KR102848324B1 - 거대 아데노-연관 벡터의 생산 - Google Patents

거대 아데노-연관 벡터의 생산

Info

Publication number
KR102848324B1
KR102848324B1 KR1020177032062A KR20177032062A KR102848324B1 KR 102848324 B1 KR102848324 B1 KR 102848324B1 KR 1020177032062 A KR1020177032062 A KR 1020177032062A KR 20177032062 A KR20177032062 A KR 20177032062A KR 102848324 B1 KR102848324 B1 KR 102848324B1
Authority
KR
South Korea
Prior art keywords
delete delete
aav
raav
genome
aav2
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
KR1020177032062A
Other languages
English (en)
Korean (ko)
Other versions
KR20170133500A (ko
Inventor
시르카 쿄스티오-무어
데이빗 소우자
카렌 빈센트
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=56943906&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=KR102848324(B1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 젠자임 코포레이션 filed Critical 젠자임 코포레이션
Priority to KR1020257027291A priority Critical patent/KR20250126875A/ko
Publication of KR20170133500A publication Critical patent/KR20170133500A/ko
Application granted granted Critical
Publication of KR102848324B1 publication Critical patent/KR102848324B1/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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a 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
    • C12N2750/14141—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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Epidemiology (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020177032062A 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산 Active KR102848324B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020257027291A KR20250126875A (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562144862P 2015-04-08 2015-04-08
US62/144,862 2015-04-08
US201562220067P 2015-09-17 2015-09-17
US62/220,067 2015-09-17
PCT/US2016/026486 WO2016164609A2 (en) 2015-04-08 2016-04-07 Production of oversized adeno-associated vectors

Related Child Applications (1)

Application Number Title Priority Date Filing Date
KR1020257027291A Division KR20250126875A (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산

Publications (2)

Publication Number Publication Date
KR20170133500A KR20170133500A (ko) 2017-12-05
KR102848324B1 true KR102848324B1 (ko) 2025-08-20

Family

ID=56943906

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020177032062A Active KR102848324B1 (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산
KR1020257027291A Pending KR20250126875A (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산

Family Applications After (1)

Application Number Title Priority Date Filing Date
KR1020257027291A Pending KR20250126875A (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산

Country Status (27)

