JP2024529369A - Ａａｖトランスファープラスミド - Google Patents

Ａａｖトランスファープラスミド Download PDF

Info

Publication number
JP2024529369A
JP2024529369A JP2024502616A JP2024502616A JP2024529369A JP 2024529369 A JP2024529369 A JP 2024529369A JP 2024502616 A JP2024502616 A JP 2024502616A JP 2024502616 A JP2024502616 A JP 2024502616A JP 2024529369 A JP2024529369 A JP 2024529369A
Authority
JP
Japan
Prior art keywords
nucleic acid
aav
sequence
plasmid
stuffer sequence
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
JP2024502616A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024529369A5 (https=
JPWO2023009622A5 (https=
Inventor
コズロースキー，クリストファー
Original Assignee
アトセナ セラピューティクス，インク．
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 アトセナ セラピューティクス，インク． filed Critical アトセナ セラピューティクス，インク．
Publication of JP2024529369A publication Critical patent/JP2024529369A/ja
Publication of JP2024529369A5 publication Critical patent/JP2024529369A5/ja
Publication of JPWO2023009622A5 publication Critical patent/JPWO2023009622A5/ja
Pending legal-status Critical Current

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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • C07K14/46—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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • 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
    • C12N2750/14145—Special targeting system for 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2024502616A 2021-07-28 2022-07-27 Ａａｖトランスファープラスミド Pending JP2024529369A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163226410P 2021-07-28 2021-07-28
US63/226,410 2021-07-28
PCT/US2022/038520 WO2023009622A1 (en) 2021-07-28 2022-07-27 Aav transfer plasmids

Publications (3)

Publication Number Publication Date
JP2024529369A true JP2024529369A (ja) 2024-08-06
JP2024529369A5 JP2024529369A5 (https=) 2025-07-08
JPWO2023009622A5 JPWO2023009622A5 (https=) 2025-07-08

Family

ID=85087966

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024502616A Pending JP2024529369A (ja) 2021-07-28 2022-07-27 Ａａｖトランスファープラスミド

Country Status (8)

Country Link
US (1) US20250099617A1 (https=)
EP (1) EP4377471A4 (https=)
JP (1) JP2024529369A (https=)
CN (1) CN118043474A (https=)
AU (1) AU2022319723A1 (https=)
CA (1) CA3225271A1 (https=)
IL (1) IL310145A (https=)
WO (1) WO2023009622A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025176847A1 (en) * 2024-02-21 2025-08-28 Ags Therapeutics Sas Ocular delivery of active agents via microalgae extracellular vesicles

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7868149B2 (en) * 1999-07-20 2011-01-11 Monsanto Technology Llc Plant genome sequence and uses thereof
US20050244851A1 (en) * 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US10059983B2 (en) * 2012-09-10 2018-08-28 Genesky Diagnostics (Suzhou) Inc. Multiplex nucleic acid analysis
MX384795B (es) * 2013-03-15 2025-03-14 Childrens Hospital Philadelphia Vectores que comprenden secuencias de polinucleotidos rellenadoras/de relleno y metodos de uso.
WO2017161273A1 (en) * 2016-03-18 2017-09-21 The Children's Hospital Of Philadelphia Therapeutic for treatment of diseases including the central nervous system
US10240205B2 (en) * 2017-02-03 2019-03-26 Population Bio, Inc. Methods for assessing risk of developing a viral disease using a genetic test
EP3870148A4 (en) * 2018-10-25 2022-11-09 Takeda Pharmaceutical Company Limited AAV TRIPLE PLASMID SYSTEM
CA3136117A1 (en) * 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders

Also Published As

Publication number Publication date
EP4377471A1 (en) 2024-06-05
CN118043474A (zh) 2024-05-14
IL310145A (en) 2024-03-01
AU2022319723A1 (en) 2024-02-22
WO2023009622A1 (en) 2023-02-02
EP4377471A4 (en) 2025-10-15
CA3225271A1 (en) 2023-02-02
US20250099617A1 (en) 2025-03-27

Similar Documents

Publication Publication Date Title
JP6174318B2 (ja) アデノ随伴ウイルス構築物を含んでなるコンダクティング気道細胞を標的とするための組成物
US20170007720A1 (en) Methods and compositions for gene delivery to on bipolar cells
CN115461066B (zh) 用于基因疗法的编码天冬氨酸酰化酶(aspa)的经修饰的核酸和载体
CN111718947B (zh) 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途
US20220347317A1 (en) Aavrh74 vectors for gene therapy of muscular dystrophies
JP2011516049A (ja) 遺伝子障害を治療する方法
JP2023539368A (ja) コドン最適化したｒｐｇｒｏｒｆ１５遺伝子およびその使用
KR20260016939A (ko) 아데노 부속 바이러스 캡시드 단백질, 이를 포함하는 아데노 부속 바이러스, 벡터 및 응용
Kochergin-Nikitsky et al. Tissue and cell-type-specific transduction using rAAV vectors in lung diseases
JP2024529369A (ja) Ａａｖトランスファープラスミド
JP2023539367A (ja) コドン最適化したｒｅｐ１遺伝子およびその使用
WO2024238907A9 (en) Single plasmid system for aav production
WO2023219533A1 (ru) Аденоассоциированный вирусный вектор на основе серотипа aav.php.b и его применение
TW202426653A (zh) 重組病毒載體、包含該重組病毒載體之重組腺相關病毒及其於治療涎酸酵素缺乏症之用途
CN112041437A (zh) 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
WO2024138833A1 (zh) 一种改造的重组凝血因子viii及其应用
CN111849998A (zh) 编码人卵黄状黄斑病蛋白1的核酸分子及其应用
RU2833225C2 (ru) Аденоассоциированный вирусный вектор, состоящий из белков капсида рнр.в, нуклеиновой кислоты, кодирующей белок smn, и его применение
WO2024254232A2 (en) Two-plasmid system for aav vector particle production
WO2025006829A1 (en) Improved adeno-associated virus vectors
AU2024406119A1 (en) Skeletal muscle-specific promoters
HK40085426A (en) Modified nucleic acids encoding aspartoacylase (aspa) and vector for gene therapy
AU725816B2 (en) Gene therapy for cystic fibrosis
CN117355603A (zh) 用于肌营养不良的基因治疗的aavrh74载体
Carlon et al. Roadmap for an early gene therapy for cystic fibrosis airway

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240321

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250630

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250630