KR20250126875A - 거대 아데노-연관 벡터의 생산 - Google Patents

거대 아데노-연관 벡터의 생산

Info

Publication number
KR20250126875A
KR20250126875A KR1020257027291A KR20257027291A KR20250126875A KR 20250126875 A KR20250126875 A KR 20250126875A KR 1020257027291 A KR1020257027291 A KR 1020257027291A KR 20257027291 A KR20257027291 A KR 20257027291A KR 20250126875 A KR20250126875 A KR 20250126875A
Authority
KR
South Korea
Prior art keywords
aav
raav
genome
vector
particles
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.)
Pending
Application number
KR1020257027291A
Other languages
English (en)
Korean (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=KR20250126875(A) "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
Publication of KR20250126875A publication Critical patent/KR20250126875A/ko
Pending legal-status Critical Current

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)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pulmonology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020257027291A 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산 Pending KR20250126875A (ko)

Applications Claiming Priority (6)

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
KR1020177032062A KR102848324B1 (ko) 2015-04-08 2016-04-07 거대 아데노-연관 벡터의 생산
PCT/US2016/026486 WO2016164609A2 (en) 2015-04-08 2016-04-07 Production of oversized adeno-associated vectors

Related Parent Applications (1)

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

Publications (1)

Publication Number Publication Date
KR20250126875A true KR20250126875A (ko) 2025-08-25

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 After (1)

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

Country Status (27)

Country Link
US (1) US10815497B2 (https=)
EP (2) EP3280799B1 (https=)
JP (3) JP6878299B2 (https=)
KR (2) KR20250126875A (https=)
CN (1) CN107864657A (https=)
AU (3) AU2016245806A1 (https=)
BR (1) BR112017021505A2 (https=)
CA (1) CA2982123A1 (https=)
CL (1) CL2017002537A1 (https=)
CO (1) CO2017011344A2 (https=)
CR (1) CR20170505A (https=)
DK (1) DK3280799T3 (https=)
EA (1) EA201792236A1 (https=)
EC (1) ECSP17074016A (https=)
ES (1) ES3008382T3 (https=)
GT (1) GT201700214A (https=)
HU (1) HUE069779T2 (https=)
IL (3) IL283291B (https=)
MX (1) MX2017012935A (https=)
PE (1) PE20171800A1 (https=)
PH (1) PH12017501836A1 (https=)
PL (1) PL3280799T3 (https=)
SG (3) SG10201912912QA (https=)
TN (1) TN2017000431A1 (https=)
TW (1) TWI707951B (https=)
UY (1) UY36611A (https=)
WO (1) WO2016164609A2 (https=)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI3889173T1 (sl) 2013-02-15 2023-11-30 Bioverativ Therapeutics Inc. Optimiran gen dejavnika VIII
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
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
TWI707951B (zh) * 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
RU2742352C2 (ru) 2015-09-24 2021-02-05 Байомарин Фармасьютикал Инк. Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции
WO2017136358A1 (en) 2016-02-01 2017-08-10 Bioverativ Therapeutics Inc. Optimized factor viii genes
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
CA3024449A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
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
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． ハンチントン病治療組成物および方法
CA3063464A1 (en) * 2017-05-19 2018-11-22 Encoded Therapeutics, Inc. High activity regulatory elements
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
TW202545984A (zh) 2017-08-09 2025-12-01 美商生物化學醫療公司 核酸分子及其用途
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
IL274926B2 (en) 2017-12-01 2026-04-01 Encoded Therapeutics Inc Pyrazolopyrimidines with activity against respiratory syncytial virus
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
EP3824095A4 (en) * 2018-07-20 2022-04-20 University of Utah Research Foundation GENE THERAPY FOR MACULATE GENERATION
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
US12281321B2 (en) 2018-09-28 2025-04-22 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
CN114302968A (zh) * 2019-08-29 2022-04-08 美国西门子医学诊断股份有限公司 用于检测aav脱落的试剂和方法
JP7803014B2 (ja) 2019-09-30 2026-01-21 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 レンチウイルスベクター製剤
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
US11254989B2 (en) * 2020-03-16 2022-02-22 The University Of North Carolina At Chapel Hill Compositions and methods for the selective detection of tumor-derived viral DNA
US12173307B2 (en) 2020-06-24 2024-12-24 Bioverativ Therapeutics Inc. Methods for the purification of viral vectors
EP4189077A1 (en) 2020-07-30 2023-06-07 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
US20220090130A1 (en) 2020-08-23 2022-03-24 Bioverativ Therapeutics Inc. MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
CA3229323A1 (en) 2021-08-23 2023-03-02 Ajay MAGHODIA Optimized factor viii genes
CA3229345A1 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal 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
CN121586778A (zh) * 2023-04-26 2026-02-27 赛诺菲 宿主细胞系以及用于鉴定和使用此类宿主细胞系的方法

