KR20210103469A - 재조합 바이러스 벡터 및 이를 생산하기 위한 핵산 - Google Patents

재조합 바이러스 벡터 및 이를 생산하기 위한 핵산 Download PDF

Info

Publication number
KR20210103469A
KR20210103469A KR1020217017068A KR20217017068A KR20210103469A KR 20210103469 A KR20210103469 A KR 20210103469A KR 1020217017068 A KR1020217017068 A KR 1020217017068A KR 20217017068 A KR20217017068 A KR 20217017068A KR 20210103469 A KR20210103469 A KR 20210103469A
Authority
KR
South Korea
Prior art keywords
sequence
nucleic acid
promoter
aav
itr
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
KR1020217017068A
Other languages
English (en)
Korean (ko)
Inventor
다비 토마스
데이비드 디스뮤크
Original Assignee
스트라이드바이오 인코포레이티드
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 스트라이드바이오 인코포레이티드 filed Critical 스트라이드바이오 인코포레이티드
Publication of KR20210103469A publication Critical patent/KR20210103469A/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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020217017068A 2018-11-21 2019-11-21 재조합 바이러스 벡터 및 이를 생산하기 위한 핵산 Ceased KR20210103469A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862770202P 2018-11-21 2018-11-21
US62/770,202 2018-11-21
PCT/US2019/062531 WO2020106916A1 (en) 2018-11-21 2019-11-21 Recombinant viral vectors and nucleic acids for producing the same

Publications (1)

Publication Number Publication Date
KR20210103469A true KR20210103469A (ko) 2021-08-23

Family

ID=70773595

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217017068A Ceased KR20210103469A (ko) 2018-11-21 2019-11-21 재조합 바이러스 벡터 및 이를 생산하기 위한 핵산

Country Status (20)

Country Link
US (1) US20210324418A1 (enExample)
EP (1) EP3883954A4 (enExample)
JP (2) JP7704998B2 (enExample)
KR (1) KR20210103469A (enExample)
CN (1) CN113302201A (enExample)
AR (1) AR117145A1 (enExample)
AU (1) AU2019385506A1 (enExample)
BR (1) BR112021009733A2 (enExample)
CA (1) CA3120289A1 (enExample)
CL (1) CL2021001327A1 (enExample)
CO (1) CO2021008120A2 (enExample)
EA (1) EA202191418A1 (enExample)
EC (1) ECSP21044840A (enExample)
IL (1) IL283344A (enExample)
MX (1) MX2021005997A (enExample)
PE (1) PE20211419A1 (enExample)
PH (1) PH12021551155A1 (enExample)
SG (1) SG11202105326WA (enExample)
TW (1) TW202039533A (enExample)
WO (1) WO2020106916A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230156799A (ko) * 2020-07-08 2023-11-14 주식회사 뉴라클제네틱스 신규한 인트론 단편
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템

