CA3120289A1 - Recombinant viral vectors and nucleic acids for producing the same - Google Patents

Recombinant viral vectors and nucleic acids for producing the same Download PDF

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Publication number
CA3120289A1
CA3120289A1 CA3120289A CA3120289A CA3120289A1 CA 3120289 A1 CA3120289 A1 CA 3120289A1 CA 3120289 A CA3120289 A CA 3120289A CA 3120289 A CA3120289 A CA 3120289A CA 3120289 A1 CA3120289 A1 CA 3120289A1
Authority
CA
Canada
Prior art keywords
nucleic acid
sequence
promoter
aav
itr
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
CA3120289A
Other languages
English (en)
French (fr)
Inventor
Darby Thomas
David DISMUKE
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
Stridebio 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 Stridebio Inc filed Critical Stridebio Inc
Publication of CA3120289A1 publication Critical patent/CA3120289A1/en
Pending legal-status Critical Current

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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

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  • 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)
CA3120289A 2018-11-21 2019-11-21 Recombinant viral vectors and nucleic acids for producing the same Pending CA3120289A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862770202P 2018-11-21 2018-11-21
US62/770,202 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
CA3120289A1 true CA3120289A1 (en) 2020-05-28

Family

ID=70773595

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3120289A Pending CA3120289A1 (en) 2018-11-21 2019-11-21 Recombinant viral vectors and nucleic acids for producing the same

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)

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WO2023240162A1 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Aav vectors for gene editing

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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
EP4598590A4 (en) * 2022-10-08 2026-05-27 Lingyi Biotech Co Ltd CONSTRUCTION TO IMPROVE GENE EXPRESSION
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
CN117701638B (zh) * 2023-12-18 2024-10-18 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
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템

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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
EP3632923A1 (en) 2015-01-16 2020-04-08 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
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
PL3298134T3 (pl) * 2015-05-16 2023-09-18 Genzyme Corporation Edycja genów mutacji głęboko intronowych
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
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SG10201912763QA (en) * 2015-11-05 2020-02-27 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
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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
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023240162A1 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Aav vectors for gene editing

Also Published As

Publication number Publication date
AR117145A1 (es) 2021-07-14
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
CN113302201A (zh) 2021-08-24
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

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