KR20140023310A - 벡터 제조 및 유전자 전달을 위한 캡시드-결핍 ａａｖ 벡터, 조성물 및 방법 - Google Patents

벡터 제조 및 유전자 전달을 위한 캡시드-결핍 ａａｖ 벡터, 조성물 및 방법 Download PDF

Info

Publication number
KR20140023310A
KR20140023310A KR1020137026982A KR20137026982A KR20140023310A KR 20140023310 A KR20140023310 A KR 20140023310A KR 1020137026982 A KR1020137026982 A KR 1020137026982A KR 20137026982 A KR20137026982 A KR 20137026982A KR 20140023310 A KR20140023310 A KR 20140023310A
Authority
KR
South Korea
Prior art keywords
vector
cell
nucleic acid
aav
expression
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.)
Ceased
Application number
KR1020137026982A
Other languages
English (en)
Korean (ko)
Inventor
뤼이스 갸르시아
씨리아끄 벨레
토마스 부와
로버트 마이클 코틴
Original Assignee
아쏘씨아씨옹 엥스띠뛰 드 미올로지
유니베르시테 피에르 에 마리에 쿠리에 (파리 6)
상뜨로 나쇼날 드 라 러쉐르쉐 샹띠피크
인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔)
유에스 디파트먼트 오브 헬쓰 앤드 휴먼 서비시스, 내셔널 인스티튜트스 오브 헬쓰
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 아쏘씨아씨옹 엥스띠뛰 드 미올로지, 유니베르시테 피에르 에 마리에 쿠리에 (파리 6), 상뜨로 나쇼날 드 라 러쉐르쉐 샹띠피크, 인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔), 유에스 디파트먼트 오브 헬쓰 앤드 휴먼 서비시스, 내셔널 인스티튜트스 오브 헬쓰 filed Critical 아쏘씨아씨옹 엥스띠뛰 드 미올로지
Priority to KR1020207027994A priority Critical patent/KR102373765B1/ko
Publication of KR20140023310A publication Critical patent/KR20140023310A/ko
Ceased legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/24—Vectors characterised by the absence of particular element, e.g. selectable marker, viral origin of replication

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Psychology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Vascular Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Cardiology (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
KR1020137026982A 2011-03-11 2012-03-12 벡터 제조 및 유전자 전달을 위한 캡시드-결핍 ａａｖ 벡터, 조성물 및 방법 Ceased KR20140023310A (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020207027994A KR102373765B1 (ko) 2011-03-11 2012-03-12 벡터 제조 및 유전자 전달을 위한 캡시드-결핍 ａａｖ 벡터, 조성물 및 방법

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161452071P 2011-03-11 2011-03-11
US61/452,071 2011-03-11
EP11157986.8 2011-03-12
EP11157986A EP2500434A1 (en) 2011-03-12 2011-03-12 Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
PCT/EP2012/054303 WO2012123430A1 (en) 2011-03-11 2012-03-12 Capsid-free aav vectors, compositions, and methods for vector production and gene delivery

Related Child Applications (1)

Application Number Title Priority Date Filing Date
KR1020207027994A Division KR102373765B1 (ko) 2011-03-11 2012-03-12 벡터 제조 및 유전자 전달을 위한 캡시드-결핍 ａａｖ 벡터, 조성물 및 방법

Publications (1)

Publication Number Publication Date
KR20140023310A true KR20140023310A (ko) 2014-02-26

Family

ID=44316426

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020137026982A Ceased KR20140023310A (ko) 2011-03-11 2012-03-12 벡터 제조 및 유전자 전달을 위한 캡시드-결핍 ａａｖ 벡터, 조성물 및 방법
KR1020207027994A Active KR102373765B1 (ko) 2011-03-11 2012-03-12 벡터 제조 및 유전자 전달을 위한 캡시드-결핍 ａａｖ 벡터, 조성물 및 방법

Family Applications After (1)

Application Number Title Priority Date Filing Date
KR1020207027994A Active KR102373765B1 (ko) 2011-03-11 2012-03-12 벡터 제조 및 유전자 전달을 위한 캡시드-결핍 ａａｖ 벡터, 조성물 및 방법

Country Status (10)

