MX2017003426A - Vectores virales adeno-asociados para tratar glaucoma de miocilina (myoc). - Google Patents

Vectores virales adeno-asociados para tratar glaucoma de miocilina (myoc).

Info

Publication number
MX2017003426A
MX2017003426A MX2017003426A MX2017003426A MX2017003426A MX 2017003426 A MX2017003426 A MX 2017003426A MX 2017003426 A MX2017003426 A MX 2017003426A MX 2017003426 A MX2017003426 A MX 2017003426A MX 2017003426 A MX2017003426 A MX 2017003426A
Authority
MX
Mexico
Prior art keywords
myoc
spondin
adeno
glaucoma
viral vectors
Prior art date
Application number
MX2017003426A
Other languages
English (en)
Spanish (es)
Inventor
Ardinger Jeffery
Scaria Abraham
Pechan Peter
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Priority claimed from PCT/US2015/050515 external-priority patent/WO2016044478A1/en
Publication of MX2017003426A publication Critical patent/MX2017003426A/es

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    • 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
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K9/0048Eye, e.g. artificial tears
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides 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
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/06Antiglaucoma agents or miotics
    • 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
    • CCHEMISTRY; METALLURGY
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    • 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
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    • 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
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
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    • 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
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    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • CCHEMISTRY; METALLURGY
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    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • A61K48/0075Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N2750/14132Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • CCHEMISTRY; METALLURGY
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    • 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
    • C12N2750/14171Demonstrated in vivo effect

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  • Biomedical Technology (AREA)
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  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
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  • Biophysics (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (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)
  • Peptides Or Proteins (AREA)
MX2017003426A 2014-09-16 2015-09-16 Vectores virales adeno-asociados para tratar glaucoma de miocilina (myoc). MX2017003426A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462051299P 2014-09-16 2014-09-16
PCT/US2015/050515 WO2016044478A1 (en) 2014-09-16 2015-09-16 Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

Publications (1)

Publication Number Publication Date
MX2017003426A true MX2017003426A (es) 2017-07-28

Family

ID=58640637

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017003426A MX2017003426A (es) 2014-09-16 2015-09-16 Vectores virales adeno-asociados para tratar glaucoma de miocilina (myoc).
MX2023005998A MX2023005998A (es) 2014-09-16 2017-03-15 Vectores virales adeno-asociados para tratar glaucoma de miocilina (myoc).

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2023005998A MX2023005998A (es) 2014-09-16 2017-03-15 Vectores virales adeno-asociados para tratar glaucoma de miocilina (myoc).

Country Status (13)

Country Link
US (1) US10821193B2 (enExample)
EP (1) EP3194601B1 (enExample)
JP (2) JP6673923B2 (enExample)
KR (3) KR20260019013A (enExample)
CN (2) CN116585492A (enExample)
AU (2) AU2015317756B2 (enExample)
BR (1) BR112017005235B1 (enExample)
CA (1) CA2961523C (enExample)
IL (1) IL251178B (enExample)
MX (2) MX2017003426A (enExample)
RU (2) RU2718047C2 (enExample)
SG (3) SG10201913430VA (enExample)
ZA (1) ZA201701881B (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL251178B (en) 2014-09-16 2022-07-01 Genzyme Corp Adeno-associated viral vector for the treatment of glaucoma myocilin (myoc)
US11845952B2 (en) * 2018-02-07 2023-12-19 Nippon Medical School Foundation Adeno-associated virus vector
US20210030719A1 (en) * 2018-02-22 2021-02-04 University Of South Florida Grp94 inhibitors to treat steroid-induced ocular hypertensions and glaucomas
WO2020176747A1 (en) 2019-02-28 2020-09-03 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
US11674154B2 (en) * 2020-12-03 2023-06-13 University Of Massachusetts Gene therapeutics for fibrodysplasia ossificans progressiva
CN113032010B (zh) * 2021-03-12 2022-09-20 歌尔科技有限公司 命令的传输控制方法、终端及计算机可读存储介质
WO2023278406A1 (en) * 2021-06-28 2023-01-05 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
KR20240117571A (ko) * 2021-12-08 2024-08-01 리제너론 파마슈티칼스 인코포레이티드 돌연변이 마이오실린 질환 모델 및 이의 용도
WO2023114446A2 (en) * 2021-12-16 2023-06-22 Peter Koulen A machine learning-based framework using electroretinography for detecting early-stage glaucoma
CN117343152B (zh) * 2023-04-18 2024-06-04 上海本导基因技术有限公司 一种用于治疗青光眼疾病的慢病毒样颗粒
CN118546217B (zh) * 2024-07-25 2024-10-29 广州译码基因科技有限公司 靶向小梁网、角膜缘及rpe的aav衣壳蛋白及其应用
CN121449764A (zh) * 2026-01-07 2026-02-03 山东伯桢生物科技有限公司 抑制Wnt信号通路的融合蛋白及其应用

