SG10201610827RA - Gene Vector - Google Patents

Gene Vector

Info

Publication number
SG10201610827RA
SG10201610827RA SG10201610827RA SG10201610827RA SG10201610827RA SG 10201610827R A SG10201610827R A SG 10201610827RA SG 10201610827R A SG10201610827R A SG 10201610827RA SG 10201610827R A SG10201610827R A SG 10201610827RA SG 10201610827R A SG10201610827R A SG 10201610827RA
Authority
SG
Singapore
Prior art keywords
gene vector
gene
vector
Prior art date
Application number
SG10201610827RA
Inventor
Alessandra Biffi
Bernhard Rudolf Gentner
Luigi Naldini
Original Assignee
Ospedale San Raffaele Srl
Fond Telethon
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 Ospedale San Raffaele Srl, Fond Telethon filed Critical Ospedale San Raffaele Srl
Publication of SG10201610827RA publication Critical patent/SG10201610827RA/en

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Classifications

    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/555Interferons [IFN]
    • C07K14/56IFN-alpha
    • 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
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • C12N2310/141MicroRNAs, miRNAs
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/51Physical structure in polymeric form, e.g. multimers, concatemers
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • 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
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted 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
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
SG10201610827RA 2009-04-30 2010-04-30 Gene Vector SG10201610827RA (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US17412409P 2009-04-30 2009-04-30

Publications (1)

Publication Number Publication Date
SG10201610827RA true SG10201610827RA (en) 2017-02-27

Family

ID=42790671

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10201610827RA SG10201610827RA (en) 2009-04-30 2010-04-30 Gene Vector
SG2011080009A SG175839A1 (en) 2009-04-30 2010-04-30 Gene vector

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG2011080009A SG175839A1 (en) 2009-04-30 2010-04-30 Gene vector

Country Status (22)

Country Link
US (4) US10287579B2 (en)
EP (2) EP2424571B1 (en)
JP (2) JP6144911B2 (en)
KR (1) KR101793615B1 (en)
CN (1) CN102596255B (en)
AU (1) AU2010243276B2 (en)
BR (1) BRPI1010873B1 (en)
CA (1) CA2759438C (en)
CY (1) CY1123218T1 (en)
DK (1) DK2424571T3 (en)
EA (1) EA023938B1 (en)
ES (1) ES2786039T3 (en)
HR (1) HRP20200647T1 (en)
HU (1) HUE049845T2 (en)
IL (1) IL215804A (en)
LT (1) LT2424571T (en)
MX (1) MX2011011508A (en)
PL (1) PL2424571T3 (en)
PT (1) PT2424571T (en)
SG (2) SG10201610827RA (en)
SI (1) SI2424571T1 (en)
WO (1) WO2010125471A2 (en)

