CN113302201A - 重组病毒载体和用于产生所述重组病毒载体的核酸 - Google Patents

重组病毒载体和用于产生所述重组病毒载体的核酸 Download PDF

Info

Publication number
CN113302201A
CN113302201A CN201980088932.7A CN201980088932A CN113302201A CN 113302201 A CN113302201 A CN 113302201A CN 201980088932 A CN201980088932 A CN 201980088932A CN 113302201 A CN113302201 A CN 113302201A
Authority
CN
China
Prior art keywords
nucleic acid
sequence
promoter
aav
seq
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
CN201980088932.7A
Other languages
English (en)
Chinese (zh)
Inventor
D·托马斯
D·迪斯姆克
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.)
Takeda Pharmaceutical Co Ltd
Original Assignee
Stridbio
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 Stridbio filed Critical Stridbio
Publication of CN113302201A publication Critical patent/CN113302201A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • 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)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurosurgery (AREA)
  • Psychology (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)
CN201980088932.7A 2018-11-21 2019-11-21 重组病毒载体和用于产生所述重组病毒载体的核酸 Pending CN113302201A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862770202P 2018-11-21 2018-11-21
US62/770202 2018-11-21
PCT/US2019/062531 WO2020106916A1 (en) 2018-11-21 2019-11-21 Recombinant viral vectors and nucleic acids for producing the same

Publications (1)

Publication Number Publication Date
CN113302201A true CN113302201A (zh) 2021-08-24

Family

ID=70773595

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980088932.7A Pending CN113302201A (zh) 2018-11-21 2019-11-21 重组病毒载体和用于产生所述重组病毒载体的核酸

Country Status (20)

Country Link
US (1) US20210324418A1 (enExample)
EP (1) EP3883954A4 (enExample)
JP (2) JP7704998B2 (enExample)
KR (1) KR20210103469A (enExample)
CN (1) CN113302201A (enExample)
AR (1) AR117145A1 (enExample)
AU (1) AU2019385506A1 (enExample)
BR (1) BR112021009733A2 (enExample)
CA (1) CA3120289A1 (enExample)
CL (1) CL2021001327A1 (enExample)
CO (1) CO2021008120A2 (enExample)
EA (1) EA202191418A1 (enExample)
EC (1) ECSP21044840A (enExample)
IL (1) IL283344A (enExample)
MX (1) MX2021005997A (enExample)
PE (1) PE20211419A1 (enExample)
PH (1) PH12021551155A1 (enExample)
SG (1) SG11202105326WA (enExample)
TW (1) TW202039533A (enExample)
WO (1) WO2020106916A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117701638A (zh) * 2023-12-18 2024-03-15 苏州新芽基因生物技术有限公司 一种抑制残留aav载体质粒和质粒骨架转录活性的方法
WO2024074143A1 (en) * 2022-10-08 2024-04-11 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107686842A (zh) * 2016-08-03 2018-02-13 南京大学 一种靶多核苷酸编辑方法及其应用
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
US12173304B2 (en) 2020-07-08 2024-12-24 Neuracle Genetics Inc. Intron fragments
US20230285596A1 (en) * 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
JP2023542614A (ja) * 2020-08-19 2023-10-11 サレプタ セラピューティクス， インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
EP4221761A4 (en) * 2020-09-29 2025-02-26 Neuexcell Therapeutics Inc. Neurod1 combination vector
WO2022072309A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Ascl1 vector
MX2023003655A (es) * 2020-09-29 2023-06-22 Neuexcell Therapeutics Inc Vector neurod1.
CA3197320A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Dlx2 vector
WO2022087321A1 (en) * 2020-10-21 2022-04-28 Duke University Dual aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
IL303390A (en) 2020-12-03 2023-08-01 Battelle Memorial Institute Compositions of polymer nanoparticles and DNA nanostructures and methods for non-viral transport
US20240058477A1 (en) * 2021-01-04 2024-02-22 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
WO2022216977A1 (en) 2021-04-07 2022-10-13 Batelle Memorial Institute Rapid design, build, test, and learn technologies for identifying and using non-viral carriers
MX2024003241A (es) * 2021-09-17 2024-05-15 Astellas Gene Therapies Inc Terapia genica con frataxina.
CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
EP4536286A1 (en) * 2022-06-08 2025-04-16 Scribe Therapeutics Inc. Aav vectors for gene editing
WO2024163678A2 (en) * 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2025072751A1 (en) 2023-09-29 2025-04-03 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
AU2024393766A1 (en) 2023-12-08 2026-06-18 Battelle Memorial Institute Use of dna origami nanostructures for molecular information based data storage systems
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016115503A1 (en) * 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
CN107849547A (zh) * 2015-05-16 2018-03-27 建新公司 深内含子突变的基因编辑
CN108603199A (zh) * 2015-12-15 2018-09-28 建新公司 用于治疗粘脂贮积症ii型的腺伴随病毒载体

