PE20211419A1 - Vectores virales recombinantes y acidos nucleicos para producirlos - Google Patents

Vectores virales recombinantes y acidos nucleicos para producirlos

Info

Publication number
PE20211419A1
PE20211419A1 PE2021000747A PE2021000747A PE20211419A1 PE 20211419 A1 PE20211419 A1 PE 20211419A1 PE 2021000747 A PE2021000747 A PE 2021000747A PE 2021000747 A PE2021000747 A PE 2021000747A PE 20211419 A1 PE20211419 A1 PE 20211419A1
Authority
PE
Peru
Prior art keywords
nucleic acids
produce
viral vectors
recombinant viral
cassettes
Prior art date
Application number
PE2021000747A
Other languages
English (en)
Inventor
Darby Thomas
David Dismuke
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 PE20211419A1 publication Critical patent/PE20211419A1/es

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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

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)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (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)

Abstract

En la presente se describen acidos nucleicos, casetes de transferencia de AAV y plasmidos utilizados en la produccion de vectores virales adenoasociados recombinantes (rAAV). Los acidos nucleicos, casetes y plasmidos descritos comprenden secuencias que expresan uno o mas transgenes que tienen eficacia terapeutica para mejorar, tratar y/o prevenir una o mas enfermedades o trastornos
PE2021000747A 2018-11-21 2019-11-21 Vectores virales recombinantes y acidos nucleicos para producirlos PE20211419A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862770202P 2018-11-21 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
PE20211419A1 true PE20211419A1 (es) 2021-08-03

Family

ID=70773595

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2021000747A PE20211419A1 (es) 2018-11-21 2019-11-21 Vectores virales recombinantes y acidos nucleicos para producirlos

Country Status (20)

Country Link
US (1) US20210324418A1 (es)
EP (1) EP3883954A4 (es)
JP (1) JP2022508182A (es)
KR (1) KR20210103469A (es)
CN (1) CN113302201A (es)
AR (1) AR117145A1 (es)
AU (1) AU2019385506A1 (es)
BR (1) BR112021009733A2 (es)
CA (1) CA3120289A1 (es)
CL (1) CL2021001327A1 (es)
CO (1) CO2021008120A2 (es)
EA (1) EA202191418A1 (es)
EC (1) ECSP21044840A (es)
IL (1) IL283344A (es)
MX (1) MX2021005997A (es)
PE (1) PE20211419A1 (es)
PH (1) PH12021551155A1 (es)
SG (1) SG11202105326WA (es)
TW (1) TW202039533A (es)
WO (1) WO2020106916A1 (es)

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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
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
MX2020010466A (es) 2018-04-03 2021-01-08 Vectores de virus que evitan anticuerpos.
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
KR20220011616A (ko) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 재조합 아데노 관련 바이러스 벡터
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
US20220010332A1 (en) * 2020-07-08 2022-01-13 Neuracle Genetics Inc. Intron fragments
WO2022026410A2 (en) * 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
KR20230068444A (ko) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
KR20230123460A (ko) * 2020-09-29 2023-08-23 뉴엑셀 테라퓨틱스 아이엔씨. Neurod1 벡터
AU2021353867A1 (en) * 2020-09-29 2023-05-11 NeuExcell Therapeutics Inc. Neurod1 combination vector
US20220098617A1 (en) * 2020-09-29 2022-03-31 NeuExcell Therapeutics Inc. Ascl1 vector
CN116887866A (zh) 2020-12-03 2023-10-13 巴特尔纪念研究院 聚合物纳米颗粒和dna纳米结构组合物及用于非病毒递送的方法
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
WO2023221942A1 (en) * 2022-05-16 2023-11-23 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating glutaric aciduria type i
WO2023240162A1 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Aav vectors for gene editing
CN118715025A (zh) * 2022-10-08 2024-09-27 凌意(杭州)生物科技有限公司 用于增强基因表达的构建体
WO2024163678A2 (en) * 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
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

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US20090221620A1 (en) * 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
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
WO2016115503A1 (en) * 2015-01-16 2016-07-21 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
EP3273999A1 (en) * 2015-03-23 2018-01-31 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
CN107849547B (zh) * 2015-05-16 2022-04-19 建新公司 深内含子突变的基因编辑
RU2021102893A (ru) * 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
SG11201804994QA (en) * 2015-12-15 2018-07-30 Genzyme Corp Adeno-associated viral vectors for treating mucolipidosis type ii
US20210254056A1 (en) * 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks

Also Published As

Publication number Publication date
TW202039533A (zh) 2020-11-01
IL283344A (en) 2021-07-29
KR20210103469A (ko) 2021-08-23
EP3883954A1 (en) 2021-09-29
MX2021005997A (es) 2021-08-11
JP2022508182A (ja) 2022-01-19
AU2019385506A1 (en) 2021-06-03
EA202191418A1 (ru) 2021-08-05
SG11202105326WA (en) 2021-06-29
AR117145A1 (es) 2021-07-14
ECSP21044840A (es) 2021-09-30
CO2021008120A2 (es) 2021-08-09
CA3120289A1 (en) 2020-05-28
EP3883954A4 (en) 2022-08-10
US20210324418A1 (en) 2021-10-21
PH12021551155A1 (en) 2021-11-03
BR112021009733A2 (pt) 2022-01-04
CN113302201A (zh) 2021-08-24
WO2020106916A1 (en) 2020-05-28
CL2021001327A1 (es) 2021-12-31

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