RU2744593C2 - Векторы на основе аденоассоциированных вирусов для лечения мукополисахаридозов - Google Patents

Векторы на основе аденоассоциированных вирусов для лечения мукополисахаридозов Download PDF

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RU2744593C2
RU2744593C2 RU2017142006A RU2017142006A RU2744593C2 RU 2744593 C2 RU2744593 C2 RU 2744593C2 RU 2017142006 A RU2017142006 A RU 2017142006A RU 2017142006 A RU2017142006 A RU 2017142006A RU 2744593 C2 RU2744593 C2 RU 2744593C2
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cag
seq
aav9
nucleotide sequence
plasmid
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RU2017142006A
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RU2017142006A3 (enExample
RU2017142006A (ru
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ТУБЕРТ Мария Фатима БОШ
АУРИГОТ Вирджиния АРЕБА
МАЛЛОЛ Сандра МОТАС
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Эстев Фармасьютикалс, С.А.
Университат Аутонома Де Барселона
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
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    • 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
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • 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
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    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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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
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
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    • 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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Dermatology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
RU2017142006A 2015-06-05 2016-06-03 Векторы на основе аденоассоциированных вирусов для лечения мукополисахаридозов RU2744593C2 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP15382297.8A EP3101125A1 (en) 2015-06-05 2015-06-05 Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
EP15382297.8 2015-06-05
PCT/EP2016/062655 WO2016193431A1 (en) 2015-06-05 2016-06-03 Adenoassociated virus vectors for the treatment of mucopolysaccharidoses

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RU2017142006A RU2017142006A (ru) 2019-07-09
RU2017142006A3 RU2017142006A3 (enExample) 2020-02-03
RU2744593C2 true RU2744593C2 (ru) 2021-03-11

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US (1) US10617771B2 (enExample)
EP (2) EP3101125A1 (enExample)
JP (1) JP6850736B2 (enExample)
KR (1) KR102609858B1 (enExample)
CN (1) CN108138154B (enExample)
AU (1) AU2016273343B2 (enExample)
CA (1) CA2988201C (enExample)
CL (1) CL2017003096A1 (enExample)
CO (1) CO2017012562A2 (enExample)
ES (1) ES2790834T3 (enExample)
IL (1) IL255939B (enExample)
MX (1) MX386615B (enExample)
MY (1) MY184586A (enExample)
NZ (1) NZ738018A (enExample)
PH (1) PH12017502192B1 (enExample)
PT (1) PT3303576T (enExample)
RU (1) RU2744593C2 (enExample)
TN (1) TN2017000506A1 (enExample)
UA (1) UA125373C2 (enExample)
WO (1) WO2016193431A1 (enExample)
ZA (1) ZA201708039B (enExample)

Cited By (2)

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WO2023022629A1 (ru) * 2021-08-18 2023-02-23 Акционерное общество "ГЕНЕРИУМ" Направленная доставка терапевтических ферментов
RU2811100C1 (ru) * 2022-12-29 2024-01-11 Акционерное общество "ГЕНЕРИУМ" Соединение, содержащее терапевтический фермент и транспортный элемент, соединенные друг с другом непосредственно или при помощи линкера

