KR20220128632A - 개선된 aav-abcd1 구축물 및 부신백질이영양증 (ald) 및/또는 부신척수신경병증 (amn)의 치료 또는 예방을 위한 용도 - Google Patents

개선된 aav-abcd1 구축물 및 부신백질이영양증 (ald) 및/또는 부신척수신경병증 (amn)의 치료 또는 예방을 위한 용도 Download PDF

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KR20220128632A
KR20220128632A KR1020227026264A KR20227026264A KR20220128632A KR 20220128632 A KR20220128632 A KR 20220128632A KR 1020227026264 A KR1020227026264 A KR 1020227026264A KR 20227026264 A KR20227026264 A KR 20227026264A KR 20220128632 A KR20220128632 A KR 20220128632A
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acid sequence
seq
optionally
amino acid
aav
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카렌 코자르스키
투그바 구벤-오즈칸
안나 트레샤코바
션 클라크
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스완바이오 테라퓨틱스 리미티드
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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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
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  • Biomedical Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Dermatology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020227026264A 2019-12-31 2020-12-31 개선된 aav-abcd1 구축물 및 부신백질이영양증 (ald) 및/또는 부신척수신경병증 (amn)의 치료 또는 예방을 위한 용도 Ceased KR20220128632A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962955667P 2019-12-31 2019-12-31
US62/955,667 2019-12-31
PCT/US2020/067664 WO2021138559A1 (en) 2019-12-31 2020-12-31 Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)

Publications (1)

Publication Number Publication Date
KR20220128632A true KR20220128632A (ko) 2022-09-21

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KR1020227026264A Ceased KR20220128632A (ko) 2019-12-31 2020-12-31 개선된 aav-abcd1 구축물 및 부신백질이영양증 (ald) 및/또는 부신척수신경병증 (amn)의 치료 또는 예방을 위한 용도

Country Status (11)

Country Link
US (2) US11779655B2 (https=)
EP (1) EP4085143B1 (https=)
JP (1) JP2023509443A (https=)
KR (1) KR20220128632A (https=)
CN (1) CN116096904A (https=)
AU (1) AU2020416291A1 (https=)
BR (1) BR112022013027A2 (https=)
CA (1) CA3166374A1 (https=)
IL (1) IL294278A (https=)
MX (1) MX2022008201A (https=)
WO (1) WO2021138559A1 (https=)

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Publication number Priority date Publication date Assignee Title
AR126727A1 (es) * 2021-08-06 2023-11-08 Wuhan Neurophth Biotechnology Ltd Company Composición y método para tratar la neuropatía óptica hereditaria de leber causada por la mutación nd4
US20240229067A9 (en) * 2022-05-12 2024-07-11 AAVnerGene Inc. Compositions and methods for recombinant parvovirus production
EP4522729A1 (en) * 2022-05-12 2025-03-19 Aavnergene Inc. Compositions and methods for recombinant parvovirus production
EP4536862A1 (en) * 2022-06-09 2025-04-16 Wisconsin Alumni Research Foundation Generation of next generation recombinant aav gene therapy vectors that adopt 3d conformation
CN121693566A (zh) * 2023-07-28 2026-03-17 瑞泽恩制药公司 使用bGH-SV40L串联PolyA在单向基因插入期间增强转基因表达

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MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EA201100225A1 (ru) * 2008-07-23 2011-08-30 Бёрингер Ингельхайм Фарма Гмбх Унд Ко. Кг Новые регуляторные элементы
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
KR20150029756A (ko) * 2011-06-10 2015-03-18 블루버드 바이오, 인코포레이티드. 부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터
US9163259B2 (en) * 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
US9938541B2 (en) * 2012-12-25 2018-04-10 Takara Bio Inc. AAV variant
WO2015153889A2 (en) * 2014-04-02 2015-10-08 University Of Florida Research Foundation, Incorporated Materials and methods for the treatment of latent viral infection
WO2016154055A1 (en) * 2015-03-20 2016-09-29 Bluebird Bio, Inc. Vector formulations
EP3285788B1 (en) * 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
EP4066862A1 (en) 2015-11-05 2022-10-05 The General Hospital Corporation Intrathecal delivery of nucleic acid sequences encoding abcd1 for treatment of adrenomyeloneuropathy
EP3411484B1 (en) * 2016-02-05 2023-10-04 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
WO2017137585A1 (en) * 2016-02-12 2017-08-17 Combigene Ab Vector
CN107287239B (zh) * 2016-04-11 2020-09-22 厦门继景生物技术有限责任公司 一种用于视网膜色素变性的基因治疗载体及药物
JP7165410B2 (ja) * 2016-08-19 2022-11-04 ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物
CA3050690A1 (en) * 2017-01-17 2018-07-26 Dana-Farber Cancer Institute, Inc. Compositions and methods for diagnosing and treating peroxisomal diseases
US12012602B2 (en) * 2017-03-10 2024-06-18 The Medical College Of Wisconsin, Inc. Riboswitch modulated gene therapy for retinal diseases
US20200377887A1 (en) * 2017-09-22 2020-12-03 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
MX2020004092A (es) * 2017-10-18 2021-01-08 Composiciones de virus adenoasociados para restaurar la funcion del gen hbb y metodos de uso de estas.
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Publication number Publication date
IL294278A (en) 2022-08-01
CA3166374A1 (en) 2021-07-08
US20240009326A1 (en) 2024-01-11
EP4085143B1 (en) 2025-08-13
WO2021138559A1 (en) 2021-07-08
US20220362403A2 (en) 2022-11-17
BR112022013027A2 (pt) 2022-09-06
JP2023509443A (ja) 2023-03-08
MX2022008201A (es) 2022-10-18
US20220175965A1 (en) 2022-06-09
AU2020416291A1 (en) 2022-07-21
EP4085143A4 (en) 2024-02-28
EP4085143A1 (en) 2022-11-09
CN116096904A (zh) 2023-05-09
US11779655B2 (en) 2023-10-10

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