Country Link
US (1) US9598703B2 (https=)
EP (3) EP2500434A1 (https=)
JP (6) JP2014513928A (https=)
KR (2) KR20140023310A (https=)
CN (1) CN103764831B (https=)
AU (1) AU2012228376B2 (https=)
BR (1) BR112013023185B8 (https=)
CA (1) CA2829518A1 (https=)
IL (1) IL228328B (https=)
WO (1) WO2012123430A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200051708A (ko) * 2017-09-08 2020-05-13 제너레이션 바이오 컴퍼니 비-바이러스성 캡시드-비함유 dna 벡터의 지질 나노입자 제형

Families Citing this family (102)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
EP3318635A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
CN105765066B (zh) 2013-11-29 2018-12-14 宝生物工程株式会社 用于定量腺伴随病毒的方法
ES2833230T3 (es) 2014-05-13 2021-06-14 Univ Pennsylvania Composiciones que comprenden AVV que expresan construcciones y usos de doble anticuerpo del mismo
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
AU2015320694B2 (en) * 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
MX2017009336A (es) 2015-01-16 2017-11-15 Voyager Therapeutics Inc Polinucleótidos dirigidos al sistema nervioso central.
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US10857240B2 (en) 2016-01-05 2020-12-08 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of ocular disorders and blinding diseases
CN108884446A (zh) * 2016-01-29 2018-11-23 西里昂生物技术有限责任公司 基于aav的条件表达系统
WO2017136435A1 (en) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Compounds for modulating fc-epsilon-ri-beta expression and uses thereof
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
JP6966463B2 (ja) * 2016-02-26 2021-11-17 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 組換えウイルス産物及びｄｕｘ４エクソンスキッピングを誘導するための方法
MX2018010633A (es) 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
DK3452583T3 (da) 2016-05-03 2022-01-10 Prec Biosciences Inc Konstruerede nukleaser, der er nyttige til behandling af hæmofili a
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
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
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
WO2018089527A1 (en) 2016-11-09 2018-05-17 Intrexon Corporation Frataxin expression constructs
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
JP2020518258A (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． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
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
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
US11549125B2 (en) 2017-08-09 2023-01-10 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Closed-ended, linear, duplex adenoassociated virus DNA, and uses thereof
CA3075168A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. Modified closed-ended dna (cedna)
RU2770922C2 (ru) * 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
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
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
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
WO2019089913A1 (en) * 2017-11-01 2019-05-09 Precision Biosciences, Inc. Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a
CA3084185A1 (en) * 2017-12-06 2019-06-13 Generation Bio Co. Gene editing using a modified closed-ended dna (cedna)
US12442015B2 (en) 2018-01-19 2025-10-14 Generation Bio Co. Closed-ended DNA vectors obtainable from cell-free synthesis and process for obtaining ceDNA vectors
KR20200120649A (ko) * 2018-02-14 2020-10-21 제너레이션 바이오 컴퍼니 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도
MA51915A (fr) * 2018-02-22 2020-12-30 Generation Bio Co Expression régulée de transgènes à l'aide de vecteurs adn à extrémités fermées (cedna)
