CN109863243B - 用于治疗粘多糖症的腺相关病毒载体 - Google Patents

用于治疗粘多糖症的腺相关病毒载体 Download PDF

Info

Publication number
CN109863243B
CN109863243B CN201780060410.7A CN201780060410A CN109863243B CN 109863243 B CN109863243 B CN 109863243B CN 201780060410 A CN201780060410 A CN 201780060410A CN 109863243 B CN109863243 B CN 109863243B
Authority
CN
China
Prior art keywords
vector
cag
aav
gns
aav9
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.)
Active
Application number
CN201780060410.7A
Other languages
English (en)
Chinese (zh)
Other versions
CN109863243A (zh
Inventor
卡莱斯·罗加·莱哈
维尔吉尼娅·A·奥里戈特-门敦卡
法蒂玛·博希·图贝特
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Esteven Pharmaceutical Co ltd
Universitat Autonoma de Barcelona UAB
Original Assignee
Esteven Pharmaceutical Co ltd
Universitat Autonoma de Barcelona UAB
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 Esteven Pharmaceutical Co ltd, Universitat Autonoma de Barcelona UAB filed Critical Esteven Pharmaceutical Co ltd
Publication of CN109863243A publication Critical patent/CN109863243A/zh
Application granted granted Critical
Publication of CN109863243B publication Critical patent/CN109863243B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06014—N-Acetylglucosamine-6-sulfatase (3.1.6.14)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0362—Animal model for lipid/glucose metabolism, e.g. obesity, type-2 diabetes
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • 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
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/90—Vector systems having a special element relevant for transcription from vertebrates avian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
CN201780060410.7A 2016-09-30 2017-09-22 用于治疗粘多糖症的腺相关病毒载体 Active CN109863243B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16382450.1 2016-09-30
EP16382450 2016-09-30
PCT/EP2017/074081 WO2018060097A1 (en) 2016-09-30 2017-09-22 Adenoassociated virus vectors for the treatment of mucopolysaccharidoses

Publications (2)

Publication Number Publication Date
CN109863243A CN109863243A (zh) 2019-06-07
CN109863243B true CN109863243B (zh) 2023-07-28

Family

ID=57123944

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201780060410.7A Active CN109863243B (zh) 2016-09-30 2017-09-22 用于治疗粘多糖症的腺相关病毒载体

Country Status (10)

Country Link
US (1) US11338046B2 (enExample)
EP (1) EP3519569B1 (enExample)
JP (1) JP7059285B2 (enExample)
CN (1) CN109863243B (enExample)
AR (1) AR109752A1 (enExample)
AU (1) AU2017333336B2 (enExample)
ES (1) ES2859661T3 (enExample)
PT (1) PT3519569T (enExample)
TW (1) TW201814044A (enExample)
WO (1) WO2018060097A1 (enExample)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
RU2692251C2 (ru) 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
US10973931B2 (en) 2014-09-16 2021-04-13 Universitat Autònoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
ES2824457T3 (es) 2014-12-05 2021-05-12 UNIV AUTòNOMA DE BARCELONA Vectores virales para el tratamiento de la diabetes
ES2900973T3 (es) 2015-01-07 2022-03-21 UNIV AUTòNOMA DE BARCELONA Constructo genético de vector individual que comprende genes de insulina y glucoquinasa
BR112020021903A2 (pt) 2018-04-27 2021-03-02 Rocket Pharmaceuticals, Ltd. terapia genética para degeneração do snc
EP3802803A1 (en) * 2018-05-30 2021-04-14 Esteve Pharmaceuticals, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a
CA3106010A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
WO2021007473A1 (en) 2019-07-10 2021-01-14 Masonic Medical Research Institute Vgll4 with ucp-1 cis-regulatory element and method 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
CN116194154A (zh) 2020-08-07 2023-05-30 太空飞船七有限责任公司 使用aav载体的plakophilin-2(pkp2)基因疗法
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