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HUE037227T2 (en) 2010-05-14 2018-08-28 Univ Oregon Health & Science Recombinant HCMV and RHCMV vectors and uses thereof
CA2804791C (en) * 2010-07-08 2019-07-30 Duke University Direct reprogramming of cells to cardiac myocyte fate
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
WO2013152230A1 (en) * 2012-04-04 2013-10-10 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
DE202013012242U1 (en) 2012-05-25 2016-02-02 Emmanuelle Charpentier Compositions for RNA-directed modification of a target DNA and for RNA-driven modulation of transcription
JP6329537B2 (en) * 2012-07-11 2018-05-23 サンガモ セラピューティクス, インコーポレイテッド Methods and compositions for delivery of biological agents
PL3138910T3 (en) 2012-12-06 2018-01-31 Sigma Aldrich Co Llc Crispr-based genome modification and regulation
HRP20231183T1 (en) 2013-02-15 2024-01-05 Bioverativ Therapeutics Inc. Optimized factor viii gene
WO2014150624A1 (en) 2013-03-14 2014-09-25 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
CA2927712A1 (en) 2013-10-24 2015-04-30 Ospedale San Raffaele S.R.L. Method for the genetic modification of haematopoietic stem and progenitor cells (hspcs) and the use of genetically modified hspcs in gene therapy
WO2016004113A1 (en) 2014-06-30 2016-01-07 Biogen Ma Inc. Optimized factor ix gene
WO2016171235A1 (en) * 2015-04-22 2016-10-27 国立大学法人京都大学 Method for sorting tissue cells
JP2018517421A (en) * 2015-06-15 2018-07-05 マードック チルドレンズ リサーチ インスティチュート How to measure chimerism
AU2016336344A1 (en) 2015-10-05 2018-04-19 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
HRP20221089T1 (en) 2016-02-01 2022-11-25 Bioverativ Therapeutics Inc. Optimized factor viii genes
US10457940B2 (en) * 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CN110036112B (en) 2016-10-18 2024-05-10 俄勒冈健康与科学大学 Cytomegalovirus vector for priming T cells restricted by major histocompatibility complex E molecules
CN106566838B (en) * 2016-11-14 2019-11-01 上海伯豪生物技术有限公司 A kind of miR-126 full-length gene knockout kit and its application based on CRISPR-Cas9 technology
KR20200020677A (en) * 2017-04-21 2020-02-26 오스페달레 산 라파엘 에스.알.엘. Gene therapy
IT201700086403A1 (en) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
SG11202000764RA (en) 2017-08-09 2020-02-27 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
BR112020015228A2 (en) 2018-02-01 2020-12-29 Bioverativ Therapeutics Inc. USE OF LENTIVIRAL VECTORS THAT EXPRESS FACTOR VIII
JP2021533753A (en) 2018-08-09 2021-12-09 バイオベラティブ セラピューティクス インコーポレイテッド Nucleic acid molecules and their use for non-viral gene therapy
CA3121786A1 (en) 2018-12-06 2020-06-11 Bioverativ Therapeutics Inc. Use of lentiviral vectors expressing factor ix
EP4038182A1 (en) 2019-09-30 2022-08-10 Bioverativ Therapeutics Inc. Lentiviral vector formulations
AU2020384286A1 (en) * 2019-11-12 2022-06-09 The Regents Of The University Of California Lentiviral vectors in hematopoietic stem cells to treat X-linked chronic granulomatous disease
CA3183557A1 (en) 2020-06-24 2021-12-30 Bioverativ Therapeutics Inc. Methods for the removal of free factor viii from preparations of lentiviral vectors modified to express said protein
CA3189673A1 (en) 2020-08-23 2022-03-03 Ajay MAGHODIA Modified baculovirus system for improved production of closed-ended dna (cedna)
CA3229323A1 (en) 2021-08-23 2023-03-02 Ajay MAGHODIA Optimized factor viii genes
WO2023028440A2 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
AU2022355088A1 (en) 2021-09-30 2024-05-16 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
WO2024026257A2 (en) * 2022-07-25 2024-02-01 Modernatx, Inc. Engineered polynucleotides for cell selective expression

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Also Published As

Publication number Publication date
CN102596255A (en) 2012-07-18
PT2424571T (en) 2020-05-06
IL215804A (en) 2016-10-31
WO2010125471A2 (en) 2010-11-04
US11407996B2 (en) 2022-08-09
US20220333103A1 (en) 2022-10-20
JP6370278B2 (en) 2018-08-08
JP2016028582A (en) 2016-03-03
US10287579B2 (en) 2019-05-14
BRPI1010873B1 (en) 2020-04-14
IL215804A0 (en) 2012-01-31
CA2759438C (en) 2021-03-30
MX2011011508A (en) 2012-02-13
HUE049845T2 (en) 2020-10-28
HRP20200647T1 (en) 2020-10-02
LT2424571T (en) 2020-08-10
US20190367911A1 (en) 2019-12-05
US9951328B2 (en) 2018-04-24
KR101793615B1 (en) 2017-11-03
EP2424571B1 (en) 2020-04-08
BRPI1010873A2 (en) 2016-03-15
KR20120038403A (en) 2012-04-23
EP3733214A1 (en) 2020-11-04
US20120128643A1 (en) 2012-05-24
ES2786039T3 (en) 2020-10-08
EA201171335A1 (en) 2012-05-30
PL2424571T3 (en) 2020-10-19
CY1123218T1 (en) 2021-10-29
AU2010243276B2 (en) 2016-09-15
AU2010243276A1 (en) 2011-11-17
US20180044670A1 (en) 2018-02-15
EP2424571A2 (en) 2012-03-07
SG175839A1 (en) 2011-12-29
CA2759438A1 (en) 2010-11-04
JP6144911B2 (en) 2017-06-07
DK2424571T3 (en) 2020-05-04
EA023938B1 (en) 2016-07-29
CN102596255B (en) 2017-10-13
JP2012525141A (en) 2012-10-22
WO2010125471A3 (en) 2010-12-29
SI2424571T1 (en) 2020-10-30

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