BRPI1010873B1 - usos de um vetor de genes, de uma composição farmacêutica e de uma célula - Google Patents

usos de um vetor de genes, de uma composição farmacêutica e de uma célula Download PDF

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Publication number
BRPI1010873B1
BRPI1010873B1 BRPI1010873A BRPI1010873A BRPI1010873B1 BR PI1010873 B1 BRPI1010873 B1 BR PI1010873B1 BR PI1010873 A BRPI1010873 A BR PI1010873A BR PI1010873 A BRPI1010873 A BR PI1010873A BR PI1010873 B1 BRPI1010873 B1 BR PI1010873B1
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BR
Brazil
Prior art keywords
cells
mir
cell
galc
expression
Prior art date
Application number
BRPI1010873A
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English (en)
Portuguese (pt)
Inventor
Biffi Alessandra
Rudolf Gentner Bernhard
Naldini Luigi
Original Assignee
Fond Centro San Raffaele Del Monte Tabor
Fond Monte Tabor
Fond Telethon
Ospedale San Raffaele Srl
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.)
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Publication date
Application filed by Fond Centro San Raffaele Del Monte Tabor, Fond Monte Tabor, Fond Telethon, Ospedale San Raffaele Srl filed Critical Fond Centro San Raffaele Del Monte Tabor
Publication of BRPI1010873A2 publication Critical patent/BRPI1010873A2/pt
Publication of BRPI1010873B1 publication Critical patent/BRPI1010873B1/pt

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    • 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
    • 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
    • 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
    • C12N15/11DNA 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
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • 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 nucleic acids [NA]
    • 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

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  • 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)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (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)
BRPI1010873A 2009-04-30 2010-04-30 usos de um vetor de genes, de uma composição farmacêutica e de uma célula BRPI1010873B1 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US17412409P 2009-04-30 2009-04-30
PCT/IB2010/001166 WO2010125471A2 (en) 2009-04-30 2010-04-30 Gene vector

Publications (2)

Publication Number Publication Date
BRPI1010873A2 BRPI1010873A2 (pt) 2016-03-15
BRPI1010873B1 true BRPI1010873B1 (pt) 2020-04-14

Family

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Family Applications (1)

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BRPI1010873A BRPI1010873B1 (pt) 2009-04-30 2010-04-30 usos de um vetor de genes, de uma composição farmacêutica e de uma célula

Country Status (23)

Country Link
US (4) US10287579B2 (cg-RX-API-DMAC7.html)
EP (2) EP3733214A1 (cg-RX-API-DMAC7.html)
JP (2) JP6144911B2 (cg-RX-API-DMAC7.html)
KR (1) KR101793615B1 (cg-RX-API-DMAC7.html)
CN (1) CN102596255B (cg-RX-API-DMAC7.html)
AU (1) AU2010243276B2 (cg-RX-API-DMAC7.html)
BR (1) BRPI1010873B1 (cg-RX-API-DMAC7.html)
CA (1) CA2759438C (cg-RX-API-DMAC7.html)
CY (1) CY1123218T1 (cg-RX-API-DMAC7.html)
DK (1) DK2424571T3 (cg-RX-API-DMAC7.html)
EA (1) EA023938B1 (cg-RX-API-DMAC7.html)
ES (1) ES2786039T3 (cg-RX-API-DMAC7.html)
HR (1) HRP20200647T1 (cg-RX-API-DMAC7.html)
HU (1) HUE049845T2 (cg-RX-API-DMAC7.html)
IL (1) IL215804A (cg-RX-API-DMAC7.html)
LT (1) LT2424571T (cg-RX-API-DMAC7.html)
MX (1) MX2011011508A (cg-RX-API-DMAC7.html)
PL (1) PL2424571T3 (cg-RX-API-DMAC7.html)
PT (1) PT2424571T (cg-RX-API-DMAC7.html)
SG (2) SG175839A1 (cg-RX-API-DMAC7.html)
SI (1) SI2424571T1 (cg-RX-API-DMAC7.html)
SM (1) SMT202000224T1 (cg-RX-API-DMAC7.html)
WO (1) WO2010125471A2 (cg-RX-API-DMAC7.html)

