JP6688812B2 - ファブリー病の遺伝子治療 - Google Patents

ファブリー病の遺伝子治療 Download PDF

Info

Publication number
JP6688812B2
JP6688812B2 JP2017558727A JP2017558727A JP6688812B2 JP 6688812 B2 JP6688812 B2 JP 6688812B2 JP 2017558727 A JP2017558727 A JP 2017558727A JP 2017558727 A JP2017558727 A JP 2017558727A JP 6688812 B2 JP6688812 B2 JP 6688812B2
Authority
JP
Japan
Prior art keywords
vector
galactosidase
sequence
nucleic acid
aav
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.)
Expired - Fee Related
Application number
JP2017558727A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018520646A5 (https=
JP2018520646A (ja
Inventor
ナスワニ、アミット
ラジ、ディーパク
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.)
UCL Business Ltd
Original Assignee
UCL Business Ltd
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 UCL Business Ltd filed Critical UCL Business Ltd
Publication of JP2018520646A publication Critical patent/JP2018520646A/ja
Publication of JP2018520646A5 publication Critical patent/JP2018520646A5/ja
Application granted granted Critical
Publication of JP6688812B2 publication Critical patent/JP6688812B2/ja
Expired - Fee Related 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • 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
    • 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
    • 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)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • 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)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • 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
    • 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
    • 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
    • C12N2750/14171—Demonstrated in vivo effect
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Environmental Sciences (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2017558727A 2015-05-11 2016-05-10 ファブリー病の遺伝子治療 Expired - Fee Related JP6688812B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1508025.2A GB201508025D0 (en) 2015-05-11 2015-05-11 Fabry disease gene therapy
GB1508025.2 2015-05-11
PCT/GB2016/051328 WO2016181122A1 (en) 2015-05-11 2016-05-10 Fabry disease gene therapy

Publications (3)

Publication Number Publication Date
JP2018520646A JP2018520646A (ja) 2018-08-02
JP2018520646A5 JP2018520646A5 (https=) 2019-06-13
JP6688812B2 true JP6688812B2 (ja) 2020-04-28

Family

ID=53489437

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2017558727A Expired - Fee Related JP6688812B2 (ja) 2015-05-11 2016-05-10 ファブリー病の遺伝子治療

Country Status (17)

Country Link
US (2) US11103596B2 (https=)
EP (2) EP3470088A1 (https=)
JP (1) JP6688812B2 (https=)
KR (1) KR20180012772A (https=)
CN (1) CN107980063B (https=)
AU (1) AU2016261453C1 (https=)
CA (1) CA2985935C (https=)
DK (1) DK3244931T3 (https=)
ES (1) ES2703814T3 (https=)
GB (1) GB201508025D0 (https=)
HR (1) HRP20190165T1 (https=)
LT (1) LT3244931T (https=)
PL (1) PL3244931T3 (https=)
PT (1) PT3244931T (https=)
TR (1) TR201820102T4 (https=)
TW (1) TW201706412A (https=)
WO (1) WO2016181122A1 (https=)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
WO2017201328A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
US20210093735A1 (en) * 2018-04-26 2021-04-01 The University Of North Carolina At Chapel Hill Methods and compositions for treatment of hemophilia
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
JP2021533799A (ja) * 2018-08-20 2021-12-09 ユーシーエル ビジネス リミテッド 第ｉｘ因子をコードするヌクレオチド
CN113631182B (zh) 2018-10-10 2025-03-21 阿米库斯治疗学公司 二硫键稳定的多肽组合物和使用方法
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
KR20220044560A (ko) * 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
CN114929735A (zh) 2019-11-01 2022-08-19 自由行疗法有限公司 因子viii构建体
WO2021183895A1 (en) * 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
EP4103211A4 (en) * 2020-04-27 2023-08-23 4D Molecular Therapeutics Inc. CODON-OPTIMISED GLA GENES AND THEIR USES
KR20230002594A (ko) * 2020-04-29 2023-01-05 살리오젠 테라퓨틱스 인코포레이티드 가족성 고콜레스테롤혈증 및 상승된 저밀도 지단백질 콜레스테롤의 치료를 위한 조성물 및 방법
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
CN114507692B (zh) * 2020-11-16 2024-07-12 舒泰神（北京）生物制药股份有限公司 用于治疗法布里病的腺相关病毒载体及其用途
EP4244360A4 (en) * 2020-11-16 2025-05-07 Beijing SoloBio Genetechnology Company Ltd. Recombinant adeno-associated viruses with enhanced liver tropism and uses thereof
AU2022261885A1 (en) * 2021-04-20 2023-11-09 Walking Fish Therapeutics, Inc. Engineering b cell-based protein factories to treat serious diseases
GB202205514D0 (en) 2022-04-13 2022-05-25 Freeline Therapeutics Ltd Mechanical lysis
CN114672501B (zh) * 2022-05-27 2022-08-23 珠海丽凡达生物技术有限公司 一种mRNA、药物组合物及其应用
TW202423959A (zh) * 2022-08-24 2024-06-16 美商步行魚醫療公司 用於治療法布瑞氏症(fabry disease)之組成物及方法
AU2024257162A1 (en) * 2023-04-17 2025-11-06 Exegenesis Bio Singapore Pte. Ltd. Compositions and methods for gene therapies
CN116656704B (zh) * 2023-06-01 2024-11-05 华东理工大学 用于治疗法布里病的转基因表达盒
CN116622750B (zh) * 2023-06-01 2024-05-31 上海勉亦生物科技有限公司 优化的人法布里转基因表达盒及其用途
WO2025087695A1 (en) 2023-10-23 2025-05-01 Ascend Advanced Therapies Ltd Method for producing recombinant adeno-associated virus particles