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101444621A (zh) * 2003-02-11 2009-06-03 夏尔人类遗传性治疗公司 使用甲酰-甘氨酸生成酶(fge)对多种硫酸酯酶缺乏症和其它病症进行诊断和治疗
WO2015092440A1 (en) * 2013-12-20 2015-06-25 Oxford Biomedica (Uk) Limited Viral vector production system
WO2015173308A1 (en) * 2014-05-14 2015-11-19 Laboratorios Del Dr. Esteve S.A. Adenoassociated virus vectors for the treatment of lysosomal storage disorders
CN105745326A (zh) * 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
WO2016130591A2 (en) * 2015-02-10 2016-08-18 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex
CN105916990A (zh) * 2013-08-02 2016-08-31 巴塞罗那自治大学 用于转导脂肪组织的腺相关病毒载体

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7232670B2 (en) * 2001-09-28 2007-06-19 St. Jude Children's Research Hospital Targeting proteins to cells expressing mannose receptors via expression in insect cells
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
CN107982548A (zh) 2012-02-07 2018-05-04 全球生物疗法有限公司 核酸输送的区室化方法及其组合物和应用
US9981021B1 (en) * 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101444621A (zh) * 2003-02-11 2009-06-03 夏尔人类遗传性治疗公司 使用甲酰-甘氨酸生成酶(fge)对多种硫酸酯酶缺乏症和其它病症进行诊断和治疗
CN105916990A (zh) * 2013-08-02 2016-08-31 巴塞罗那自治大学 用于转导脂肪组织的腺相关病毒载体
CN105745326A (zh) * 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
WO2015092440A1 (en) * 2013-12-20 2015-06-25 Oxford Biomedica (Uk) Limited Viral vector production system
WO2015173308A1 (en) * 2014-05-14 2015-11-19 Laboratorios Del Dr. Esteve S.A. Adenoassociated virus vectors for the treatment of lysosomal storage disorders
WO2016130591A2 (en) * 2015-02-10 2016-08-18 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex

Also Published As

Publication number Publication date
AU2017333336B2 (en) 2023-11-09
AU2017333336A1 (en) 2019-04-11
JP2019528793A (ja) 2019-10-17
US11338046B2 (en) 2022-05-24
TW201814044A (zh) 2018-04-16
EP3519569B1 (en) 2020-12-02
CN109863243A (zh) 2019-06-07
ES2859661T3 (es) 2021-10-04
US20190231900A1 (en) 2019-08-01
AR109752A1 (es) 2019-01-23
EP3519569A1 (en) 2019-08-07
WO2018060097A1 (en) 2018-04-05
JP7059285B2 (ja) 2022-04-25
PT3519569T (pt) 2021-03-04

Similar Documents

Publication Publication Date Title
CN109863243B (zh) 用于治疗粘多糖症的腺相关病毒载体
AU2018261769B2 (en) Compositions and methods for expressing Otoferlin
AU2020207840B2 (en) Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
CA2187626C (en) Aav-mediated delivery of dna to cells of the nervous system
KR20210118112A (ko) 오토페를린 유전자을 회복시키는 aav-매개된 유전자 치료요법
EP2579900B1 (en) Vectors and sequences for the treatment of diseases
CN108822217A (zh) 一种基因碱基编辑器
JP2018517414A (ja) ムコ多糖症治療用のアデノ随伴ウイルスベクター
KR20230040328A (ko) 인플루엔자 헤마글루티닌을 발현하는 재조합 hvt 벡터 및 면역원성 조성물, 및 이의 제조 및 용도
JP7369403B2 (ja) ムコ多糖症ｉｖａ型治療用のアデノ随伴ウイルスベクター
CN115698300A (zh) 佛司可林诱导型启动子和低氧诱导型启动子
HK40087492A (zh) 佛司可林诱导型启动子和低氧诱导型启动子
HK40012896A (en) Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
HK1184071A (en) Vectors and sequences for the treatment of diseases
HK1184071B (en) Vectors and sequences for the treatment of diseases

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant