IL283344A - Recombinant viral vectors and nucleic acids for compatible production - Google Patents

Recombinant viral vectors and nucleic acids for compatible production

Info

Publication number
IL283344A
IL283344A IL283344A IL28334421A IL283344A IL 283344 A IL283344 A IL 283344A IL 283344 A IL283344 A IL 283344A IL 28334421 A IL28334421 A IL 28334421A IL 283344 A IL283344 A IL 283344A
Authority
IL
Israel
Prior art keywords
producing
same
nucleic acids
viral vectors
recombinant viral
Prior art date
Application number
IL283344A
Other languages
English (en)
Hebrew (he)
Inventor
Erin M Foley
Erich Veitenheimer
Michael S Tuscan
Original Assignee
Stridebio Inc
Erin M Foley
Erich Veitenheimer
Michael S Tuscan
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, Erin M Foley, Erich Veitenheimer, Michael S Tuscan filed Critical Stridebio Inc
Publication of IL283344A publication Critical patent/IL283344A/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
IL283344A 2018-11-21 2021-05-20 Recombinant viral vectors and nucleic acids for compatible production IL283344A (en)

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
IL283344A true IL283344A (en) 2021-07-29

Family

ID=70773595

Family Applications (1)

Application Number Title Priority Date Filing Date
IL283344A IL283344A (en) 2018-11-21 2021-05-20 Recombinant viral vectors and nucleic acids for compatible production

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)

Families Citing this family (28)

* 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
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
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
MX2023002016A (es) * 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
MX2023003656A (es) * 2020-09-29 2023-06-22 Neuexcell Therapeutics Inc Vector dlx2.
EP4222270A4 (en) * 2020-09-29 2025-02-26 Neuexcell Therapeutics Inc. NEUROD1-VECTOR
US20220098617A1 (en) * 2020-09-29 2022-03-31 NeuExcell Therapeutics Inc. Ascl1 vector
MX2023003654A (es) * 2020-09-29 2023-06-22 Neuexcell Therapeutics Inc Vector de combinación de neurod1.
EP4232152A4 (en) * 2020-10-21 2025-04-23 Duke University Dual AAV vector-mediated deletion of a large mutation hotspot for the treatment of Duchenne muscular dystrophy
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
WO2022147575A1 (en) * 2021-01-04 2022-07-07 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
JP2024516108A (ja) 2021-04-07 2024-04-12 バテル・メモリアル・インスティテュート 非ウイルス性担体を同定および使用するための迅速な設計、構築、試験、および学習技術
US20240376497A1 (en) * 2021-09-17 2024-11-14 Astellas Gene Therapies, Inc. Frataxin gene therapy
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
US20250361525A1 (en) * 2022-06-08 2025-11-27 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
WO2025072751A1 (en) 2023-09-29 2025-04-03 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
US12441996B2 (en) 2023-12-08 2025-10-14 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

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002081632A2 (en) * 2001-04-04 2002-10-17 The Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services 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
EP2986635B1 (en) * 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
WO2015044704A1 (en) * 2013-09-30 2015-04-02 Sanofi Use of neuroglobin agonist for preventing or treating mitochondrial rcci and/or rcciii deficiency disease
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
WO2016094783A1 (en) * 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US20180021364A1 (en) 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US10450563B2 (en) * 2015-02-10 2019-10-22 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
WO2016164642A1 (en) * 2015-04-08 2016-10-13 The United States Of America, As Represented By The Secretary Of Health And Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
RU2021132397A (ru) * 2015-05-16 2022-02-24 Джензим Корпорейшн Генное редактирование глубоких интронных мутаций
PT3313991T (pt) * 2015-06-23 2024-09-27 Childrens Hospital Philadelphia Fator ix modificado e composições, métodos e utilizações para transferência de genes para células, órgãos e tecidos
US10266844B2 (en) * 2015-07-31 2019-04-23 California Institute Of Technology Activity-dependent expression of nucleic acids
PE20181156A1 (es) * 2015-11-05 2018-07-19 Bamboo Therapeutics Inc Genes de ataxia de friedreich modificados y vectores para terapia genica
RU2742612C2 (ru) * 2015-12-15 2021-02-09 Джензим Корпорейшн Векторы на основе аденоассоциированного вируса для лечения муколипидоза типа ii
US11564997B2 (en) * 2016-06-29 2023-01-31 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.

Also Published As

Publication number Publication date
BR112021009733A2 (pt) 2022-01-04
EA202191418A1 (ru) 2021-08-05
JP2025102783A (ja) 2025-07-08
CN113302201A (zh) 2021-08-24
ECSP21044840A (es) 2021-09-30
SG11202105326WA (en) 2021-06-29
TW202039533A (zh) 2020-11-01
PH12021551155A1 (en) 2021-11-03
JP2022508182A (ja) 2022-01-19
AR117145A1 (es) 2021-07-14
KR20210103469A (ko) 2021-08-23
US20210324418A1 (en) 2021-10-21
CL2021001327A1 (es) 2021-12-31
WO2020106916A1 (en) 2020-05-28
EP3883954A4 (en) 2022-08-10
PE20211419A1 (es) 2021-08-03
AU2019385506A1 (en) 2021-06-03
CA3120289A1 (en) 2020-05-28
CO2021008120A2 (es) 2021-08-09
JP7704998B2 (ja) 2025-07-09
MX2021005997A (es) 2021-08-11
EP3883954A1 (en) 2021-09-29

Similar Documents

Publication Publication Date Title
SG11202105326WA (en) Recombinant viral vectors and nucleic acids for producing the same
IL277666A (en) Virus vector evasion antibody
IL271993A (en) Viral vectors of cell lysis and their uses
SG11202009009UA (en) Viral and non-viral nanoplasmid vectors with improved production
IL277664A (en) Virus vector evasion antibody
EP3325633A4 (en) RECOMBINANT VECTORS COMPRISING A 2A PEPTIDE
IL288419A (en) Recombinant viral herpes vector
IL286475A (en) Methods for the manufacture of recombinant viral vectors
GB201814590D0 (en) Viral vector production system
HUE073342T2 (hu) PRRSV minor fehérjét tartalmazó rekombináns vírusvektorok és eljárások elõállításukra és alkalmazásukra
IL280840A (en) Production methods for viral vectors
EP3512871A4 (en) LONG UNIFORM RECOMBINANT PROTEIN FIBERS
WO2015134332A3 (en) Recombinant isfahan viral vectors
ZA202100859B (en) Recombinant protein variants
ZA202104244B (en) Recombinant viruses and the uses thereof
ZA202101433B (en) An improved process for the preparation of recombinant lectin protein
ZA202100242B (en) Recombinant nucleic acid construct
ZA202108996B (en) Viral vector
PT3250234T (pt) Mdv1 recombinante e suas utilizações
IL277128A (en) Hepatitis B vaccines and their uses
WO2016160976A3 (en) Monovalent tnf binding proteins
HK40053027A (en) Recombinant viral vectors and nucleic acids for producing the same
HK40067782A (en) Methods for the manufacture of recombinant viral vectors
PT3833786T (pt) Construção de ácido nucleico recombinante
HK40042266A (en) Recombinant viral vaccines