Families Citing this family (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107686842A (zh) * 2016-08-03 2018-02-13 南京大学 一种靶多核苷酸编辑方法及其应用
US12509492B2 (en) 2018-01-19 2025-12-30 Duke University Genome engineering with CRISPR-Cas systems in eukaryotes
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
US20230285596A1 (en) * 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
MX2023002016A (es) * 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
MX2023003656A (es) * 2020-09-29 2023-06-22 Neuexcell Therapeutics Inc Vector dlx2.
EP4222270A4 (en) * 2020-09-29 2025-02-26 Neuexcell Therapeutics Inc. NEUROD1-VECTOR
US20220098617A1 (en) * 2020-09-29 2022-03-31 NeuExcell Therapeutics Inc. Ascl1 vector
MX2023003654A (es) * 2020-09-29 2023-06-22 Neuexcell Therapeutics Inc Vector de combinación de neurod1.
EP4232152A4 (en) * 2020-10-21 2025-04-23 Duke University Dual AAV vector-mediated deletion of a large mutation hotspot for the treatment of Duchenne muscular dystrophy
CN116887866A (zh) 2020-12-03 2023-10-13 巴特尔纪念研究院 聚合物纳米颗粒和dna纳米结构组合物及用于非病毒递送的方法
WO2022147575A1 (en) * 2021-01-04 2022-07-07 University Of Florida Research Foundation, Incorporated Methods and compositions for treatment of friedreich's ataxia
JP2024516108A (ja) 2021-04-07 2024-04-12 バテル・メモリアル・インスティテュート 非ウイルス性担体を同定および使用するための迅速な設計、構築、試験、および学習技術
US20240376497A1 (en) * 2021-09-17 2024-11-14 Astellas Gene Therapies, Inc. Frataxin gene therapy
CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
US20250361525A1 (en) * 2022-06-08 2025-11-27 Scribe Therapeutics Inc. Aav vectors for gene editing
CN118715025A (zh) * 2022-10-08 2024-09-27 凌意(杭州)生物科技有限公司 用于增强基因表达的构建体
WO2024163678A2 (en) * 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2025072751A1 (en) 2023-09-29 2025-04-03 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
US12441996B2 (en) 2023-12-08 2025-10-14 Battelle Memorial Institute Use of DNA origami nanostructures for molecular information based data storage systems
CN117701638B (zh) * 2023-12-18 2024-10-18 苏州新芽基因生物技术有限公司 一种抑制残留aav载体质粒和质粒骨架转录活性的方法

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002081632A2 (en) * 2001-04-04 2002-10-17 The Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services Nucleic acids for transgene expression
US20090221620A1 (en) * 2008-02-20 2009-09-03 Celera Corporation Gentic polymorphisms associated with stroke, methods of detection and uses thereof
EP2986635B1 (en) * 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
WO2015044704A1 (en) * 2013-09-30 2015-04-02 Sanofi Use of neuroglobin agonist for preventing or treating mitochondrial rcci and/or rcciii deficiency disease
US10947596B2 (en) 2014-05-06 2021-03-16 Dana-Farber Cancer Institute, Inc. Compositions and methods for identification, assessment, prevention, and treatment of cancer using NFS1 biomarkers and modulators
WO2016094783A1 (en) * 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
US20180021364A1 (en) 2015-01-16 2018-01-25 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
US10450563B2 (en) * 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
WO2016150964A1 (en) * 2015-03-23 2016-09-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with friedreich ataxia
WO2016164642A1 (en) * 2015-04-08 2016-10-13 The United States Of America, As Represented By The Secretary Of Health And Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
RU2021132397A (ru) * 2015-05-16 2022-02-24 Джензим Корпорейшн Генное редактирование глубоких интронных мутаций
PT3313991T (pt) * 2015-06-23 2024-09-27 Childrens Hospital Philadelphia Fator ix modificado e composições, métodos e utilizações para transferência de genes para células, órgãos e tecidos
US10266844B2 (en) * 2015-07-31 2019-04-23 California Institute Of Technology Activity-dependent expression of nucleic acids
PE20181156A1 (es) * 2015-11-05 2018-07-19 Bamboo Therapeutics Inc Genes de ataxia de friedreich modificados y vectores para terapia genica
RU2742612C2 (ru) * 2015-12-15 2021-02-09 Джензим Корпорейшн Векторы на основе аденоассоциированного вируса для лечения муколипидоза типа ii
US11564997B2 (en) * 2016-06-29 2023-01-31 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
US20200056206A1 (en) * 2016-12-01 2020-02-20 UNIVERSITé LAVAL Crispr-based treatment of friedreich ataxia
US20210254056A1 (en) * 2017-05-05 2021-08-19 Camp4 Therapeutics Corporation Identification and targeted modulation of gene signaling networks
MX2020003042A (es) * 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230156799A (ko) * 2020-07-08 2023-11-14 주식회사 뉴라클제네틱스 신규한 인트론 단편
WO2026019230A1 (ko) * 2024-07-15 2026-01-22 삼성바이오에피스 주식회사 스터퍼를 포함하는 재조합 aav 생산을 위한 벡터 시스템

