338P 2019-10-22 2019-10-22
US62/924338 2019-10-22
PCT/US2020/056862 WO2021081203A1 (en) 2019-10-22 2020-10-22 Adeno-associated virus (aav)vectors for the treatment of age-related macular degeneration and other ocular diseases and disorders

Publications (1)

Publication Number Publication Date
CN115175706A true CN115175706A (zh) 2022-10-11

Family

ID=75586875

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080089391.2A Pending CN115175706A (zh) 2019-10-22 2020-10-22 用于治疗年龄相关性黄斑变性及其它眼部疾病和病症的腺伴随病毒(aav)载体

Country Status (10)

Country Link
US (1) US20210123076A1 (https=)
EP (1) EP4048321A4 (https=)
JP (1) JP2022553309A (https=)
KR (1) KR20230019062A (https=)
CN (1) CN115175706A (https=)
AU (1) AU2020371663A1 (https=)
CA (1) CA3158517A1 (https=)
IL (1) IL292379A (https=)
MX (1) MX2022004770A (https=)
WO (1) WO2021081203A1 (https=)

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101160412A (zh) * 2005-02-14 2008-04-09 爱荷华大学研究基金会 治疗和诊断年龄相关性黄斑变性的方法和试剂
US20080152659A1 (en) * 2006-07-13 2008-06-26 University Of Iowa Research Foundation Methods and reagents for treatment and diagnosis of vascular disorders and age-related macular degeneration
US20100069467A1 (en) * 2006-04-28 2010-03-18 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
CN103920142A (zh) * 2005-02-14 2014-07-16 爱荷华大学研究基金会 治疗和诊断年龄相关性黄斑变性的方法和试剂
CN108291216A (zh) * 2015-09-24 2018-07-17 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017114401A1 (zh) * 2015-12-31 2017-07-06 江苏匡亚生物医药科技有限公司 具有补体调节活性的重组补体因子h-免疫球蛋白融合蛋白及其制备方法与应用
GB201706808D0 (en) * 2017-04-28 2017-06-14 Univ Newcastle Modified complement proteins and uses thereof
EP3697920A4 (en) * 2017-10-20 2022-03-02 Gemini Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF AGE-RELATED MACULAR DEGENERATION

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101160412A (zh) * 2005-02-14 2008-04-09 爱荷华大学研究基金会 治疗和诊断年龄相关性黄斑变性的方法和试剂
CN103920142A (zh) * 2005-02-14 2014-07-16 爱荷华大学研究基金会 治疗和诊断年龄相关性黄斑变性的方法和试剂
US20100069467A1 (en) * 2006-04-28 2010-03-18 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
US20080152659A1 (en) * 2006-07-13 2008-06-26 University Of Iowa Research Foundation Methods and reagents for treatment and diagnosis of vascular disorders and age-related macular degeneration
CN108291216A (zh) * 2015-09-24 2018-07-17 宾夕法尼亚州大学信托人 用于治疗补体介导的疾病的组合物和方法
US20190177381A1 (en) * 2015-09-24 2019-06-13 The Trustees Of The University Of Pennsylvania Composition and method for treating complement-mediated disease

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
C Q SCHMIDT 等: "Translational mini-review series on complement factor H: structural and functional correlations for factor H", CLIN EXP IMMUNOL, vol. 151, no. 1, 7 December 2007 (2007-12-07), pages 14 - 24, XP055218093, DOI: 10.1111/j.1365-2249.2007.03553.x *
DANIEL GRIGSBY 等: "AAV Gene Augmentation of Truncated Complement Factor H Differentially Rescues Ocular Complement Dysregulation in a Mouse Model", INVEST OPHTHALMOL VIS SCI, vol. 64, no. 10, 3 July 2023 (2023-07-03), pages 1 - 11 *

Also Published As

Publication number Publication date
EP4048321A4 (en) 2023-11-22
WO2021081203A1 (en) 2021-04-29
US20210123076A1 (en) 2021-04-29
CA3158517A1 (en) 2021-04-29
AU2020371663A1 (en) 2022-05-19
MX2022004770A (es) 2022-10-07
EP4048321A1 (en) 2022-08-31
IL292379A (en) 2022-06-01
KR20230019062A (ko) 2023-02-07
JP2022553309A (ja) 2022-12-22

Similar Documents

Publication Publication Date Title
US12241077B2 (en) Adeno-associated virus (AAV) systems for treatment of genetic hearing loss
US20250121092A1 (en) Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
JP6072772B2 (ja) 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
AU2017202320B2 (en) Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
CA3241202A1 (en) Gene therapy for lamin a - associated deficiencies
CN115715327A (zh) 用于治疗颗粒蛋白前体相关的神经退行性疾病或病症的腺伴随病毒(aav)系统
US20240384297A1 (en) Gene therapy construct for treating pompe disease, pharmaceutical composition, and method
CN115175706A (zh) 用于治疗年龄相关性黄斑变性及其它眼部疾病和病症的腺伴随病毒(aav)载体
WO2023039175A2 (en) Predictors of achromatopsia treatment efficacy
WO2022271984A1 (en) Methods for the treatment of achromatopsia and other cngb3 associated diseases
AU2019250266A1 (en) Melatonin agonist treatment

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20221011

WD01 Invention patent application deemed withdrawn after publication