US20210002667A1 (en) * 2018-03-15 2021-01-07 Intergalactic Therapeutics, Inc. Synthetic dna vectors and methods of use
GB201806333D0 (en) * 2018-04-18 2018-05-30 Glaxosmithkline Ip Dev Ltd Parvovirus vector production
AU2019265663B2 (en) 2018-05-08 2024-06-13 Rutgers, The State University Of New Jersey AAV-compatible laminin-linker polymerization proteins
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
EP3810782A2 (en) * 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
GB201810301D0 (en) * 2018-06-22 2018-08-08 Evox Therapeutics Ltd Combinatorial gene therapy
CN112770812A (zh) 2018-07-24 2021-05-07 沃雅戈治疗公司 产生基因治疗制剂的系统和方法
EP3833766A1 (en) * 2018-08-09 2021-06-16 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
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
WO2020072844A1 (en) 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
CN113166781A (zh) 2018-10-15 2021-07-23 沃雅戈治疗公司 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体
KR20210090619A (ko) * 2018-11-09 2021-07-20 제너레이션 바이오 컴퍼니 대칭인 변형된 역말단반복을 포함하는 변형된 폐쇄형 DNA(ceDNA)
JP2022518504A (ja) * 2019-01-24 2022-03-15 ジェネレーション バイオ カンパニー 閉端ｄｎａ（ｃｅｄｎａ）ならびに遺伝子療法または核酸療法に関連する免疫応答を低減させる方法における使用
CN113454232B (zh) * 2019-02-15 2024-10-29 世代生物公司 封闭端dna（cedna）产生中对rep蛋白活性的调节
MA55223A (fr) * 2019-03-06 2022-01-12 Generation Bio Co Adn à extrémité fermée (cedna) et composés de modulation immunitaire
SG11202109850SA (en) 2019-03-13 2021-10-28 Generation Bio Co Non-viral dna vectors and uses thereof for expressing fviii therapeutics
EP3938515A4 (en) * 2019-03-13 2022-12-28 Generation Bio Co. NON-VIRAL DNA VECTORS AND THEIR USES FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) THERAPEUTIC AGENTS
FI3953483T3 (fi) 2019-04-11 2023-11-30 Regenxbio Inc Kokoekskluusiokromatografiamenetelmiä rekombinanttien adenoassosioituneiden viruskoostumusten karakterisoimiseksi
EP3987043A4 (en) * 2019-06-21 2023-07-19 Asklepios Biopharmaceutical, Inc. PRODUCTION OF VECTORS USING THE PHAGEN REPLICATION ORIGIN
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US10801042B1 (en) 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
CA3147414A1 (en) 2019-07-17 2021-01-21 Generation Bio Co. Synthetic production of single-stranded adeno associated viral dna vectors
WO2021042004A1 (en) * 2019-08-29 2021-03-04 Siemens Healthcare Diagnostics Inc. Reagents and methods for detecting aav shedding
WO2021046265A1 (en) * 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
KR20220128607A (ko) 2019-09-18 2022-09-21 인터갈락틱 테라퓨틱스, 인크. 합성 dna 벡터 및 사용 방법
JP6924289B2 (ja) 2020-01-21 2021-08-25 シャープ株式会社 端末装置、基地局装置、および、方法
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
JP7746280B2 (ja) 2020-03-27 2025-09-30 ジェネレーション バイオ カンパニー 新規脂質及びそれらのナノ粒子組成物
GB202009618D0 (en) 2020-06-24 2020-08-05 Ucl Business Ltd Therapeutics for the treatment of FSHD
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
CN112662706A (zh) * 2020-12-31 2021-04-16 深圳市第二人民医院(深圳市转化医学研究院) 一种外泌体包裹aav载体的制备及纯化方法
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
WO2023069948A1 (en) * 2021-10-18 2023-04-27 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods
CA3262218A1 (en) 2022-07-19 2024-01-25 Rampart Bioscience, Inc. CIRCULAR NON-IMMUNOGENEOUS NON-VIRAL DNA VECTORS
WO2024098035A2 (en) * 2022-11-04 2024-05-10 National Resilience, Inc. Methods and compositions for preparing recombinant adeno associated viruses and uses thereof
US11891617B1 (en) * 2023-03-27 2024-02-06 Genecraft Inc. Modified complex platform of adeno-associated virus with improved rate of expression of loaded genes and reduced genotoxicity
CN119320802A (zh) * 2023-07-17 2025-01-17 苏州吉恒基因科技有限公司 单链单极DNA重组腺相关病毒的cis包装元件、spAAV载体和应用