Country Link
US (1) US10815497B2 (enExample)
EP (2) EP3280799B1 (enExample)
JP (3) JP6878299B2 (enExample)
KR (2) KR102848324B1 (enExample)
CN (1) CN107864657A (enExample)
AU (3) AU2016245806A1 (enExample)
BR (1) BR112017021505A2 (enExample)
CA (1) CA2982123A1 (enExample)
CL (1) CL2017002537A1 (enExample)
CO (1) CO2017011344A2 (enExample)
CR (1) CR20170505A (enExample)
DK (1) DK3280799T3 (enExample)
EA (1) EA201792236A1 (enExample)
EC (1) ECSP17074016A (enExample)
ES (1) ES3008382T3 (enExample)
GT (1) GT201700214A (enExample)
HU (1) HUE069779T2 (enExample)
IL (3) IL283291B (enExample)
MX (1) MX2017012935A (enExample)
PE (1) PE20171800A1 (enExample)
PH (1) PH12017501836A1 (enExample)
PL (1) PL3280799T3 (enExample)
SG (3) SG10201912912QA (enExample)
TN (1) TN2017000431A1 (enExample)
TW (1) TWI707951B (enExample)
UY (1) UY36611A (enExample)
WO (1) WO2016164609A2 (enExample)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2956477T4 (da) 2013-02-15 2024-04-15 Bioverativ Therapeutics Inc Optimeret faktor viii-gen
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
US11008561B2 (en) 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
CN112553229A (zh) 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
RU2020108189A (ru) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. Композиции и способы лечения бокового амиотрофического склероза (als)
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
EP3230441A4 (en) 2014-12-12 2018-10-03 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
TWI707951B (zh) * 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
BR112018006074A2 (pt) 2015-09-24 2018-10-09 Biomarin Pharm Inc vetores de fator viii de vírus adeno-associado, partículas virais associadas e formulações terapêuticas compreendendo as mesmas
KR20180118659A (ko) 2016-02-01 2018-10-31 바이오버라티브 테라퓨틱스 인크. 최적화된 viii 인자 유전자
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics, Inc. composições e métodos de tratamento da doença de huntington
AU2017267665C1 (en) 2016-05-18 2023-10-05 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
WO2018154096A1 (en) 2017-02-24 2018-08-30 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject
CA3061652A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
KR102636351B1 (ko) 2017-05-19 2024-02-15 엔코디드 테라퓨틱스, 인크. 고활성 조절 요소
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CN111132626B (zh) 2017-07-17 2024-01-30 沃雅戈治疗公司 轨迹阵列引导系统
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
TWI904068B (zh) 2017-08-09 2025-11-11 美商生物化學醫療公司 核酸分子及其用途
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
SG11202004926WA (en) 2017-12-01 2020-06-29 Encoded Therapeutics Inc Engineered dna binding proteins
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
US20210338838A1 (en) * 2018-07-20 2021-11-04 University Of Utah Research Foundation Gene therapy for macular degeneration
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
EP4461814A3 (en) * 2018-10-12 2025-02-26 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
WO2021042004A1 (en) * 2019-08-29 2021-03-04 Siemens Healthcare Diagnostics Inc. Reagents and methods for detecting aav shedding
TW202126284A (zh) 2019-09-30 2021-07-16 美商百歐維拉提夫治療公司 慢病毒載體配製物
CN111218446B (zh) * 2019-12-25 2023-03-28 劲帆生物医药科技（武汉）有限公司 一种肝脏特异性启动子及其应用
IL296509A (en) * 2020-03-16 2022-11-01 Univ North Carolina Chapel Hill Preparations and methods for selective detection of tumor-derived viral DNA
WO2021262963A1 (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
JP2023536148A (ja) 2020-07-30 2023-08-23 シェイプ セラピューティクス インク． Ｒａａｖウイルス粒子の誘導産生のための安定な細胞株
BR112023002395A2 (pt) 2020-08-23 2023-03-21 Bioverativ Therapeutics Inc Sistema de baculovírus modificado para produção aprimorada de dna com extremidades fechadas (cedna)
CA3229668A1 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
AR126845A1 (es) 2021-08-23 2023-11-22 Bioverativ Therapeutics Inc Producción de adn de extremo cerrado con secuencias repetidas de terminal invertida
KR20240049332A (ko) 2021-08-23 2024-04-16 바이오버라티브 테라퓨틱스 인크. 최적화된 인자 viii 유전자
EP4408874A1 (en) 2021-09-30 2024-08-07 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
JP2024537896A (ja) 2021-10-18 2024-10-16 リジェネロン・ファーマシューティカルズ・インコーポレイテッド アデノウイルス随伴ウイルスポリヌクレオチドを含む真核細胞
TW202509211A (zh) * 2023-04-26 2025-03-01 法商賽諾菲公司 宿主細胞系以及鑑定和使用該等宿主細胞系之方法

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ATE403715T1 (de) 1999-08-09 2008-08-15 Targeted Genetics Corp Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
WO2001027303A1 (en) * 1999-10-12 2001-04-19 The University Of North Carolina At Chapel Hill Adeno-associated virus vectors encoding factor viii and methods of using the same
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
CN103555677B (zh) 2001-11-13 2018-01-30 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
US20040092008A1 (en) * 2002-11-12 2004-05-13 Snyder Richard O. Recombinant AAV vectors for gene therapy of hemophilia A
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
ES2521682T3 (es) 2003-05-21 2014-11-13 Genzyme Corporation Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
HUE040636T2 (hu) 2009-06-16 2019-03-28 Genzyme Corp Javított eljárások rekombináns AAV-vektorok tisztítására
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
ES2724800T3 (es) 2011-02-17 2019-09-16 Univ Pennsylvania Composiciones y métodos para alterar la especificidad de tejido y mejorar la transferencia génica mediada por AAV9
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
ES2813698T3 (es) 2013-09-12 2021-03-24 Biomarin Pharm Inc Vectores de AAV que comprenden un gen que codifica el factor VIII
WO2015168666A2 (en) 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
NZ772772A (en) 2015-02-10 2024-12-20 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
TWI707951B (zh) * 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Hum. Gene Ther., Vol.22, No.5, pp.613-624(2011.03.18.)*

