TR201820102T4 - Fabri hastalığı gen tedavisi. - Google Patents

Fabri hastalığı gen tedavisi. Download PDF

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Publication number
TR201820102T4
TR201820102T4 TR2018/20102T TR201820102T TR201820102T4 TR 201820102 T4 TR201820102 T4 TR 201820102T4 TR 2018/20102 T TR2018/20102 T TR 2018/20102T TR 201820102 T TR201820102 T TR 201820102T TR 201820102 T4 TR201820102 T4 TR 201820102T4
Authority
TR
Turkey
Prior art keywords
galactosidase
vector
mice
fabri
levels
Prior art date
Application number
TR2018/20102T
Other languages
English (en)
Turkish (tr)
Inventor
Nathwani Amit
Raj Deepak
Original Assignee
Ucl Business Plc
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 Plc filed Critical Ucl Business Plc
Publication of TR201820102T4 publication Critical patent/TR201820102T4/tr

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    • 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
    • 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
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Environmental Sciences (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
TR2018/20102T 2015-05-11 2016-05-10 Fabri hastalığı gen tedavisi. TR201820102T4 (tr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB1508025.2A GB201508025D0 (en) 2015-05-11 2015-05-11 Fabry disease gene therapy

Publications (1)

Publication Number Publication Date
TR201820102T4 true TR201820102T4 (tr) 2019-01-21

Family

ID=53489437

Family Applications (1)

Application Number Title Priority Date Filing Date
TR2018/20102T TR201820102T4 (tr) 2015-05-11 2016-05-10 Fabri hastalığı gen tedavisi.

Country Status (17)

Country Link
US (2) US11103596B2 (enExample)
EP (2) EP3244931B1 (enExample)
JP (1) JP6688812B2 (enExample)
KR (1) KR20180012772A (enExample)
CN (1) CN107980063B (enExample)
AU (1) AU2016261453C1 (enExample)
CA (1) CA2985935C (enExample)
DK (1) DK3244931T3 (enExample)
ES (1) ES2703814T3 (enExample)
GB (1) GB201508025D0 (enExample)
HR (1) HRP20190165T1 (enExample)
LT (1) LT3244931T (enExample)
PL (1) PL3244931T3 (enExample)
PT (1) PT3244931T (enExample)
TR (1) TR201820102T4 (enExample)
TW (1) TW201706412A (enExample)
WO (1) WO2016181122A1 (enExample)

Families Citing this family (29)