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2462795A1 (en) 2001-10-03 2003-04-10 Incyte Genomics, Inc. Secreted proteins
GB0403600D0 (en) * 2004-02-18 2004-03-24 Trinity College Dublin Methods and reagents for treating disease
WO2005117938A2 (en) * 2004-04-13 2005-12-15 Regents Of The University Of Minnesota Methods of treating ocular conditions
WO2008075796A1 (ja) * 2006-12-21 2008-06-26 Kyowa Hakko Kirin Co., Ltd. 血球回復促進剤
KR20100061792A (ko) * 2007-10-01 2010-06-09 알콘 리서치, 리미티드 안 질환을 치료 또는 예방하기 위한 간섭 rna 분자의 자기-상보적 aav 매개 전달
GB201106395D0 (en) * 2011-04-14 2011-06-01 Hubrecht Inst Compounds
DK4005603T3 (da) * 2011-04-22 2024-11-25 Univ California Adeno-associerede virus-virioner med variant kapsid og fremgangsmåder til anvendelse heraf
EP2554662A1 (en) * 2011-08-05 2013-02-06 M Maria Pia Cosma Methods of treatment of retinal degeneration diseases
MX366804B (es) * 2012-02-11 2019-07-25 Genentech Inc Translocaciones de la r-espondina y sus metodos de uso.
WO2013170078A1 (en) * 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
IL251178B (en) 2014-09-16 2022-07-01 Genzyme Corp Adeno-associated viral vector for the treatment of glaucoma myocilin (myoc)

Also Published As

Publication number Publication date
CN107001436B (zh) 2023-01-06
JP6673923B2 (ja) 2020-03-25
JP2017529395A (ja) 2017-10-05
JP7097398B2 (ja) 2022-07-07
EP3194601B1 (en) 2021-10-27
KR20230172610A (ko) 2023-12-22
SG11201702055PA (en) 2017-04-27
MX2023005998A (es) 2023-06-08
BR112017005235A2 (pt) 2017-12-12
RU2017112972A (ru) 2018-10-17
RU2746991C2 (ru) 2021-04-23
BR112017005235B1 (pt) 2023-10-31
CN116585492A (zh) 2023-08-15
CA2961523C (en) 2026-01-13
US10821193B2 (en) 2020-11-03
US20170304465A1 (en) 2017-10-26
RU2020111038A3 (enExample) 2020-10-15
KR20260019013A (ko) 2026-02-09
RU2017112972A3 (enExample) 2019-04-16
CN107001436A (zh) 2017-08-01
AU2015317756B2 (en) 2022-01-13
KR102612871B1 (ko) 2023-12-13
AU2015317756A1 (en) 2017-04-13
CA2961523A1 (en) 2016-03-24
RU2718047C2 (ru) 2020-03-30
RU2020111038A (ru) 2020-04-29
SG10201902285SA (en) 2019-04-29
AU2022202413A1 (en) 2022-05-05
KR102916865B1 (ko) 2026-01-23
JP2020105194A (ja) 2020-07-09
IL251178B (en) 2022-07-01
ZA201701881B (en) 2023-09-27
KR20170054493A (ko) 2017-05-17
EP3194601A1 (en) 2017-07-26
IL251178A0 (en) 2017-05-29
SG10201913430VA (en) 2020-03-30

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