Also Published As

Publication number Publication date
EP3280799B1 (en) 2024-10-30
NZ774736A (en) 2024-08-30
AU2022203942B2 (en) 2023-12-14
PH12017501836A1 (en) 2018-04-23
IL254933A0 (en) 2017-12-31
EP4512429A3 (en) 2025-05-21
CL2017002537A1 (es) 2018-05-11
IL294965A (en) 2022-09-01
KR20250126875A (ko) 2025-08-25
WO2016164609A2 (en) 2016-10-13
AU2016245806A1 (en) 2017-11-30
NZ737009A (en) 2024-01-26
SG10201909381WA (en) 2019-11-28
DK3280799T3 (da) 2025-01-27
TW201710498A (zh) 2017-03-16
UY36611A (es) 2016-11-30
JP6878299B2 (ja) 2021-05-26
EA201792236A1 (ru) 2018-03-30
CA2982123A1 (en) 2016-10-13
GT201700214A (es) 2018-10-24
TN2017000431A1 (en) 2019-04-12
IL283291A (en) 2021-07-29
TWI707951B (zh) 2020-10-21
HK1246339A1 (en) 2018-09-07
AU2024201619A1 (en) 2024-03-28
AU2022203942A1 (en) 2022-06-23
US20190048362A1 (en) 2019-02-14
SG10201912912QA (en) 2020-02-27
PL3280799T3 (pl) 2025-04-14
EP4512429A2 (en) 2025-02-26
IL283291B (en) 2022-08-01
US10815497B2 (en) 2020-10-27
HUE069779T2 (hu) 2025-04-28
JP2023116678A (ja) 2023-08-22
PE20171800A1 (es) 2017-12-28
WO2016164609A3 (en) 2016-12-15
CO2017011344A2 (es) 2018-03-28
EP3280799A2 (en) 2018-02-14
ECSP17074016A (es) 2018-02-28
JP2021118724A (ja) 2021-08-12
MX2017012935A (es) 2018-02-12
JP2018510648A (ja) 2018-04-19
ES3008382T3 (en) 2025-03-24
KR20170133500A (ko) 2017-12-05
BR112017021505A2 (pt) 2018-07-03
CR20170505A (es) 2018-01-26
SG11201708203UA (en) 2017-11-29
IL254933B (en) 2021-06-30
CN107864657A (zh) 2018-03-30

Similar Documents

Publication Publication Date Title
KR102848324B1 (ko) 거대 아데노-연관 벡터의 생산
JP7814430B2 (ja) 深部イントロン突然変異の遺伝子編集
US12201698B2 (en) Gene therapy for retinitis pigmentosa
JP2021118740A (ja) バリアントＲＮＡｉ
JP2020535803A (ja) バリアントＲＮＡｉ
HK40120936A (en) Production of oversized adeno-associated vectors
HK40026944B (en) Gene therapy for retinitis pigmentosa
HK1246339B (en) Production of oversized adeno-associated vectors
HK1252469B (en) Gene editing of deep intronic mutations

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

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

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

R17-X000 Change to representative recorded

St.27 status event code: A-3-3-R10-R17-oth-X000

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-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

PA0201 Request for examination

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

R17-X000 Change to representative recorded

St.27 status event code: A-3-3-R10-R17-oth-X000

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

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

T11-X000 Administrative time limit extension requested

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

T11-X000 Administrative time limit extension requested

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

T11-X000 Administrative time limit extension requested

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

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-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

PE0902 Notice of grounds for rejection

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

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

E701 Decision to grant or registration of patent right
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)

PA0104 Divisional application for international application

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

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)

G17 Pta granted

Free format text: ST27 STATUS EVENT CODE: A-4-4-G10-G17-TAD-PR1102 (AS PROVIDED BY THE NATIONAL OFFICE)

PR1102 Extension of patent period due to registration delay

St.27 status event code: A-4-4-G10-G17-tad-PR1102

Protection beyond ip right term event data comment text: Claim Total Quantity : 0, Period Limitation Text : 161