KR102916865B1 - 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 - Google Patents

마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

Info

Publication number
KR102916865B1
KR102916865B1 KR1020237042363A KR20237042363A KR102916865B1 KR 102916865 B1 KR102916865 B1 KR 102916865B1 KR 1020237042363 A KR1020237042363 A KR 1020237042363A KR 20237042363 A KR20237042363 A KR 20237042363A KR 102916865 B1 KR102916865 B1 KR 102916865B1
Authority
KR
South Korea
Prior art keywords
myoc
raav
rspo1
cys
promoter
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
KR1020237042363A
Other languages
English (en)
Korean (ko)
Other versions
KR20230172610A (ko
Inventor
피터 퍼찬
에이브러햄 스카리아
제프리 아딘저
Original Assignee
젠자임 코포레이션
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 젠자임 코포레이션 filed Critical 젠자임 코포레이션
Priority to KR1020267001828A priority Critical patent/KR20260019013A/ko
Priority claimed from PCT/US2015/050515 external-priority patent/WO2016044478A1/en
Publication of KR20230172610A publication Critical patent/KR20230172610A/ko
Application granted granted Critical
Publication of KR102916865B1 publication Critical patent/KR102916865B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0075—Medicinal 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
    • 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
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • 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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • 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)
KR1020237042363A 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 Active KR102916865B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020267001828A KR20260019013A (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201462051299P 2014-09-16 2014-09-16
US62/051,299 2014-09-16
PCT/US2015/050515 WO2016044478A1 (en) 2014-09-16 2015-09-16 Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
KR1020177010012A KR102612871B1 (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020177010012A Division KR102612871B1 (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

Related Child Applications (1)

Application Number Title Priority Date Filing Date
KR1020267001828A Division KR20260019013A (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

Publications (2)

Publication Number Publication Date
KR20230172610A KR20230172610A (ko) 2023-12-22
KR102916865B1 true KR102916865B1 (ko) 2026-01-23

Family

ID=58640637

Family Applications (3)

Application Number Title Priority Date Filing Date
KR1020177010012A Active KR102612871B1 (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
KR1020267001828A Pending KR20260019013A (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
KR1020237042363A Active KR102916865B1 (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

Family Applications Before (2)

Application Number Title Priority Date Filing Date
KR1020177010012A Active KR102612871B1 (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
KR1020267001828A Pending KR20260019013A (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

Country Status (13)

Country Link
US (1) US10821193B2 (https=)
EP (1) EP3194601B1 (https=)
JP (2) JP6673923B2 (https=)
KR (3) KR102612871B1 (https=)
CN (2) CN107001436B (https=)
AU (2) AU2015317756B2 (https=)
BR (1) BR112017005235B1 (https=)
CA (2) CA3280614A1 (https=)
IL (1) IL251178B (https=)
MX (2) MX2017003426A (https=)
RU (2) RU2718047C2 (https=)
SG (3) SG11201702055PA (https=)
ZA (1) ZA201701881B (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201702055PA (en) 2014-09-16 2017-04-27 Genzyme Corp Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
WO2019155833A1 (ja) * 2018-02-07 2019-08-15 学校法人日本医科大学 改良型アデノ随伴ウイルスベクター
WO2019165241A1 (en) * 2018-02-22 2019-08-29 University Of South Florida Grp94 inhibitors to treat steroid-induced ocular hypertensions and glaucomas
US20220047618A1 (en) 2019-02-28 2022-02-17 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
KR20230117177A (ko) * 2020-12-03 2023-08-07 유니버시티 오브 매사추세츠 진행성 골화성 섬유이형성증을 위한 신규 유전자 치료제의개발
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
US20250087355A1 (en) * 2021-12-16 2025-03-13 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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008075796A1 (ja) 2006-12-21 2008-06-26 Kyowa Hakko Kirin Co., Ltd. 血球回復促進剤
JP2010540564A (ja) 2007-10-01 2010-12-24 アルコン リサーチ， リミテッド 自己相補的なａａｖが媒介する眼の障害を処置または予防するための干渉ｒｎａ分子の送達
JP2014518614A (ja) 2011-04-22 2014-08-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法

Family Cites Families (8)