Family Cites Families (189)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6846968B1 (en) 1988-02-26 2005-01-25 Large Scale Biology Corporation Production of lysosomal enzymes in plants by transient expression
US5179023A (en) 1989-03-24 1993-01-12 Research Corporation Technologies, Inc. Recombinant α-galactosidase a therapy for Fabry disease
CA2222574A1 (en) 1995-06-07 1996-12-19 Yale University Oral delivery of adeno-associated viral vectors
US6458574B1 (en) 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
MX340738B (es) 1996-09-13 2016-07-22 Shire Human Genetic Therapies Terapia para deficiencia de a-galactosidasa a.
AU762400B2 (en) 1996-09-13 2003-06-26 Shire Human Genetic Therapies, Inc. Therapy for alpha-galactosidase a deficiency
US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
US20020082224A1 (en) 1997-01-14 2002-06-27 Douglas J. Jolly Non-immunogenic prodrugs and selectable markers for use in gene therapy
GB9708526D0 (en) 1997-04-25 1997-06-18 Royal Free Hosp School Med Eukaryotic gene expression cassette and uses thereof
US6210666B1 (en) 1997-10-21 2001-04-03 Orphan Medical, Inc. Truncated α-galactosidase A to treat fabry disease
IL135578A0 (en) 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
EP1658857A1 (en) 1997-10-29 2006-05-24 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
US6607879B1 (en) 1998-02-09 2003-08-19 Incyte Corporation Compositions for the detection of blood cell and immunological response gene expression
CA2319212A1 (en) 1998-02-17 1999-08-19 Genzyme Corporation Methods for purified aav vector production
WO1999057296A1 (en) 1998-05-01 1999-11-11 Genzyme Corporation Partially deleted adenoviral vectors
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
US20090148906A1 (en) 1998-09-29 2009-06-11 Shire Human Genetic Therapies, Inc. A Delaware Corporation Optimized messenger rna
AU2004242550B2 (en) 1999-03-11 2008-04-03 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
GB9909066D0 (en) 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
US7927587B2 (en) 1999-08-05 2011-04-19 Regents Of The University Of Minnesota MAPC administration for the treatment of lysosomal storage disorders
WO2002064799A2 (en) 1999-09-28 2002-08-22 Transkaryotic Therapies, Inc. Optimized messenger rna
EP2275559A3 (en) 1999-09-28 2011-03-23 Shire Human Genetic Therapies, Inc. Optimized messenger RNA
WO2001032896A1 (en) 1999-11-05 2001-05-10 Jena Bioscience Gmbh Protein expression systems for non-pathogenic <i>kinetoplastidae</i>
ES2284545T3 (es) 1999-11-16 2007-11-16 Genzyme Corporation Vectores y transgenes con elementos reguladores para la administracion de genes del higado.
AU2352201A (en) 1999-12-30 2001-07-16 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
WO2001060414A2 (en) 2000-02-17 2001-08-23 Genzyme Corporation Genetic modification of the lung as a portal for gene delivery
AU2001236713A1 (en) 2000-02-17 2001-08-27 Genzyme Corporation Methods for treatment of lysosomal storage diseases
US20020081654A1 (en) 2000-04-07 2002-06-27 Sandrin Mauro Sergio Targeting hydrolase enzymes
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20020014242A1 (en) 2000-07-31 2002-02-07 Abraham Scaria Use of rapamycin to inhibit immune response and induce tolerance to gene therapy vector and encoded transgene products
JP2004516016A (ja) 2000-09-18 2004-06-03 ジェンザイム・コーポレイション ハイブリッドユビキチンプロモーターを含む発現ベクター
EP1624067A3 (en) 2000-09-18 2006-03-15 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
DK1325138T3 (da) 2000-10-11 2013-10-21 Shire Human Genetic Therapies Optimeret messenger rna
US7723296B2 (en) 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
JP4742191B2 (ja) 2001-06-14 2011-08-10 独立行政法人産業技術総合研究所 糖蛋白質およびその製造方法