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5843742A (en) * 1994-12-16 1998-12-01 Avigen Incorporated Adeno-associated derived vector systems for gene delivery and integration into target cells
GB9426053D0 (en) * 1994-12-19 1995-02-22 Trigon Ind Ltd A carbon dioxide sensitive material
SI1496944T1 (sl) * 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
CN1570121A (zh) * 2003-07-25 2005-01-26 北京三诺佳邑生物技术有限责任公司 高效生产重组腺病毒伴随病毒的病毒载体、细胞系、方法及其组成的生产系统
JP2008503590A (ja) * 2004-06-21 2008-02-07 メドトロニック・インコーポレーテッド 組成物を細胞にデリバリーするための医学用システム及び方法
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
CN1286980C (zh) * 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类反义受磷蛋白基因的重组腺相关病毒及其制备方法
EP2311966A3 (en) * 2005-10-20 2012-09-05 Amsterdam Molecular Therapeutics (AMT) B.V. Improved AAV vectors produced in insect cells
CN103849629B (zh) * 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200051708A (ko) * 2017-09-08 2020-05-13 제너레이션 바이오 컴퍼니 비-바이러스성 캡시드-비함유 dna 벡터의 지질 나노입자 제형

Also Published As

Publication number Publication date
BR112013023185A2 (https=) 2017-01-03
CN103764831A (zh) 2014-04-30
EP4227415A3 (en) 2025-04-16
US9598703B2 (en) 2017-03-21
KR102373765B1 (ko) 2022-03-15
CN103764831B (zh) 2019-11-01
US20140107186A1 (en) 2014-04-17
CA2829518A1 (en) 2012-09-20
WO2012123430A1 (en) 2012-09-20
EP2683829A1 (en) 2014-01-15
EP4227415A2 (en) 2023-08-16
JP2022121364A (ja) 2022-08-19
AU2012228376B2 (en) 2017-06-22
JP2022008560A (ja) 2022-01-13
IL228328B (en) 2018-08-30
AU2012228376A1 (en) 2013-09-26
JP2024123132A (ja) 2024-09-10
JP2020007312A (ja) 2020-01-16
BR112013023185B8 (pt) 2022-03-22
JP2014513928A (ja) 2014-06-19
EP2500434A1 (en) 2012-09-19
JP2017192385A (ja) 2017-10-26
BR112013023185B1 (pt) 2022-02-01
KR20200116550A (ko) 2020-10-12

Similar Documents

Publication Publication Date Title
KR102373765B1 (ko) 벡터 제조 및 유전자 전달을 위한 캡시드-결핍 ａａｖ 벡터, 조성물 및 방법
US20250121092A1 (en) Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
JP7303816B2 (ja) Ａａｖベクター
CN103608455A (zh) 用于治疗色盲和其它疾病的启动子、表达盒、载体、药盒和方法
CN108697774A (zh) 治疗mps i-相关失明的aav-idua载体
AU2023206111B2 (en) Gene therapy with dysferlin dual vectors
CN115715185A (zh) 用于治疗神经系统疾病的组合物和方法
US20240376493A1 (en) Optimized ap4m1 polyneucleotides and expression cassettes and their use
US20150191527A1 (en) Methods of treating alzheimer's disease with apo a-1 milano
CN116670159A (zh) 组合物及其用于治疗安格尔曼综合征的用途
US20160237141A1 (en) Methods of treating alzheimer's disease with apo a-1 milano
EA047041B1 (ru) Генная терапия с использованием двойных векторов дисферлина
NZ753155B2 (en) Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20131011

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20170228

Comment text: Request for Examination of Application

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

Comment text: Notification of reason for refusal

Patent event date: 20180627

Patent event code: PE09021S01D

AMND Amendment
E90F Notification of reason for final refusal
PE0902 Notice of grounds for rejection

Comment text: Final Notice of Reason for Refusal

Patent event date: 20190426

Patent event code: PE09021S02D

AMND Amendment
E601 Decision to refuse application
PE0601 Decision on rejection of patent

Patent event date: 20200224

Comment text: Decision to Refuse Application

Patent event code: PE06012S01D

Patent event date: 20190426

Comment text: Final Notice of Reason for Refusal

Patent event code: PE06011S02I

Patent event date: 20180627

Comment text: Notification of reason for refusal

Patent event code: PE06011S01I

X091 Application refused [patent]
E601 Decision to refuse application
E801 Decision on dismissal of amendment
PE0601 Decision on rejection of patent

Patent event date: 20200626

Comment text: Decision to Refuse Application

Patent event code: PE06012S01D

Patent event date: 20190426

Comment text: Final Notice of Reason for Refusal

Patent event code: PE06011S02I

Patent event date: 20180627

Comment text: Notification of reason for refusal

Patent event code: PE06011S01I

PE0801 Dismissal of amendment

Patent event code: PE08012E01D

Comment text: Decision on Dismissal of Amendment

Patent event date: 20200626

Patent event code: PE08011R01I

Comment text: Amendment to Specification, etc.

Patent event date: 20200525

Patent event code: PE08011R01I

Comment text: Amendment to Specification, etc.

Patent event date: 20191028

Patent event code: PE08011R01I

Comment text: Amendment to Specification, etc.

Patent event date: 20181227

A107 Divisional application of patent
PA0104 Divisional application for international application

Comment text: Divisional Application for International Patent

Patent event code: PA01041R01D

Patent event date: 20200928