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)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (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)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pulmonology (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
KR1020257027291A Pending KR20250126875A (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산
KR1020177032062A Active KR102848324B1 (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산

Family Applications Before (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) KR20250126875A (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
LT2956477T (lt) 2013-02-15 2021-02-10 Bioverativ Therapeutics Inc. Optimizuotas faktoriaus viii genas
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3160478A4 (en) 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Optimized factor ix gene
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
KR102584655B1 (ko) 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
US11697825B2 (en) 2014-12-12 2023-07-11 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
MA42934A (fr) 2015-09-24 2018-08-01 Biomarin Pharm Inc Vecteurs de facteur viii à virus adéno-associé, particules virales associées et formulations thérapeutiques comprenant ceux-ci
PL3411478T3 (pl) 2016-02-01 2022-10-03 Bioverativ Therapeutics Inc. Geny zoptymalizowanego czynnika VIII
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
JP2019531787A (ja) 2016-08-30 2019-11-07 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 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
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
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
KR20240024326A (ko) * 2017-05-19 2024-02-23 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
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
JP7374883B2 (ja) 2017-08-09 2023-11-07 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 核酸分子およびその使用
EP3687582A4 (en) 2017-09-29 2021-07-14 Voyager Therapeutics, Inc. RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CN111727259B (zh) 2017-12-01 2024-04-19 编码治疗公司 工程化的dna结合蛋白
MX2020012077A (es) 2018-05-15 2021-03-09 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de la enfermedad de parkinson.
EP3824095A4 (en) * 2018-07-20 2022-04-20 University of Utah Research Foundation GENE THERAPY FOR MACULATE GENERATION
CN119955796A (zh) 2018-08-09 2025-05-09 比奥维拉迪维治疗股份有限公司 核酸分子及其用于非病毒基因疗法的用途
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
EP3863662A1 (en) * 2018-10-12 2021-08-18 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
CN115279896A (zh) 2019-09-30 2022-11-01 比奥维拉迪维治疗股份有限公司 慢病毒载体配制品
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
KR20220154141A (ko) * 2020-03-16 2022-11-21 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 종양-유래 바이러스 dna의 선택적 검출을 위한 조성물 및 방법
MX2023000156A (es) 2020-06-24 2023-02-16 Bioverativ Therapeutics Inc Metodos para la eliminacion de factor viii libre de preparaciones de vectores lentivirales modificados para expresar dicha proteina.
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
CA3189673A1 (en) 2020-08-23 2022-03-03 Ajay MAGHODIA Modified baculovirus system for improved production of closed-ended dna (cedna)
EP4392443A1 (en) 2021-08-23 2024-07-03 Bioverativ Therapeutics Inc. Optimized factor viii genes
IL310963A (en) 2021-08-23 2024-04-01 Bioverativ Therapeutics Inc Production of closed DNA with inverted repeat sequences
AU2022334711A1 (en) 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Baculovirus expression system
CA3232988A1 (en) 2021-09-30 2023-04-06 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
AU2022373653A1 (en) 2021-10-18 2024-05-02 Regeneron Pharmaceuticals, Inc. Eukaryotic cells comprising adenovirus-associated virus polynucleotides
US20240392318A1 (en) * 2023-04-26 2024-11-28 Sanofi Host cell lines and methods for identifying and using such host cell lines

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
DE60039766D1 (de) 1999-08-09 2008-09-18 Targeted Genetics Corp Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
EP1224312A1 (en) * 1999-10-12 2002-07-24 The University of North Carolina at Chapel Hill Adeno-associated virus vectors encoding factor viii and methods of using the same
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
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
CA2915124C (en) 2001-11-13 2018-08-14 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
PT1625210E (pt) 2003-05-21 2011-03-15 Genzyme Corp Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And 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
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
CN102803478B (zh) 2009-06-16 2016-04-27 建新公司 用于纯化重组aav载体的改进方法
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
JP6224459B2 (ja) 2011-02-17 2017-11-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法
CA2888931C (en) 2012-10-26 2023-09-05 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
EP3044231B1 (en) * 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii
EP4600255A3 (en) 2014-05-02 2025-10-22 Genzyme Corporation Aav vectors for retinal and cns gene therapy
AU2016219398A1 (en) 2015-02-10 2017-09-28 Genzyme Corporation 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
BR112017021505A2 (pt) 2018-07-03
CO2017011344A2 (es) 2018-03-28
CL2017002537A1 (es) 2018-05-11
KR20250126875A (ko) 2025-08-25
AU2016245806A1 (en) 2017-11-30
HUE069779T2 (hu) 2025-04-28
PL3280799T3 (pl) 2025-04-14
HK1246339A1 (en) 2018-09-07
NZ737009A (en) 2024-01-26
SG10201909381WA (en) 2019-11-28
SG10201912912QA (en) 2020-02-27
IL254933B (en) 2021-06-30
IL294965A (en) 2022-09-01
ECSP17074016A (es) 2018-02-28
IL283291B (en) 2022-08-01
EP4512429A3 (en) 2025-05-21
KR20170133500A (ko) 2017-12-05
TWI707951B (zh) 2020-10-21
CA2982123A1 (en) 2016-10-13
WO2016164609A2 (en) 2016-10-13
AU2022203942B2 (en) 2023-12-14
US20190048362A1 (en) 2019-02-14
PE20171800A1 (es) 2017-12-28
EA201792236A1 (ru) 2018-03-30
JP2023116678A (ja) 2023-08-22
MX2017012935A (es) 2018-02-12
AU2024201619A1 (en) 2024-03-28
AU2022203942A1 (en) 2022-06-23
PH12017501836A1 (en) 2018-04-23
IL283291A (en) 2021-07-29
JP6878299B2 (ja) 2021-05-26
US10815497B2 (en) 2020-10-27
CN107864657A (zh) 2018-03-30
DK3280799T3 (da) 2025-01-27
EP3280799A2 (en) 2018-02-14
CR20170505A (es) 2018-01-26
GT201700214A (es) 2018-10-24
JP2018510648A (ja) 2018-04-19
TN2017000431A1 (en) 2019-04-12
TW201710498A (zh) 2017-03-16
EP3280799B1 (en) 2024-10-30
ES3008382T3 (en) 2025-03-24
UY36611A (es) 2016-11-30
NZ774736A (en) 2024-08-30
SG11201708203UA (en) 2017-11-29
JP2021118724A (ja) 2021-08-12
IL254933A0 (en) 2017-12-31
WO2016164609A3 (en) 2016-12-15
EP4512429A2 (en) 2025-02-26

Similar Documents

Publication Publication Date Title
KR102848324B1 (ko) 거대 아데노-연관 벡터의 생산
US20240197837A1 (en) Gene editing of deep intronic mutations
US12201698B2 (en) Gene therapy for retinitis pigmentosa
JP2021118740A (ja) バリアントＲＮＡｉ
JP2020535803A (ja) バリアントＲＮＡｉ
JP2024527313A (ja) タウ低減遺伝子療法のための方法および組成物
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

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

PG1601 Publication of registration

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

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