* 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
EP3831842A1 (en) 2015-09-28 2021-06-09 The University of North Carolina at Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017201328A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，インコーポレイテッド 抗体を回避するウイルスベクター
KR20210006358A (ko) 2018-04-03 2021-01-18 스트라이드바이오 인코포레이티드 안 조직을 표적으로 하기 위한 바이러스 벡터
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
AU2019323747A1 (en) * 2018-08-20 2021-03-04 Ucl Business Ltd Factor ix encoding nucleotides
BR112021006829A2 (pt) 2018-10-10 2021-07-20 Amicus Therapeutics, Inc. composições de polipeptídeos estabilizados com ligações dissulfeto e métodos de uso
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
CN114423428A (zh) * 2019-08-07 2022-04-29 阿米库斯治疗学公司 治疗在gla基因中具有突变的患者的法布里病的方法
US11905523B2 (en) 2019-10-17 2024-02-20 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C
WO2021084275A1 (en) 2019-11-01 2021-05-06 Freeline Therapeutics Limited Factor viii polypeptide
WO2021183895A1 (en) * 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
KR20230026986A (ko) * 2020-04-27 2023-02-27 4디 몰레큘러 테라퓨틱스 아이엔씨. 코돈 최적화된 gla 유전자 및 이의 용도
KR20230002594A (ko) * 2020-04-29 2023-01-05 살리오젠 테라퓨틱스 인코포레이티드 가족성 고콜레스테롤혈증 및 상승된 저밀도 지단백질 콜레스테롤의 치료를 위한 조성물 및 방법
IL300728A (en) 2020-08-19 2023-04-01 Sarepta Therapeutics Inc Adeno-associated virus vectors for the 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
US20240024514A1 (en) * 2020-11-16 2024-01-25 Beijing Solobio Genetechnology Co., Ltd Recombinant adeno-associated viruses with enhanced liver tropism and uses thereof
KR20230173145A (ko) * 2021-04-20 2023-12-26 워킹 피쉬 테라퓨틱스 인코포레이티드 심각한 질병을 치료하기 위한 b 세포 기반 단백질 공장 조작
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
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
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
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
JPH11507231A (ja) 1995-06-07 1999-06-29 イェール ユニバーシティ アデノ関連ウイルスベクターの経口送達
US6458574B1 (en) 1996-09-12 2002-10-01 Transkaryotic Therapies, Inc. Treatment of a α-galactosidase a deficiency
US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
ATE286120T1 (de) * 1996-09-13 2005-01-15 Transkaryotic Therapies Inc Therapie für alpha galactosidase a insuffizienz
AU762400B2 (en) 1996-09-13 2003-06-26 Shire Human Genetic Therapies, Inc. Therapy for alpha-galactosidase a deficiency
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
ES2268799T3 (es) 1997-10-29 2007-03-16 Genzyme Corporation Terapia genica para la enfermedad de gaucher.
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
US20090017533A1 (en) 1998-09-29 2009-01-15 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
JP2003513632A (ja) 1999-11-05 2003-04-15 イェーナ・バイオサイエンス・ゲーエムベーハー 非病原性キネトプラスチダエのタンパク質発現システム
EP1232276B8 (en) 1999-11-16 2007-06-27 Genzyme Corporation Vectors and transgenes with regulatory elements for gene delivery to the liver
IL150314A0 (en) 1999-12-30 2002-12-01 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
AU2001236713A1 (en) 2000-02-17 2001-08-27 Genzyme Corporation Methods for treatment of lysosomal storage diseases
AU2001237050A1 (en) 2000-02-17 2001-08-27 Genzyme Corporation Genetic modification of the lung as a portal for gene delivery
US20020081654A1 (en) 2000-04-07 2002-06-27 Sandrin Mauro Sergio Targeting hydrolase enzymes
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker 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
EP1624067A3 (en) 2000-09-18 2006-03-15 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
ATE310096T1 (de) 2000-09-18 2005-12-15 Genzyme Corp Expressionsvektoren mit hybriden ubiquitin- promotoren
CA2425732A1 (en) 2000-10-11 2002-08-22 Transkaryotic Therapies, Inc. Optimized 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
JP2004535801A (ja) 2001-05-24 2004-12-02 ジェンザイム コーポレイション 筋特異的発現ベクター
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
US20030064000A1 (en) 2001-09-24 2003-04-03 Wilson Burgess Methods of sterilizing biological mixtures using stabilizer mixtures
US6783968B2 (en) 2001-09-24 2004-08-31 Clearant, Inc. Methods for sterilizing preparations of glycosidases
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
US20040258678A1 (en) 2002-02-22 2004-12-23 Genentech, Inc. Compositions and methods for the treatment of immune related diseases
ES2367257T3 (es) 2002-04-25 2011-10-31 Shire Human Genetic Therapies, Inc. Tratamiento de deficit de alfa-galactosidasa a.
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
CA2503390A1 (en) 2002-11-01 2004-05-21 Genentech, Inc. Compositions and methods for the treatment of immune related diseases
AU2003302386B2 (en) 2002-11-26 2010-04-01 Genentech, Inc. Compositions and methods for the treatment of immune related diseases
PT1587923E (pt) 2003-01-22 2011-12-07 Univ Duke Construções melhoradas para a expressão de polipéptidos lisossomais
ES2531478T3 (es) 2003-01-31 2015-03-16 The Mount Sinai School Of Medicine Of New York University Terapia de combinación para tratar trastornos de deficiencia de proteínas
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
WO2004083404A2 (en) 2003-03-19 2004-09-30 Isogenis, Inc. Gene therapy vectors having reduced immunogenicity based on cd8 alpha-chain
US7951557B2 (en) 2003-04-27 2011-05-31 Protalix Ltd. Human lysosomal proteins from plant cell culture
US20100196345A1 (en) 2003-04-27 2010-08-05 Protalix Production of high mannose proteins in plant culture
JP4979067B2 (ja) 2003-05-01 2012-07-18 ジェンザイム・コーポレーション 神経代謝性疾患についての遺伝子治療
WO2004098532A2 (en) 2003-05-05 2004-11-18 Genzyme Corporation Methods of reducing an immune response
CN107058313A (zh) 2003-06-24 2017-08-18 建新公司 新的β‑肌动蛋白和RPS21启动子及其应用
WO2005019258A2 (en) 2003-08-11 2005-03-03 Genentech, Inc. Compositions and methods for the treatment of immune related diseases
JP2007513188A (ja) 2003-12-04 2007-05-24 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ リソソーム蓄積症の処置のための組成物および方法
WO2005074888A2 (en) 2004-02-03 2005-08-18 Biodelivery Sciences International, Inc. Replacement enzyme cochleates
CA2556245A1 (en) 2004-02-06 2005-08-25 Biomarin Pharmaceutical Inc. 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
NZ555830A (en) 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
EP3520823B1 (en) 2005-05-02 2021-06-30 Genzyme Corporation Gene therapy for neurometabolic disorders
US8500720B2 (en) 2005-05-09 2013-08-06 Medtronic, Inc Method and apparatus for treatment of cardiac disorders
WO2007010533A2 (en) 2005-07-18 2007-01-25 Protalix Ltd. Mucosal or enteral administration of biologically active macromolecules
EP2250884B1 (en) 2005-10-31 2012-12-05 Velico Medical, Inc. Novel alpha-galactosidases
WO2007058381A1 (ja) 2005-11-18 2007-05-24 Tokyo Metropolitan Organization For Medical Research 基質特異性を変換した新規高機能酵素
AR059089A1 (es) 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
WO2007089632A2 (en) 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
EP3456331B1 (en) 2006-02-08 2021-05-26 Genzyme Corporation Gene therapy for niemann-pick disease type a
JP2009526066A (ja) 2006-02-09 2009-07-16 ジェンザイム・コーポレーション 遅速性脳室内送達
DK3457135T3 (da) 2006-05-16 2025-02-24 Amicus Therapeutics Inc Behandlingsmuligheder til fabrys sygdom
WO2008075957A1 (en) 2006-12-21 2008-06-26 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Treatment of fabry disease
DK2457920T3 (en) 2007-01-18 2018-01-22 Genzyme Corp Oligosaccharides comprising an amino oxy 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
CA2682441A1 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
JP2010525084A (ja) 2007-04-26 2010-07-22 アミカス セラピューティックス インコーポレイテッド 薬理シャペロンを用いたリソソーム蓄積症治療のための投薬計画
EP3252161B1 (en) 2007-06-06 2021-11-17 Genzyme Corporation Gene therapy for lysosomal storage diseases
EP2185187A1 (en) 2007-08-29 2010-05-19 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
WO2009066069A1 (en) 2007-11-21 2009-05-28 Summit Corporation Plc Treatment of protein folding disorders
AU2009209408B2 (en) 2008-01-29 2015-06-11 Beacon Therapeutics Limited Recombinant virus production using mammalian cells in suspension
PL3470077T3 (pl) 2008-02-12 2021-04-06 Amicus Therapeutics, Inc. Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi
WO2009114942A1 (en) 2008-03-20 2009-09-24 University Health Network Thymidylate kinase fusions and uses thereof
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
HUE025852T2 (en) 2008-06-26 2016-04-28 Orphazyme Aps Use of HSP70 to regulate enzyme activity
EP2323652A2 (en) 2008-08-06 2011-05-25 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
CN105879047A (zh) 2008-12-16 2016-08-24 建新公司 寡糖-蛋白缀合物
US8889641B2 (en) 2009-02-11 2014-11-18 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
WO2011019980A1 (en) 2009-08-14 2011-02-17 University Of Miami 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
PL2493487T3 (pl) 2009-10-27 2017-02-28 Erytech Pharma Kompozycja do indukowania swoistej tolerancji immunologicznej
WO2011057243A2 (en) 2009-11-09 2011-05-12 University Of Central Florida Research Foundation, Inc. Administration of plant expressed oral tolerance agents
US20120230974A1 (en) 2009-11-17 2012-09-13 Protalix Ltd Alkaline alpha galactosidase for the treatment of fabry disease
US9194011B2 (en) 2009-11-17 2015-11-24 Protalix Ltd. Stabilized alpha-galactosidase and uses thereof
RS54978B1 (sr) 2009-11-27 2016-11-30 Genzyme Corp Genz 112638 za lečenje gošeove ili fabrijeve bolesti u kombinacionoj terapiji
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
IN2012MN02262A (enExample) 2010-03-02 2015-06-12 Protalix Ltd
EP3284821B1 (en) 2010-03-23 2022-01-05 Precigen, Inc. Vectors conditionally expressing therapeutic proteins, host cells comprising the vectors, and uses thereof
US8809282B2 (en) 2010-05-06 2014-08-19 Duke University Method of reducing titers of antibodies specific for a therapeutic agent
RU2626514C2 (ru) 2010-06-25 2017-07-28 Шир Хьюман Дженетик Терапис, Инк. Доставка к цнс лечебных агентов
US20130224757A1 (en) 2010-08-19 2013-08-29 Novozymes A/S Induced sporulation screening method
EP2622088A2 (en) 2010-09-29 2013-08-07 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
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
JP5913372B2 (ja) 2011-01-20 2016-04-27 プロタリクス リミテッド 植物および植物細胞におけるα−ガラクトシダーゼの発現のための核酸発現構築物
CN103974619B (zh) 2011-03-11 2017-02-15 阿米库斯治疗学公司 治疗法布里病的给药方案
EP2691529B1 (en) 2011-03-31 2019-06-12 University of Iowa Research Foundation AAV2 particle comprising an AAV2 capsid protein and a vector comprising a nucleic acid encoding a tripeptidyl peptidase 1 (TPP1) for use in treating late infantile ceroid lipofuscinosis (LINCL) in a non-rodent mammal by intraventricular injection or ICV delivery.
US20120283290A1 (en) 2011-05-06 2012-11-08 Amicus Therapeutics Inc. Quantitation of gl3 in urine
EP4043025A1 (en) * 2011-06-08 2022-08-17 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
CA2860085A1 (en) 2011-12-22 2013-06-27 Centogene Ip Gmbh Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol
US9821114B2 (en) 2012-02-07 2017-11-21 Global Bio Therapeutics, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
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
JP6283655B2 (ja) 2012-03-29 2018-02-21 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド イオン化可能なカチオン性脂質
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
CN108949772A (zh) 2012-04-02 2018-12-07 现代泰克斯公司 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
AU2013243955B2 (en) 2012-04-02 2018-02-22 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9957495B2 (en) 2012-04-18 2018-05-01 Vib Vzw Means and methods for generating improved proteins
US11911487B2 (en) 2012-05-03 2024-02-27 Indiana University Research And Technology Corporation Hydrodynamic methods for delivering fluids to kidney tissues and related materials and methods
WO2013181454A1 (en) 2012-05-30 2013-12-05 Biostrategies LC Plant lectins as carriers of associated drug substances into animal and human cells
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
JP6225182B2 (ja) 2012-07-11 2017-11-01 サンガモ セラピューティクス， インコーポレイテッド リソソーム貯蔵疾患の処置のための方法および組成物
CA2917995C (en) 2012-07-17 2021-01-26 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
CN104662150B (zh) 2012-07-31 2018-07-10 比奥阿赛斯技术有限公司 脱磷酸化的溶酶体贮积症蛋白及其使用方法
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
WO2014089486A1 (en) 2012-12-07 2014-06-12 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
WO2014120900A1 (en) 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease
EP2970946A4 (en) 2013-03-13 2016-09-07 Philadelphia Children Hospital ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF
KR20150127582A (ko) 2013-03-14 2015-11-17 샤이어 휴먼 지네틱 테라피즈 인크. 4''-티오 개질된 뉴클레오티드를 갖는 리보핵산 및 관련 방법
WO2014143932A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
EP3714893A1 (en) 2013-03-15 2020-09-30 Amicus Therapeutics, Inc. Chemical crosslinkers
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses 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
WO2014201252A2 (en) 2013-06-13 2014-12-18 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
US10391184B2 (en) 2013-07-26 2019-08-27 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
JP6553033B2 (ja) 2013-08-13 2019-07-31 ノースウェスタン ユニバーシティ ペプチドコンジュゲート粒子
TWI793159B (zh) 2013-10-23 2023-02-21 美商健臻公司 重組醣蛋白及其用途
EA036394B1 (ru) 2013-10-24 2020-11-05 ЮНИКЬЮРЕ АйПи Б.В. Псевдотипированный вектор на основе аденоассоциированного вируса aav-5 для генной терапии неврологических заболеваний
RS60929B1 (sr) 2013-11-15 2020-11-30 Univ Pennsylvania Kompozicije za supresiju stvaranja inhibitora protiv faktora viii kod pacijenata sa hemofilijom a
WO2015089067A1 (en) 2013-12-11 2015-06-18 Genzyme Corporation Glucosylceramide synthase inhibitors
MX374840B (es) 2013-12-23 2025-03-06 Bcn Peptides Sa Analogos de bicalutamida o (s)-bicalutamida como compuestos activadores de la exocitosis para su uso en el tratamiento de un desorden de acumulo lisosomal o glucogenosis.
EP3102291A4 (en) 2014-02-04 2017-08-30 New York University Progranulin (pgrn) and its derivatives for diagnosis and treatment of lysosomal storage diseases
SI3137497T2 (sl) 2014-05-02 2025-07-31 Genzyme Corporation Vektorji AAV za gensko terapijo mrežnice in centralnega živčnega sistema
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
KR20240123409A (ko) 2014-09-07 2024-08-13 셀렉타 바이오사이언시즈, 인크. 유전자 편집 항-바이러스 전달 벡터 면역 반응을 약화시키기 위한 방법 및 조성물
EP3218000A2 (en) 2014-11-10 2017-09-20 Alexion Pharmaceuticals, Inc. Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof
TW201632626A (zh) 2014-11-21 2016-09-16 臺北榮民總醫院 製備誘導性多功能幹細胞之方法
ES2946969T3 (es) 2014-12-12 2023-07-28 CureVac SE Moléculas de ácido nucleico artificiales para una expresión proteica mejorada
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
ES2944889T3 (es) 2014-12-22 2023-06-26 Codexis Inc Variantes de alfa-galactosidasa humana
SG11201704681QA (en) 2014-12-30 2017-07-28 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
EP3247808B1 (en) 2015-01-21 2021-05-05 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
EP3256594B1 (en) 2015-02-10 2021-10-20 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex
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
ES2860712T3 (es) 2015-03-24 2021-10-05 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
WO2016154313A1 (en) 2015-03-24 2016-09-29 Zywie, Llc High drug loading liquid oral pharmaceutical compositions
US9981021B1 (en) 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof
CA3019315A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
HUE057795T2 (hu) 2015-05-15 2022-06-28 Regenxbio Inc Adenoasszociált vírus terápiás szerek központi idegrendszerbe történõ bejuttatására
WO2016197121A1 (en) 2015-06-05 2016-12-08 Dana-Farber Cancer Institute, Inc. Compositions and methods for transient gene therapy with enhanced stability
AU2016285473A1 (en) 2015-07-01 2018-01-18 The Regents Of The University Of California Retroviral vectors containing a reverse orientation human ubiquitin C promoter
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
MY187933A (en) 2015-12-08 2021-10-29 Regeneron Pharma Compositions and methods for internalizing enzymes
CN116478254A (zh) 2015-12-14 2023-07-25 北卡罗来纳大学教堂山分校 增强细小病毒载体递送的修饰衣壳蛋白
GB201522243D0 (en) 2015-12-16 2016-01-27 Ucl Business Plc Treatment
CN108779154B (zh) 2015-12-23 2022-12-27 丹尼斯科美国公司 增强的蛋白质产生及其方法
US20190365656A1 (en) 2016-01-04 2019-12-05 Cour Pharmaceuticals Development Company, Inc. Particles encapsulating fusion proteins containing linked epitopes
WO2017122093A1 (en) 2016-01-11 2017-07-20 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
JP2019504888A (ja) 2016-02-05 2019-02-21 エモリー ユニバーシティ 一本鎖アデノ随伴ウイルス９または自己相補型アデノ随伴ウイルス９の脳脊髄液への注射
EP3452101A2 (en) 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
US20180126003A1 (en) 2016-05-04 2018-05-10 Curevac Ag New targets for rna therapeutics

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