US7063962B2 (en) 2001-07-20 2006-06-20 Novozymes A/S DNA sequences for regulating transcription
US6749851B2 (en) 2001-08-31 2004-06-15 Clearant, Inc. Methods for sterilizing preparations of digestive enzymes
US6783968B2 (en) 2001-09-24 2004-08-31 Clearant, Inc. Methods for sterilizing preparations of glycosidases
US20030064000A1 (en) 2001-09-24 2003-04-03 Wilson Burgess Methods of sterilizing biological mixtures using stabilizer mixtures
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1336411A1 (en) 2002-02-14 2003-08-20 Academisch Medisch Centrum bij de Universiteit van Amsterdam Compositions and methods for improving enzyme replacement therapy of lysosomal storage diseases
EP1575480A4 (en) 2002-02-22 2008-08-06 Genentech Inc COMPOSITIONS AND METHODS OF TREATING IMMUNE-INFLAMMATORY DISEASES
DK1503788T3 (da) 2002-04-25 2011-10-17 Shire Human Genetic Therapies Behandling af alpha-galactosidase A-deficiens
US20030225260A1 (en) 2002-04-30 2003-12-04 Snyder Richard O. Production of recombinant AAV virions
WO2003100031A2 (en) 2002-05-20 2003-12-04 Board Of Regents, The University Of Texas System Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone, and other tissues
US20030224477A1 (en) 2002-05-31 2003-12-04 Heartlein Michael W. Optimized promoter constructs
US20040033217A1 (en) 2002-05-31 2004-02-19 Padmavathy Vanguri Intraperitoneal delivery of genetically engineered mesenchymal stem cells
EP1578367A4 (en) 2002-11-01 2012-05-02 Genentech Inc COMPOSITIONS AND METHODS FOR THE TREATMENT OF IMMUNE DISEASES
JP2011516026A (ja) 2002-11-26 2011-05-26 ジェネンテック・インコーポレーテッド 免疫関連疾患の治療のための組成物と方法
ES2371913T3 (es) 2003-01-22 2012-01-11 Duke University Constructos mejorados para expresar polipéptidos lisosomales.
EP2857036B1 (en) 2003-01-31 2016-12-21 Mount Sinai School of Medicine of New York University Combination therapy for treating protein deficiency disorders
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
US20050042217A1 (en) 2003-03-19 2005-02-24 Yan Qi Specific inhibition of allorejection
US20100196345A1 (en) 2003-04-27 2010-08-05 Protalix Production of high mannose proteins in plant culture
US7951557B2 (en) 2003-04-27 2011-05-31 Protalix Ltd. Human lysosomal proteins from plant cell culture
EP1620133B1 (en) 2003-05-01 2015-12-09 Genzyme Corporation Gene therapy for neurometabolic disorders
EP1628628A4 (en) 2003-05-05 2007-03-21 Genzyme Corp METHODS FOR REDUCING AN IMMUNE RESPONSE
HUE036449T2 (hu) 2003-06-24 2018-07-30 Genzyme Corp Új béta-aktin és RPS21 promóterek és alkalmazásaik
WO2005019258A2 (en) 2003-08-11 2005-03-03 Genentech, Inc. Compositions and methods for the treatment of immune related diseases
EP1694345A1 (en) 2003-12-04 2006-08-30 Regents Of The University Of Minnesota Compositions and methods for the treatment of lysosomal storage disorders
WO2005074888A2 (en) 2004-02-03 2005-08-18 Biodelivery Sciences International, Inc. Replacement enzyme cochleates
US20080014188A1 (en) 2004-02-06 2008-01-17 Zankel Todd C Manufacture of Highly Phosphorylated Lysosomal Enzymes and Uses Thereof
US20050208090A1 (en) 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
US8889127B2 (en) 2004-07-01 2014-11-18 Icahn School Of Medicine At Mount Sinai Targeted protein replacement for the treatment of lysosomal storage disorders
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
JP5829372B2 (ja) 2005-05-02 2015-12-09 ジェンザイム・コーポレーション 神経代謝性疾患のための遺伝子治療
US8500720B2 (en) 2005-05-09 2013-08-06 Medtronic, Inc Method and apparatus for treatment of cardiac disorders
EP2484768A3 (en) 2005-07-18 2012-11-21 Protalix Ltd. Mucosal or enteral administration of biologically active macromolecules
WO2007053565A2 (en) 2005-10-31 2007-05-10 Zymequest, Inc. Novel a-galactosidases
BRPI0618583A2 (pt) 2005-11-18 2011-09-06 Tokyo Metropolitan Org Med Res enzima altamente funcional tendo especifidade ao substrato modificada
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
AR059371A1 (es) 2006-02-08 2008-03-26 Genzyme Corp Terapia genica para la enfermedad de niemann-pick tipo a
HUE040490T2 (hu) 2006-02-09 2019-03-28 Genzyme Corp Lassú intraventrikuláris szállítás
PL2533050T6 (pl) 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
WO2008075957A1 (en) 2006-12-21 2008-06-26 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Treatment of fabry disease
WO2008089403A2 (en) 2007-01-18 2008-07-24 Genzyme Corporation Oligosaccharides comprising an aminooxy group and conjugates thereof
CA2584494A1 (en) 2007-03-27 2008-09-27 Jeffrey A. Medin Vector encoding therapeutic polypeptide and safety elements to clear transduced cells
MX2009010557A (es) 2007-03-30 2009-11-19 Amicus Therapeutics Inc Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas.
MX2009011473A (es) 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
ES2635726T3 (es) 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
WO2009032171A1 (en) 2007-08-29 2009-03-12 Shire Human Genetic Therapies, Inc. Subcutaneous administration of alpha-galatosidase a
WO2009030254A1 (en) 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
EP2252288A1 (en) 2007-11-21 2010-11-24 Summit Corporation Plc Treatment of protein folding disorders
ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
US8568709B2 (en) * 2008-03-20 2013-10-29 University Health Network Thymidylate kinase fusions and uses thereof
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US8540985B2 (en) 2008-06-26 2013-09-24 Orphazyme Aps Use of Hsp70 as a regulator of enzymatic activity
WO2010015816A2 (en) 2008-08-06 2010-02-11 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
CN102369023A (zh) 2008-12-16 2012-03-07 建新公司 寡糖-蛋白缀合物
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US20120195876A1 (en) 2009-08-14 2012-08-02 Jochen Reiser Novel role of alpha-galactosidase activity as a biomarker in kidney disease
KR102000383B1 (ko) 2009-09-29 2019-07-15 유니버시테이트 젠트 만노스-1-포스포-6-만노스 결합의 포스포-6-만노스로의 가수분해
AU2010305284A1 (en) 2009-10-06 2012-05-03 Angiochem Inc. Compositions and methods for the transport of therapeutic agents
AU2010311515B2 (en) 2009-10-27 2014-02-20 Erytech Pharma Composition to induce specific immune tolerance
WO2011057243A2 (en) 2009-11-09 2011-05-12 University Of Central Florida Research Foundation, Inc. Administration of plant expressed oral tolerance agents
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
US20120230974A1 (en) 2009-11-17 2012-09-13 Protalix Ltd Alkaline alpha galactosidase for the treatment of fabry disease
CA3140959A1 (en) 2009-11-27 2011-06-03 Genzyme Corporation An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
SI2865751T1 (sl) 2010-03-02 2017-01-31 Protalix Ltd. Stabilizirana alfa-galaktozidaza in njene uporabe
KR20130010121A (ko) 2010-03-23 2013-01-25 인트렉손 코포레이션 치료적 단백질을 조건부로 발현하는 벡터，상기 벡터를 포함하는 숙주 세포 및 이의 용도
WO2011139379A2 (en) 2010-05-06 2011-11-10 Duke University A method of treating patients undergoing protein replacement therapy, gene replacement therapy, or other therapeutic modalities
LT2588130T (lt) 2010-06-25 2016-12-12 Shire Human Genetic Therapies, Inc. Terapinės priemonės pristatymas cns
US20130224757A1 (en) 2010-08-19 2013-08-29 Novozymes A/S Induced sporulation screening method
US9347050B2 (en) 2010-09-29 2016-05-24 Oxyrane Uk Limited Mannosidases capable of uncapping mannose-1-phospho-6-mannose linkages and demannosylating phosphorylated N-glycans and methods of facilitating mammalian cellular uptake of glycoproteins
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
KR101883803B1 (ko) 2011-01-20 2018-07-31 프로탈릭스 리미티드 식물 및 식물 세포에서 알파-갈락토시다제의 발현용 핵산 작제물
SG10201604757RA (en) 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
WO2012135857A1 (en) 2011-03-31 2012-10-04 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
US20120283290A1 (en) 2011-05-06 2012-11-08 Amicus Therapeutics Inc. Quantitation of gl3 in urine
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
KR102076585B1 (ko) 2011-12-22 2020-02-12 센토제너 아이피 게엠베하 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물
JP6188728B2 (ja) 2012-02-07 2017-08-30 グローバル・バイオ・セラピューティクス・インコーポレイテッドＧｌｏｂａｌ Ｂｉｏ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 核酸送達の区画化方法ならびにその組成物および使用
WO2013120497A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
WO2013151665A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
CN104411338A (zh) 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
WO2013156552A1 (en) 2012-04-18 2013-10-24 Vib Vzw Means and methods for generating improved proteins
CA2874316A1 (en) 2012-05-03 2013-11-07 Indiana University Research And Technology Corporation Hydrodynamic methods for delivering fluids to kidney tissues and related materials and methods
EP2854834A4 (en) 2012-05-30 2016-05-18 Biostrategies LC PLANT LENSES AS CARRIER CORRESPONDING AGENTS IN ANIMAL AND HUMAN CELLS
EP3196301B1 (en) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of monogenic diseases
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
WO2014014938A1 (en) 2012-07-17 2014-01-23 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
GB201213117D0 (en) 2012-07-24 2012-09-05 Ucl Business Plc Transgene expression
WO2014017915A2 (en) 2012-07-27 2014-01-30 Universiteit Utrecht Holding B.V. Urea and guanidinium derivatives of iminosugars
CA3140358A1 (en) 2012-07-31 2014-02-06 Bioasis Technologies, Inc. Dephosphorylated lysosomal storage disease proteins and methods of use thereof
US10138474B2 (en) 2012-08-17 2018-11-27 Research Foundation Of The City University Of New York Scavenger receptor uptake for fabry disease enzyme replacement therapy
EP4331620A3 (en) 2012-12-07 2024-12-04 Translate Bio, Inc. Lipidic nanoparticles for mrna delivery
WO2014120900A1 (en) 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease
CA2905952A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
EP3750903A1 (en) 2013-03-14 2020-12-16 Translate Bio, Inc. Ribonucleic acids with 4'-thio-modified nucleotides and related methods
WO2014143932A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
AU2014228231B2 (en) 2013-03-15 2019-02-21 Amicus Therapeutics, Inc. Chemical crosslinkers
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
CN115120746A (zh) 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
US20160122727A1 (en) 2013-06-13 2016-05-05 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
US20140377246A1 (en) 2013-06-19 2014-12-25 Carol Ann Foundation and International Morquio Organization Enzyme replacement therapy for treating mps vii related bone lesions using a chemically modified enzyme
EP3024497B1 (en) 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
KR20160042079A (ko) 2013-08-13 2016-04-18 노쓰웨스턴유니버시티 펩티드-접합된 입자
TWI793159B (zh) 2013-10-23 2023-02-21 美商健臻公司 重組醣蛋白及其用途
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
PT3068869T (pt) 2013-11-15 2020-10-08 Univ Pennsylvania Composições para supressão da formação de inibidores contra fator viii em pacientes de hemofilia a
EP3079695B1 (en) 2013-12-11 2021-03-17 Genzyme Corporation Glucosylceramide synthase inhibitors
NZ720499A (en) 2013-12-23 2020-04-24 Bcn Peptides Sa Bicalutamide analogs or (s)-bicalutamide as exocytosis activating compounds for use in the treatment of a lysosomal storage disorder or glycogenosis
US10357542B2 (en) 2014-02-04 2019-07-23 New York University Progranulin (PGRN) and its derivatives for diagnosis and treatment of lysosomal storage diseases
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
JP6652334B2 (ja) 2014-05-31 2020-02-19 Ｊｃｒファーマ株式会社 ウリジンとｎ−アセチル−ｄ−マンノサミンとを含有する培地
JP7242180B2 (ja) 2014-09-07 2023-03-20 セレクタ バイオサイエンシーズ インコーポレーテッド 抗ウイルス導入ベクター免疫応答を減弱化するための方法および組成物
US20180185495A1 (en) 2014-11-10 2018-07-05 Alexion Pharmaceuticals, Inc. Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof
US20170130205A1 (en) 2014-11-21 2017-05-11 Taipei Veterans General Hospital Method for preparing induced pluripotent stem cells
PT4023755T (pt) 2014-12-12 2023-07-05 CureVac SE Moléculas de ácido nucleico artificiais para uma expressão melhorada de proteína
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
LT3237621T (lt) 2014-12-22 2023-09-25 Codexis, Inc. Žmogaus alfa-galaktozidazės variantai
EP3240558A1 (en) 2014-12-30 2017-11-08 CureVac AG Artificial nucleic acid molecules
US10907176B2 (en) 2015-01-14 2021-02-02 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
WO2016118780A1 (en) 2015-01-21 2016-07-28 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
CN107530447A (zh) 2015-02-10 2018-01-02 建新公司 病毒颗粒至纹状体和皮质的增强递送
US20180030456A1 (en) 2015-02-19 2018-02-01 Danisco Us Inc. Enhanced protein expression
WO2016134338A1 (en) 2015-02-19 2016-08-25 University Of Florida Research Foundation, Inc. Recombinant aav vectors for gene therapy of human hematopoietic disorders
WO2016146760A1 (en) 2015-03-17 2016-09-22 Greenovation Biotech Gmbh Glycosylated lysosomal proteins, method of production and uses
WO2016154313A1 (en) 2015-03-24 2016-09-29 Zywie, Llc High drug loading liquid oral pharmaceutical compositions
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
US9981021B1 (en) 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof
EP3285788B1 (en) 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
HK1256341A1 (zh) 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
EP4219532A3 (en) 2015-06-05 2023-08-16 Dana-Farber Cancer Institute, Inc. Compositions and methods for transient gene therapy with enhanced stability
PE20180772A1 (es) 2015-07-01 2018-05-07 Univ California Vectores retrovirales que contienen un promotor de ubiquitina c humana de orientacion inversa
WO2017024137A1 (en) 2015-08-04 2017-02-09 New York University Progranulin (pgrn) fragments and derivatives for treatment or alleviation of lysosomal storage diseases
US20170051267A1 (en) 2015-08-20 2017-02-23 Research Foundation Of The City University Of New York Novel alpha-galactosidase a derivatives
WO2017049161A1 (en) 2015-09-18 2017-03-23 Duke University ACID ALPHA-GLUCOSIDASE AND A β-2 AGONIST FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDER
DK3386534T3 (da) 2015-12-08 2020-11-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
JP7406783B2 (ja) 2015-12-14 2023-12-28 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質
GB201522243D0 (en) 2015-12-16 2016-01-27 Ucl Business Plc Treatment
EP3380496B1 (en) 2015-12-23 2020-05-20 Danisco US Inc. Enhanced protein production and methods thereof
EP3400069A4 (en) 2016-01-04 2019-09-25 Cour Pharmaceuticals Development Company Inc. Particles for the Encapsulation of Fusion Proteins with Bundled Epitopes
US20190030128A1 (en) 2016-01-11 2019-01-31 Medgenics Medical Israel Ltd. Compositions and Methods for Treatment of Central Nervous System Diseases
AR107483A1 (es) 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
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
WO2017191274A2 (en) 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
US20180126003A1 (en) 2016-05-04 2018-05-10 Curevac Ag New targets for rna therapeutics

