KR20230019402A - 프로그래뉼린 연관 신경변성 질환 또는 장애의 치료를 위한 아데노-연관 바이러스 (aav) 시스템 - Google Patents

프로그래뉼린 연관 신경변성 질환 또는 장애의 치료를 위한 아데노-연관 바이러스 (aav) 시스템 Download PDF

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KR20230019402A
KR20230019402A KR1020227017064A KR20227017064A KR20230019402A KR 20230019402 A KR20230019402 A KR 20230019402A KR 1020227017064 A KR1020227017064 A KR 1020227017064A KR 20227017064 A KR20227017064 A KR 20227017064A KR 20230019402 A KR20230019402 A KR 20230019402A
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aav
nucleic acid
seq
promoter
sequence
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Korean (ko)
Inventor
주안 리
타나즈 파리바르
사비트리 만다파디
스티븐 페녹
마크 셔먼
주디스 뉴마크
아드리안 티멀스
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어플라이드 제네틱스 테크놀로지스 코퍼레이션
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Publication of KR20230019402A publication Critical patent/KR20230019402A/ko
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    • 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
    • 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
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    • C07—ORGANIC CHEMISTRY
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    • 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
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
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    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16051—Methods of production or purification of viral material
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • C—CHEMISTRY; METALLURGY
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Endocrinology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020227017064A 2019-10-22 2020-10-22 프로그래뉼린 연관 신경변성 질환 또는 장애의 치료를 위한 아데노-연관 바이러스 (aav) 시스템 Ceased KR20230019402A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962924340P 2019-10-22 2019-10-22
US62/924,340 2019-10-22
PCT/US2020/056860 WO2021081201A1 (en) 2019-10-22 2020-10-22 Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders

Publications (1)

Publication Number Publication Date
KR20230019402A true KR20230019402A (ko) 2023-02-08

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Application Number Title Priority Date Filing Date
KR1020227017064A Ceased KR20230019402A (ko) 2019-10-22 2020-10-22 프로그래뉼린 연관 신경변성 질환 또는 장애의 치료를 위한 아데노-연관 바이러스 (aav) 시스템

Country Status (10)

Country Link
US (2) US20210147872A1 (https=)
EP (1) EP4048799A4 (https=)
JP (1) JP2022553307A (https=)
KR (1) KR20230019402A (https=)
CN (1) CN115715327A (https=)
AU (1) AU2020371662A1 (https=)
CA (1) CA3158516A1 (https=)
IL (1) IL292382A (https=)
MX (1) MX2022004812A (https=)
WO (1) WO2021081201A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021533126A (ja) * 2018-07-31 2021-12-02 コーネル・ユニバーシティーCornell University 臓器機能を制御するための遺伝子治療方法
KR20230128001A (ko) * 2020-12-01 2023-09-01 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도
US20240033332A1 (en) * 2020-12-17 2024-02-01 Kyoto University Agent for activating a neuron
WO2024035649A1 (en) * 2022-08-08 2024-02-15 Shape Therapeutics Inc. Compositions for progranulin expression and methods of use thereof
KR20250131772A (ko) * 2022-11-13 2025-09-03 알렉시온 파마 인터내셔널 오퍼레이션즈 리미티드 전두측두엽 치매에 대한 유전자 요법
WO2025251041A1 (en) * 2024-05-31 2025-12-04 Bluerock Therapeutics Lp METHODS AND COMPOSITIONS FOR TREATING PROGRANULIN DEFICIENCIES USING iPSC-DERIVED CELLS

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009089635A1 (en) * 2008-01-16 2009-07-23 Neurodyn, Inc. Treating neurodegenerative diseases with progranulin (pgrn)
JP5879256B2 (ja) * 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
WO2016125330A1 (ja) * 2015-02-02 2016-08-11 原　英彰 網膜再生促進薬
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
WO2018136680A1 (en) * 2017-01-19 2018-07-26 New York University Progranulin and progranulin derivatives in treating impaired fracture healing
EP3692075A4 (en) * 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
KR102709597B1 (ko) * 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
US20220111005A1 (en) * 2019-02-01 2022-04-14 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
BR112021015817A2 (pt) * 2019-02-22 2021-10-13 The Trustees Of The University Of Pennsylvania Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn

Also Published As

Publication number Publication date
WO2021081201A1 (en) 2021-04-29
MX2022004812A (es) 2023-02-23
AU2020371662A1 (en) 2022-05-12
CA3158516A1 (en) 2021-04-29
EP4048799A4 (en) 2023-11-15
CN115715327A (zh) 2023-02-24
WO2021081201A8 (en) 2022-04-14
US20240318199A1 (en) 2024-09-26
US20210147872A1 (en) 2021-05-20
JP2022553307A (ja) 2022-12-22
IL292382A (en) 2022-06-01
EP4048799A1 (en) 2022-08-31

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