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WO2012006577A2 (en) * 2010-07-08 2012-01-12 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
GB2537000C (en) 2012-05-25 2019-10-09 Univ California Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
CA2878037C (en) * 2012-07-11 2021-08-31 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
CA2891347C (en) 2012-12-06 2018-02-27 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
PL2956477T5 (pl) 2013-02-15 2024-05-27 Bioverativ Therapeutics Inc. Zoptymalizowany gen czynnika viii
AU2014235794A1 (en) 2013-03-14 2015-10-22 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
EP3060670B1 (en) 2013-10-24 2019-07-10 Ospedale San Raffaele S.r.l. Method
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
EP3318630B1 (en) * 2015-04-22 2020-11-04 iPS Academia Japan, Inc. Method for sorting tissue cells
WO2016201507A1 (en) * 2015-06-15 2016-12-22 Murdoch Childrens Research Institute Method of measuring chimerism
US10849920B2 (en) 2015-10-05 2020-12-01 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
RS63548B1 (sr) 2016-02-01 2022-09-30 Bioverativ Therapeutics Inc Optimizovani geni faktora viii
GB201612214D0 (en) 2016-07-14 2016-08-31 Univ Oxford Innovation Ltd Method
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CA3040303A1 (en) * 2016-10-18 2018-04-26 Oregon Health & Science University Cytomegalovirus vectors eliciting t cells restricted by major histocompatibility complex e molecules
CN106566838B (zh) * 2016-11-14 2019-11-01 上海伯豪生物技术有限公司 一种基于CRISPR-Cas9技术的miR-126全长基因敲除试剂盒及其应用
WO2018193119A1 (en) * 2017-04-21 2018-10-25 Ospedale San Raffaele S.R.L. Gene therapy
IT201700086403A1 (it) * 2017-07-27 2019-01-27 Univ Del Piemonte Orientale Endothelial-specific promoter sequences and uses thereof
KR20250040746A (ko) 2017-08-09 2025-03-24 바이오버라티브 테라퓨틱스 인크. 핵산 분자 및 이의 용도
CN111918674A (zh) 2018-02-01 2020-11-10 比奥维拉迪维治疗股份有限公司 表达因子viii的慢病毒载体的用途
JP7602454B2 (ja) 2018-08-09 2024-12-18 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 核酸分子及び非ウイルス遺伝子治療のためのそれらの使用
SG11202105880TA (en) 2018-12-06 2021-07-29 Bioverativ Therapeutics Inc Use of lentiviral vectors expressing factor ix
US12403164B2 (en) 2019-09-30 2025-09-02 Bioverativ Therapeutics Inc. Lentiviral vector formulations
CA3161175A1 (en) * 2019-11-12 2021-05-20 The Regents Of The University Of California Lentiviral vectors in hematopoietic stem cells to treat x-linked chronic granulomatous disease
BR112022026127A2 (pt) 2020-06-24 2023-01-17 Bioverativ Therapeutics Inc Métodos para remoção de fator viii livre de preparações de vetores lentivirais modificados para expressar referida proteína
AU2021330936A1 (en) 2020-08-23 2023-05-04 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
JP2024534123A (ja) 2021-08-23 2024-09-18 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 バキュロウイルス発現システム
TW202323274A (zh) 2021-08-23 2023-06-16 美商百歐維拉提夫治療公司 優化因子viii基因
CN115725656A (zh) * 2021-09-01 2023-03-03 联邦生物科技(珠海横琴)有限公司 一种以细胞质rna病毒为载体递送干扰rna的方法
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WO2025122739A1 (en) * 2023-12-05 2025-06-12 University Of Pittsburgh-Of The Commonwealth System Of Higher Education Compositions and methods for treating a heart disease

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Publication number Publication date
US20120128643A1 (en) 2012-05-24
JP2016028582A (ja) 2016-03-03
SG10201610827RA (en) 2017-02-27
CY1123218T1 (el) 2021-10-29
IL215804A0 (en) 2012-01-31
LT2424571T (lt) 2020-08-10
IL215804A (en) 2016-10-31
DK2424571T3 (da) 2020-05-04
EA023938B1 (ru) 2016-07-29
AU2010243276B2 (en) 2016-09-15
ES2786039T3 (es) 2020-10-08
KR101793615B1 (ko) 2017-11-03
MX2011011508A (es) 2012-02-13
JP6370278B2 (ja) 2018-08-08
WO2010125471A3 (en) 2010-12-29
CN102596255A (zh) 2012-07-18
WO2010125471A2 (en) 2010-11-04
US20190367911A1 (en) 2019-12-05
EP2424571A2 (en) 2012-03-07
JP6144911B2 (ja) 2017-06-07
US9951328B2 (en) 2018-04-24
PL2424571T3 (pl) 2020-10-19
US10287579B2 (en) 2019-05-14
EP3733214A1 (en) 2020-11-04
CA2759438A1 (en) 2010-11-04
US20180044670A1 (en) 2018-02-15
HUE049845T2 (hu) 2020-10-28
AU2010243276A1 (en) 2011-11-17
JP2012525141A (ja) 2012-10-22
US20220333103A1 (en) 2022-10-20
HRP20200647T1 (hr) 2020-10-02
EA201171335A1 (ru) 2012-05-30
SI2424571T1 (sl) 2020-10-30
CN102596255B (zh) 2017-10-13
US11407996B2 (en) 2022-08-09
SG175839A1 (en) 2011-12-29
CA2759438C (en) 2021-03-30
SMT202000224T1 (it) 2020-05-08
EP2424571B1 (en) 2020-04-08
PT2424571T (pt) 2020-05-06
BRPI1010873A2 (pt) 2016-03-15
KR20120038403A (ko) 2012-04-23

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B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]
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