Also Published As

Publication number Publication date
AU2016261453B2 (en) 2019-03-28
AU2016261453A1 (en) 2017-11-23
CN107980063B (zh) 2021-12-10
KR20180012772A (ko) 2018-02-06
EP3470088A1 (en) 2019-04-17
EP3244931A1 (en) 2017-11-22
WO2016181122A1 (en) 2016-11-17
CA2985935A1 (en) 2016-11-17
US20220016263A1 (en) 2022-01-20
US20180110878A1 (en) 2018-04-26
CN107980063A (zh) 2018-05-01
US11103596B2 (en) 2021-08-31
JP2018520646A (ja) 2018-08-02
HRP20190165T1 (hr) 2019-04-05
DK3244931T3 (en) 2019-02-11
PL3244931T3 (pl) 2019-04-30
EP3244931B1 (en) 2018-10-24
TR201820102T4 (tr) 2019-01-21
ES2703814T3 (es) 2019-03-12
PT3244931T (pt) 2019-01-21
CA2985935C (en) 2022-03-22
US12370268B2 (en) 2025-07-29
AU2016261453C1 (en) 2019-09-12
TW201706412A (zh) 2017-02-16
LT3244931T (lt) 2019-02-11
GB201508025D0 (en) 2015-06-24

Similar Documents

Publication Publication Date Title
JP6688812B2 (ja) ファブリー病の遺伝子治療
US20220331409A1 (en) Factor ix gene therapy
AU2020200041B2 (en) Capsid-modified, rAAV3 vector compositions and uses in gene therapy of human liver cancer
EP3154560B1 (en) Methods and materials for increasing viral vector infectivity
KR20160026841A (ko) 스터퍼/필러 폴리누클레오티드 서열을 포함하는 벡터 및 사용 방법
KR20160033217A (ko) 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
US11077208B2 (en) Wilson's disease gene therapy
US10426845B2 (en) Diabetes gene therapy
WO2023140971A1 (en) Methods for treatment of ornithine transcarbamylase (otc) deficiency
US20240269328A1 (en) Recombinant adeno-associated viruses for lesch-nyhan disorders and uses thereof

Legal Events

Date Code Title Description
RD01 Notification of change of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7426

Effective date: 20180307

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20180307

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190509

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20190509

TRDD Decision of grant or rejection written
A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20200228

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20200310

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20200406

R150 Certificate of patent or registration of utility model

Ref document number: 6688812

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

LAPS Cancellation because of no payment of annual fees