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US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
US11085055B2 (en) 2014-12-05 2021-08-10 Universitat Autonoma De Barcelona Viral vectors for the treatment of diabetes
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
US11253612B2 (en) 2016-04-15 2022-02-22 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
KR20240093919A (ko) 2017-04-14 2024-06-24 리젠엑스바이오 인크. 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료
KR102765034B1 (ko) 2017-05-24 2025-02-11 유니버시타트 아우토노마 데 바르셀로나 섬유아세포 성장 인자 21 (ｆｇｆ21) 코딩 서열을 포함하는 바이러스 발현 컨스트럭트
EP3684938A1 (en) * 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
CN108715867A (zh) * 2018-05-31 2018-10-30 深圳市免疫基因治疗研究院 一种Sanfilippo B综合症慢病毒载体、慢病毒及其制备方法和应用
CN108795985A (zh) * 2018-05-31 2018-11-13 深圳市免疫基因治疗研究院 一种黏多糖贮积症慢病毒载体、慢病毒及其制备方法和应用
BR112021001498A2 (pt) * 2018-07-27 2021-04-27 Regenxbio Inc. vírus adeno-associado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav e métodos para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva)
EP3880810A1 (en) * 2018-11-15 2021-09-22 Esteve Pharmaceuticals, S.A. Animal model of mucopolysaccharidoses type iva
IL283452B2 (en) 2018-11-26 2026-04-01 Univ Barcelona Autonoma FIBROBLAST GROWTH FACTOR 21 (FGF21)-BASED GENE THERAPY
TWI923551B (zh) * 2019-03-28 2026-05-01 西班牙商艾斯提夫製藥股份有限公司 製造重組病毒載體之方法
ES2948620T3 (es) 2019-07-10 2023-09-14 Masonic Medical Res Laboratory Vgll4 con elemento regulador CIS de UCP-1 y método de uso del mismo
KR20220112283A (ko) * 2019-12-10 2022-08-10 다케다 야쿠힌 고교 가부시키가이샤 Hunter 질환 치료용 아데노-연합된 바이러스 벡터
WO2021207077A1 (en) * 2020-04-06 2021-10-14 Homology Medicines, Inc. Adeno-associated virus compositions for ids gene transfer and methods of use thereof
CN111718947B (zh) * 2020-06-18 2022-08-23 舒泰神（北京）生物制药股份有限公司 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途
CN114480454B (zh) * 2020-10-27 2024-03-29 华东理工大学 用于治疗粘多糖贮积症ⅱ型的腺相关病毒载体、药物及其应用
AU2022221284A1 (en) * 2021-02-10 2023-08-17 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
WO2022221421A2 (en) * 2021-04-13 2022-10-20 Capsida, Inc. Aav compositions with high brain expression for treating mucopolysaccharidosis ii
US20230055381A1 (en) * 2021-06-25 2023-02-23 Oxford Biomedica Solutions Llc Adeno-associated virus packaging systems

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023022629A1 (ru) * 2021-08-18 2023-02-23 Акционерное общество "ГЕНЕРИУМ" Направленная доставка терапевтических ферментов
RU2811100C1 (ru) * 2022-12-29 2024-01-11 Акционерное общество "ГЕНЕРИУМ" Соединение, содержащее терапевтический фермент и транспортный элемент, соединенные друг с другом непосредственно или при помощи линкера

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ES2790834T3 (es) 2020-10-29
EP3303576B1 (en) 2020-02-12
US20180169272A1 (en) 2018-06-21
WO2016193431A1 (en) 2016-12-08
TN2017000506A1 (en) 2019-04-12
PH12017502192B1 (en) 2022-09-02
US10617771B2 (en) 2020-04-14
KR20180012843A (ko) 2018-02-06
IL255939A (en) 2018-01-31
NZ738018A (en) 2025-03-28
PH12017502192A1 (en) 2018-06-11
MY184586A (en) 2021-04-06
BR112017025892A2 (pt) 2018-08-14
CA2988201C (en) 2024-01-16
ZA201708039B (en) 2018-11-28
CN108138154A (zh) 2018-06-08
MX386615B (es) 2025-03-19
CL2017003096A1 (es) 2018-08-10
AU2016273343A1 (en) 2017-12-21
RU2017142006A3 (enExample) 2020-02-03
IL255939B (en) 2021-05-31
EP3101125A1 (en) 2016-12-07
KR102609858B1 (ko) 2023-12-05
EP3303576A1 (en) 2018-04-11
CN108138154B (zh) 2022-01-18
JP6850736B2 (ja) 2021-03-31
JP2018517414A (ja) 2018-07-05
RU2017142006A (ru) 2019-07-09
MX2017015725A (es) 2018-08-15
CO2017012562A2 (es) 2018-02-28
AU2016273343B2 (en) 2022-04-28
CA2988201A1 (en) 2016-12-08
PT3303576T (pt) 2020-05-08
UA125373C2 (uk) 2022-03-02

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