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2002254529A1 (en) * 2001-04-04 2002-10-21 The Government Of The United States Of America, As Represented By The Secretary Of The Department Of Nucleic acids for transgene expression
US20090221620A1 (en) * 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
DK2986635T3 (en) * 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
US20160256571A1 (en) * 2013-09-30 2016-09-08 Sanofi Invention
US10947596B2 (en) 2014-05-06 2021-03-16 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of cancer using NFS1 biomarkers and modulators
US11697825B2 (en) * 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
CA3255657A1 (en) * 2015-02-10 2025-12-01 Genzyme Corporation VARIANT RNAi
WO2016150964A1 (en) * 2015-03-23 2016-09-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with friedreich ataxia
EP3280451B8 (en) * 2015-04-08 2019-09-25 The United States of America, as represented by the Secretary, Department of Health and Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
EP4378487A3 (en) * 2015-06-23 2024-09-04 The Children's Hospital of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2017023708A1 (en) * 2015-07-31 2017-02-09 California Institute Of Technology Activity-dependent expression of nucleic acids
SG10201912763QA (en) * 2015-11-05 2020-02-27 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
CA3029119A1 (en) * 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
US20200056206A1 (en) * 2016-12-01 2020-02-20 UNIVERSITé LAVAL Crispr-based treatment of friedreich ataxia
US20210254056A1 (en) * 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
MX2020003042A (es) * 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016115503A1 (en) * 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
CN107849547A (zh) * 2015-05-16 2018-03-27 建新公司 深内含子突变的基因编辑
CN108603199A (zh) * 2015-12-15 2018-09-28 建新公司 用于治疗粘脂贮积症ii型的腺伴随病毒载体

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
EVANS-GALEA, M.V.等: "frataxin, mitochondrial isoform 1 preproprotein [Homo sapiens]", GENBANK DATABASE *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024074143A1 (en) * 2022-10-08 2024-04-11 Lingyi Biotech Co., Ltd. Construct for enhancing gene expression
CN117701638A (zh) * 2023-12-18 2024-03-15 苏州新芽基因生物技术有限公司 一种抑制残留aav载体质粒和质粒骨架转录活性的方法

Also Published As

Publication number Publication date
AR117145A1 (es) 2021-07-14
CA3120289A1 (en) 2020-05-28
EP3883954A1 (en) 2021-09-29
MX2021005997A (es) 2021-08-11
BR112021009733A2 (pt) 2022-01-04
CO2021008120A2 (es) 2021-08-09
AU2019385506A1 (en) 2021-06-03
PH12021551155A1 (en) 2021-11-03
JP7704998B2 (ja) 2025-07-09
ECSP21044840A (es) 2021-09-30
US20210324418A1 (en) 2021-10-21
CL2021001327A1 (es) 2021-12-31
IL283344A (en) 2021-07-29
EP3883954A4 (en) 2022-08-10
KR20210103469A (ko) 2021-08-23
WO2020106916A1 (en) 2020-05-28
EA202191418A1 (ru) 2021-08-05
TW202039533A (zh) 2020-11-01
JP2025102783A (ja) 2025-07-08
SG11202105326WA (en) 2021-06-29
PE20211419A1 (es) 2021-08-03
JP2022508182A (ja) 2022-01-19

Similar Documents

Publication Publication Date Title
KR20210103469A (ko) 재조합 바이러스 벡터 및 이를 생산하기 위한 핵산
JP7433360B2 (ja) Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法
CN112368390B (zh) Cns变性的基因疗法
KR102918544B1 (ko) 다논병을 치료하기 위한 유전자 요법 벡터
JP3755827B2 (ja) 組み込み可能な組み換えアデノウィルス、それらの製造及びそれらの治療的利用
KR100403708B1 (ko) 재조합아데노-수반바이러스(aav)제조방법및이의용도
CN116194154A (zh) 使用aav载体的plakophilin-2(pkp2)基因疗法
CN113227385A (zh) 核酸分子及其用于非病毒基因疗法的用途
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
CN117545842A (zh) SMN1和miR-23a在治疗脊髓性肌萎缩中的协同效应
CN116745409A (zh) 用于治疗雷特综合征的腺相关病毒载体
KR20250102136A (ko) 중추 신경계에서의 유전자 발현을 위한 핵산 조절 요소 및 사용 방법
US6806080B2 (en) Hybrid vectors for gene therapy
KR20240025645A (ko) 아데노-연관된 바이러스 패키징 시스템
CN115029360B (zh) 用于治疗粘多糖贮积症iiia型的转基因表达盒
EP3262065A1 (en) Methods and compositions for treating dystroglycanopathy disorders
CN115484975A (zh) 用于ids基因转移的腺相关病毒组合物和其使用方法
RU2839898C2 (ru) Синергетическое действие SMN1 и miR-23a при лечении спинальной мышечной атрофии
EA047326B1 (ru) СИНЕРГЕТИЧЕСКОЕ ДЕЙСТВИЕ SMN1 И miR-23a ПРИ ЛЕЧЕНИИ СПИНАЛЬНОЙ МЫШЕЧНОЙ АТРОФИИ
HK40104471A (en) Expression cassettes for target genes and their applications
CN117716042A (zh) 腺相关病毒包装系统
WO2024094009A1 (zh) 用于目的基因的表达盒及其应用
HK40106789A (en) Synergistic effect of smn1 and mir-23a in treating spinal muscular atrophy
EA049295B1 (ru) Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения

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
TA01 Transfer of patent application right

Effective date of registration: 20240115

Address after: Osaka, Japan

Applicant after: TAKEDA PHARMACEUTICAL Co.,Ltd.

Address before: North Carolina, USA

Applicant before: Stridbio

TA01 Transfer of patent application right