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6995006B2 (en) 1997-09-05 2006-02-07 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
PT1916258E (pt) 1999-08-09 2014-07-29 Genzyme Corp Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
AU7879000A (en) 1999-10-12 2001-04-23 University Of North Carolina At Chapel Hill, The 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
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
US20040092008A1 (en) * 2002-11-12 2004-05-13 Snyder Richard O. Recombinant AAV vectors for gene therapy of hemophilia A
WO2004075861A2 (en) 2003-02-26 2004-09-10 Children's Hospital, Inc. Recombinant adeno-associated virus production
DE602004030327D1 (de) 2003-05-21 2011-01-13 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
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
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
HUE028341T2 (en) 2009-06-16 2016-12-28 Genzyme Corp Improved methods for purification of recombinant aav vectors
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
DK2675902T3 (da) 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
EP2911687B1 (en) * 2012-10-26 2019-02-13 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
RS61039B1 (sr) * 2013-09-12 2020-12-31 Biomarin Pharm Inc Aav vektori koji sadrže gen koji kodira faktor viii
IL248102B (en) 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
TN2017000353A1 (en) 2015-02-10 2019-01-16 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

Also Published As

Publication number Publication date
BR112017021505A2 (pt) 2018-07-03
TW201710498A (zh) 2017-03-16
WO2016164609A3 (en) 2016-12-15
ES3008382T3 (en) 2025-03-24
MX2017012935A (es) 2018-02-12
PH12017501836A1 (en) 2018-04-23
JP2023116678A (ja) 2023-08-22
KR20170133500A (ko) 2017-12-05
EP4512429A3 (en) 2025-05-21
IL283291A (en) 2021-07-29
AU2022203942B2 (en) 2023-12-14
IL254933B (en) 2021-06-30
NZ737009A (en) 2024-01-26
SG10201909381WA (en) 2019-11-28
CL2017002537A1 (es) 2018-05-11
SG11201708203UA (en) 2017-11-29
IL254933A0 (en) 2017-12-31
EP3280799B1 (en) 2024-10-30
CA2982123A1 (en) 2016-10-13
AU2022203942A1 (en) 2022-06-23
HUE069779T2 (hu) 2025-04-28
TWI707951B (zh) 2020-10-21
US10815497B2 (en) 2020-10-27
UY36611A (es) 2016-11-30
GT201700214A (es) 2018-10-24
AU2016245806A1 (en) 2017-11-30
IL283291B (en) 2022-08-01
EP3280799A2 (en) 2018-02-14
JP6878299B2 (ja) 2021-05-26
SG10201912912QA (en) 2020-02-27
HK1246339A1 (en) 2018-09-07
IL294965A (en) 2022-09-01
JP2021118724A (ja) 2021-08-12
WO2016164609A2 (en) 2016-10-13
TN2017000431A1 (en) 2019-04-12
AU2024201619A1 (en) 2024-03-28
CO2017011344A2 (es) 2018-03-28
PE20171800A1 (es) 2017-12-28
CR20170505A (es) 2018-01-26
JP2018510648A (ja) 2018-04-19
CN107864657A (zh) 2018-03-30
US20190048362A1 (en) 2019-02-14
EP4512429A2 (en) 2025-02-26
PL3280799T3 (pl) 2025-04-14
NZ774736A (en) 2024-08-30
KR102848324B1 (ko) 2025-08-20
DK3280799T3 (da) 2025-01-27
ECSP17074016A (es) 2018-02-28
EA201792236A1 (ru) 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
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

PG1501 Laying open of application

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

Q12 Application published

Free format text: ST27 STATUS EVENT CODE: A-1-1-Q10-Q12-NAP-PG1501 (AS PROVIDED BY THE NATIONAL OFFICE)