* 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
GB201106395D0 (en) * 2011-04-14 2011-06-01 Hubrecht Inst Compounds
EP2554662A1 (en) * 2011-08-05 2013-02-06 M Maria Pia Cosma Methods of treatment of retinal degeneration diseases
KR102148303B1 (ko) * 2012-02-11 2020-08-26 제넨테크, 인크. R-스폰딘 전위 및 그의 사용 방법
WO2013170078A1 (en) * 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
SG11201702055PA (en) 2014-09-16 2017-04-27 Genzyme Corp Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008075796A1 (ja) 2006-12-21 2008-06-26 Kyowa Hakko Kirin Co., Ltd. 血球回復促進剤
JP2010540564A (ja) 2007-10-01 2010-12-24 アルコン リサーチ， リミテッド 自己相補的なａａｖが媒介する眼の障害を処置または予防するための干渉ｒｎａ分子の送達
JP2014518614A (ja) 2011-04-22 2014-08-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法

Also Published As

Publication number Publication date
JP6673923B2 (ja) 2020-03-25
ZA201701881B (en) 2023-09-27
KR20260019013A (ko) 2026-02-09
AU2022202413A1 (en) 2022-05-05
KR20230172610A (ko) 2023-12-22
SG10201913430VA (en) 2020-03-30
MX2017003426A (es) 2017-07-28
JP2020105194A (ja) 2020-07-09
EP3194601B1 (en) 2021-10-27
RU2718047C2 (ru) 2020-03-30
US10821193B2 (en) 2020-11-03
IL251178B (en) 2022-07-01
CN116585492A (zh) 2023-08-15
RU2746991C2 (ru) 2021-04-23
EP3194601A1 (en) 2017-07-26
CA2961523A1 (en) 2016-03-24
AU2015317756B2 (en) 2022-01-13
CN107001436B (zh) 2023-01-06
SG10201902285SA (en) 2019-04-29
BR112017005235A2 (pt) 2017-12-12
IL251178A0 (en) 2017-05-29
RU2020111038A (ru) 2020-04-29
JP2017529395A (ja) 2017-10-05
RU2017112972A3 (https=) 2019-04-16
CA2961523C (en) 2026-01-13
JP7097398B2 (ja) 2022-07-07
US20170304465A1 (en) 2017-10-26
RU2017112972A (ru) 2018-10-17
CN107001436A (zh) 2017-08-01
MX2023005998A (es) 2023-06-08
RU2020111038A3 (https=) 2020-10-15
SG11201702055PA (en) 2017-04-27
AU2015317756A1 (en) 2017-04-13
BR112017005235B1 (pt) 2023-10-31
KR102612871B1 (ko) 2023-12-13
CA3280614A1 (en) 2026-03-02
KR20170054493A (ko) 2017-05-17

Similar Documents

Publication Publication Date Title
KR102916865B1 (ko) 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
AU2021200242B9 (en) AAV vectors for retinal and CNS gene therapy
JP2020059737A (ja) 網膜色素変性症のための遺伝子治療
ES3011359T3 (en) Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
EP4012035B1 (en) Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
HK40076538B (en) Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
HK40076538A (en) Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
HK1241924B (en) Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
HK40071052A (en) Aav vectors for retinal and cns gene therapy

Legal Events

Date Code Title Description
A107 Divisional application of patent
PA0104 Divisional application for international application

St.27 status event code: A-0-1-A10-A18-div-PA0104

St.27 status event code: A-0-1-A10-A16-div-PA0104

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

D22 Grant of ip right intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D22-EXM-PE0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701

A16 Divisional, continuation or continuation in part application filed

Free format text: ST27 STATUS EVENT CODE: A-0-1-A10-A16-DIV-PA0104 (AS PROVIDED BY THE NATIONAL OFFICE)

F11 Ip right granted following substantive examination

Free format text: ST27 STATUS EVENT CODE: A-2-4-F10-F11-EXM-PR0701 (AS PROVIDED BY THE NATIONAL OFFICE)

PA0104 Divisional application for international application

St.27 status event code: A-0-1-A10-A16-div-PA0104

PR0701 Registration of establishment

St.27 status event code: A-2-4-F10-F11-exm-PR0701

PR1002 Payment of registration fee

St.27 status event code: A-2-2-U10-U12-oth-PR1002

Fee payment year number: 1

U12 Designation fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U12-OTH-PR1002 (AS PROVIDED BY THE NATIONAL OFFICE)

Year of fee payment: 1

PG1601 Publication of registration

St.27 status event code: A-4-4-Q10-Q13-nap-PG1601

Q13 Ip right document published

Free format text: ST27 STATUS EVENT CODE: A-4-4-Q10-Q13-NAP-PG1601 (AS PROVIDED BY THE NATIONAL OFFICE)