Also Published As

Publication number Publication date
BR112021009733A2 (pt) 2022-01-04
EA202191418A1 (ru) 2021-08-05
JP2025102783A (ja) 2025-07-08
CN113302201A (zh) 2021-08-24
ECSP21044840A (es) 2021-09-30
SG11202105326WA (en) 2021-06-29
TW202039533A (zh) 2020-11-01
PH12021551155A1 (en) 2021-11-03
JP2022508182A (ja) 2022-01-19
AR117145A1 (es) 2021-07-14
US20210324418A1 (en) 2021-10-21
CL2021001327A1 (es) 2021-12-31
WO2020106916A1 (en) 2020-05-28
EP3883954A4 (en) 2022-08-10
PE20211419A1 (es) 2021-08-03
IL283344A (en) 2021-07-29
AU2019385506A1 (en) 2021-06-03
CA3120289A1 (en) 2020-05-28
CO2021008120A2 (es) 2021-08-09
JP7704998B2 (ja) 2025-07-09
MX2021005997A (es) 2021-08-11
EP3883954A1 (en) 2021-09-29

Similar Documents

Publication Publication Date Title
CN113302201A (zh) 重组病毒载体和用于产生所述重组病毒载体的核酸
KR102918544B1 (ko) 다논병을 치료하기 위한 유전자 요법 벡터
US11951183B2 (en) Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
CN112368390B (zh) Cns变性的基因疗法
JP7433360B2 (ja) Ｐａｈ遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法
KR20230043869A (ko) Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
US5756283A (en) Method for improved production of recombinant adeno-associated viruses for gene therapy
CN116745409A (zh) 用于治疗雷特综合征的腺相关病毒载体
KR20210131370A (ko) Grn-연관 성인-발병 신경퇴화의 치료를 위한 재조합 아데노-연관 바이러스
AU2018262427B2 (en) Gene therapy for ciliopathies
CN117545842A (zh) SMN1和miR-23a在治疗脊髓性肌萎缩中的协同效应
US6806080B2 (en) Hybrid vectors for gene therapy
KR20250102136A (ko) 중추 신경계에서의 유전자 발현을 위한 핵산 조절 요소 및 사용 방법
KR20240025645A (ko) 아데노-연관된 바이러스 패키징 시스템
CN115029360B (zh) 用于治疗粘多糖贮积症iiia型的转基因表达盒
KR20220164743A (ko) Ids 유전자 전달을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법
RU2839898C2 (ru) Синергетическое действие SMN1 и miR-23a при лечении спинальной мышечной атрофии
RU2858237C2 (ru) Полинуклеотид и вектор генной терапии для экспрессии плакофилина-2 (pkp2) в клетке, способ, фармацевтическая композиция, применение вектора и набор для лечения или предупреждения связанного с ним заболевания
RU2808459C2 (ru) Геннотерапевтические векторы для лечения болезни данона
WO2024094009A1 (zh) 用于目的基因的表达盒及其应用
HK40104471A (zh) 用於目的基因的表达盒及其应用
EA047326B1 (ru) СИНЕРГЕТИЧЕСКОЕ ДЕЙСТВИЕ SMN1 И miR-23a ПРИ ЛЕЧЕНИИ СПИНАЛЬНОЙ МЫШЕЧНОЙ АТРОФИИ
TW202521698A (zh) 用於目標基因的表現匣及其應用
HK40106789A (zh) Smn1和mir-23a在治疗脊髓性肌萎缩中的协同效应
EA049295B1 (ru) Рекомбинантные вирусные векторы и нуклеиновые кислоты для их получения

Legal Events

Date Code Title Description
PA0105 International application

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

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

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

A201 Request for examination
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

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

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

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

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

B15 Application refused following examination

Free format text: ST27 STATUS EVENT CODE: N-2-6-B10-B15-EXM-PE0601 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0601 Decision on rejection of patent

St.27 status event code: N-2-6-B10-B15-exm-PE0601