ES2824829T3 - Vectores de virus adenoasociado que codifican G6PC modificada y usos de estos - Google Patents

Vectores de virus adenoasociado que codifican G6PC modificada y usos de estos Download PDF

Info

Publication number
ES2824829T3
ES2824829T3 ES15830932T ES15830932T ES2824829T3 ES 2824829 T3 ES2824829 T3 ES 2824829T3 ES 15830932 T ES15830932 T ES 15830932T ES 15830932 T ES15830932 T ES 15830932T ES 2824829 T3 ES2824829 T3 ES 2824829T3
Authority
ES
Spain
Prior art keywords
raav
g6pc
g6pase
vector
seq
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
ES15830932T
Other languages
English (en)
Spanish (es)
Inventor
Janice J Chou
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.)
US Department of Health and Human Services
Original Assignee
US Department of Health and Human Services
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 US Department of Health and Human Services filed Critical US Department of Health and Human Services
Application granted granted Critical
Publication of ES2824829T3 publication Critical patent/ES2824829T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • 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
    • 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
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • 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
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03009—Glucose-6-phosphatase (3.1.3.9)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Virology (AREA)
  • Endocrinology (AREA)
  • Urology & Nephrology (AREA)
  • Emergency Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
ES15830932T 2014-12-23 2015-12-22 Vectores de virus adenoasociado que codifican G6PC modificada y usos de estos Active ES2824829T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201462096400P 2014-12-23 2014-12-23
PCT/US2015/067338 WO2016106303A1 (en) 2014-12-23 2015-12-22 Adeno-associated virus vectors encoding modified g6pc and uses thereof

Publications (1)

Publication Number Publication Date
ES2824829T3 true ES2824829T3 (es) 2021-05-13

Family

ID=55272587

Family Applications (1)

Application Number Title Priority Date Filing Date
ES15830932T Active ES2824829T3 (es) 2014-12-23 2015-12-22 Vectores de virus adenoasociado que codifican G6PC modificada y usos de estos

Country Status (8)

Country Link
US (3) US10415044B2 (https=)
EP (1) EP3236984B1 (https=)
JP (2) JP6824169B2 (https=)
CN (1) CN107636153B (https=)
CA (1) CA2972038C (https=)
ES (1) ES2824829T3 (https=)
IL (1) IL253103B (https=)
WO (1) WO2016106303A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6824169B2 (ja) 2014-12-23 2021-02-03 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用
AU2018212002A1 (en) * 2017-01-30 2019-08-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Recombinant virus vectors for the treatment of glycogen storage disease
US11963974B2 (en) 2017-03-10 2024-04-23 National Center For Child Health And Development Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type Ia
CN111511915A (zh) 2018-03-09 2020-08-07 第一三共株式会社 糖原病Ia型治疗药
CA3107572A1 (en) * 2018-08-08 2020-02-13 Genethon Mini-gde for the treatment of glycogen storage disease iii
WO2020056147A2 (en) * 2018-09-13 2020-03-19 Modernatx, Inc. Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease
ES2969222T3 (es) * 2018-12-18 2024-05-17 Ultragenyx Pharmaceutical Inc Métodos y composiciones para el tratamiento de enfermedades del almacenamiento de glucógeno
EP3913060A1 (en) * 2020-05-22 2021-11-24 Genethon Vectors encoding a glucose-6-phosphatase (g6pase-a) for gene therapy
CN111972355A (zh) * 2020-08-31 2020-11-24 中国医学科学院北京协和医院 GSDIa型糖原累积症小鼠模型及其构建方法
EP4504947A4 (en) * 2022-04-07 2026-03-25 Univ Duke COMPOSITIONS AND METHODS TO PROMOTE LIVER REGENERATION BY GENE EDITING IN METABOLIC LIVER DISEASE
WO2025165616A1 (en) * 2024-01-31 2025-08-07 The Johns Hopkins University Compositions and methods for treating and preventing cancer

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6627425B1 (en) * 2000-06-02 2003-09-30 Millennium Pharmaceuticals, Inc. Human glucose-6-phosphatase molecules and uses thereof
DE602004030327D1 (de) 2003-05-21 2011-01-13 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
DK2242840T3 (da) 2008-01-29 2019-10-21 Applied Genetic Tech Corporation Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension
US20120093775A1 (en) 2009-03-27 2012-04-19 Proyecto De Biomedicina Cima, S.L. Methods and compositions for the treatment of cirrhosis and liver fibrosis
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
DK2529020T3 (en) 2010-01-28 2018-08-06 Childrens Hospital Philadelphia SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY
WO2015081101A1 (en) 2013-11-26 2015-06-04 The United States Of America, As Represented By The Secretary Department Of Health And Human Services Adeno-associated virus vectors for treatment of glycogen storage disease
JP6824169B2 (ja) 2014-12-23 2021-02-03 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ 改変ｇ６ｐｃをコードするアデノ随伴ウイルスベクターおよびその使用

Also Published As

Publication number Publication date
EP3236984A1 (en) 2017-11-01
US12440580B2 (en) 2025-10-14
CN107636153B (zh) 2020-12-11
JP2020054367A (ja) 2020-04-09
IL253103B (en) 2019-12-31
US20230144834A1 (en) 2023-05-11
CA2972038A1 (en) 2016-06-30
EP3236984B1 (en) 2020-07-22
WO2016106303A1 (en) 2016-06-30
US11535870B2 (en) 2022-12-27
CN107636153A (zh) 2018-01-26
US10415044B2 (en) 2019-09-17
JP2018501791A (ja) 2018-01-25
JP6824169B2 (ja) 2021-02-03
IL253103A0 (en) 2017-08-31
US20190345502A1 (en) 2019-11-14
CA2972038C (en) 2023-05-16
US20170362670A1 (en) 2017-12-21

Similar Documents

Publication Publication Date Title
ES2824829T3 (es) Vectores de virus adenoasociado que codifican G6PC modificada y usos de estos
ES2984259T3 (es) Capside de aav modificada con peptidos
ES2690643T3 (es) Vectores virales adeno-asociados para el tratamiento de enfermedad de almacenamiento de glucógeno
CN110606874B (zh) 用于基因转移到细胞、器官和组织中的变异aav和组合物、方法及用途
ES2989774T3 (es) Factor IX modificado, y composiciones, métodos y utilizaciones para la transferencia génica a células, órganos y tejidos
CN113518628A (zh) 治疗威尔逊病的基因疗法构建体
US12359219B2 (en) Transgene cassettes designed to express a human MECP2 gene
WO2023237748A1 (en) Peptide-modified aav capsid with enhanced muscle transduction efficiency
ES2978297T3 (es) Terapia génica para la esclerosis tuberosa
JP7641911B2 (ja) 新型の酵素組成物
JP2023526498A (ja) グルコース－６－ホスファターゼ（Ｇ６Ｐａｓｅ－ａ）をコードする遺伝子治療用ベクター
KR20230009444A (ko) 디스페린 이중 벡터에 의한 유전자 요법
ES2969222T3 (es) Métodos y composiciones para el tratamiento de enfermedades del almacenamiento de glucógeno
CN121866337A (zh) 经修饰的腺相关病毒载体及其在治疗神经疾病中的用途
CN121752725A (zh) 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途
RU2837753C2 (ru) Вариант aav, композиции и способы, в которых он используется, а также способы его применения для переноса генов в клетки, органы и ткани