AU2016261453C1 - Fabry disease gene therapy - Google Patents

Fabry disease gene therapy Download PDF

Info

Publication number
AU2016261453C1
AU2016261453C1 AU2016261453A AU2016261453A AU2016261453C1 AU 2016261453 C1 AU2016261453 C1 AU 2016261453C1 AU 2016261453 A AU2016261453 A AU 2016261453A AU 2016261453 A AU2016261453 A AU 2016261453A AU 2016261453 C1 AU2016261453 C1 AU 2016261453C1
Authority
AU
Australia
Prior art keywords
vector
nucleic acid
galactosidase
acid molecule
nucleotide sequence
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.)
Ceased
Application number
AU2016261453A
Other languages
English (en)
Other versions
AU2016261453B2 (en
AU2016261453A1 (en
Inventor
Amit Nathwani
Deepak RAJ
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 AU2016261453A1 publication Critical patent/AU2016261453A1/en
Application granted granted Critical
Publication of AU2016261453B2 publication Critical patent/AU2016261453B2/en
Publication of AU2016261453C1 publication Critical patent/AU2016261453C1/en
Assigned to UCL BUSINESS LTD reassignment UCL BUSINESS LTD Request to Amend Deed and Register Assignors: UCL BUSINESS PLC
Ceased 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
    • 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)
    • 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
    • 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)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hematology (AREA)
  • Obesity (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)
AU2016261453A 2015-05-11 2016-05-10 Fabry disease gene therapy Ceased AU2016261453C1 (en)

Applications Claiming Priority (3)

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

Publications (3)

Publication Number Publication Date
AU2016261453A1 AU2016261453A1 (en) 2017-11-23
AU2016261453B2 AU2016261453B2 (en) 2019-03-28
AU2016261453C1 true AU2016261453C1 (en) 2019-09-12

Family

ID=53489437

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2016261453A Ceased AU2016261453C1 (en) 2015-05-11 2016-05-10 Fabry disease gene therapy

Country Status (17)

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

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
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
EP3896164A1 (en) 2016-05-18 2021-10-20 ModernaTX, Inc. Polynucleotides encoding alpha-galactosidase a for the treatment of fabry disease
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
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
EP3810647A4 (en) * 2018-04-26 2022-08-17 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR THE TREATMENT OF HEMOPHILIA
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
SG11202101560QA (en) * 2018-08-20 2021-03-30 Ucl Business Ltd Factor ix encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
EA202190999A1 (ru) 2018-10-10 2021-10-15 Амикус Терапьютикс, Инк. Композиции на основе полипептидов, стабилизированных с помощью дисульфидной связи, и способы применения
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
CA3147055A1 (en) * 2019-08-07 2021-02-11 Elfrida Benjamin Methods of treating fabry disease in patients having a mutation in the gla gene
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
WO2021084277A2 (en) 2019-11-01 2021-05-06 Freeline Therapeutics Limited Transcription regulatory elements
WO2021183895A1 (en) * 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
TWI769795B (zh) 2020-04-27 2022-07-01 美商４Ｄ分子療法公司 密碼子優化的ｇｌａ基因及其用途
KR20230002594A (ko) * 2020-04-29 2023-01-05 살리오젠 테라퓨틱스 인코포레이티드 가족성 고콜레스테롤혈증 및 상승된 저밀도 지단백질 콜레스테롤의 치료를 위한 조성물 및 방법
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION 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
CA3197999A1 (en) * 2020-11-16 2022-05-19 Tingting Zhang Recombinant adeno-associated viruses with enhanced liver tropism and uses thereof
JP2024515113A (ja) * 2021-04-20 2024-04-04 ウォーキング・フィッシュ・セラピューティクス・インコーポレイテッド 重篤な疾患を処置するためのｂ細胞ベースのタンパク質工場の操作
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
WO2024044697A2 (en) * 2022-08-24 2024-02-29 Walking Fish Therapeutics, Inc. Compositions and methods for treatment of fabry disease
CN121002183A (zh) * 2023-04-17 2025-11-21 杭州复因生物科技有限公司 用于基因治疗的组合物和方法
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
AU719950B2 (en) 1995-06-07 2000-05-18 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
DK2374876T3 (en) 1996-09-13 2016-05-30 Shire Human Genetic Therapies A process for the purification of alpha-galactosidase 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
EP1658857A1 (en) 1997-10-29 2006-05-24 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
AU734290B2 (en) 1997-10-29 2001-06-07 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
AU750972B2 (en) 1998-02-17 2002-08-01 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
EP2275559A3 (en) 1999-09-28 2011-03-23 Shire Human Genetic Therapies, Inc. Optimized messenger RNA
WO2002064799A2 (en) 1999-09-28 2002-08-22 Transkaryotic Therapies, Inc. Optimized messenger rna
ES2301495T3 (es) 1999-11-05 2008-07-01 Jena Bioscience Gmbh Sistemas de expresion de proteinas para kinetoplastidae no patogenos.
ATE360082T1 (de) 1999-11-16 2007-05-15 Genzyme Corp Vektoren und transgene mit regulatorischen elementen zur genverabreichung in leber
AU2352201A (en) 1999-12-30 2001-07-16 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
WO2001060377A2 (en) 2000-02-17 2001-08-23 Genzyme Corporation Methods for treatment of lysosomal storage diseases using biphosphonates
JP2003533437A (ja) 2000-02-17 2003-11-11 ジェンザイム・コーポレイション 遺伝子デリバリーのための門戸としての肺の遺伝的修飾
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
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation 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
DE60115070T2 (de) 2000-09-18 2006-07-27 Genzyme Corp., Cambridge 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
CA2448120A1 (en) 2001-05-24 2002-11-28 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
WO2003072035A2 (en) 2002-02-22 2003-09-04 Genentech, Inc. Compositions and methods for the treatment of immune related diseases
JP5570677B2 (ja) 2002-04-25 2014-08-13 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 α−ガラクトシダーゼＡ欠損症の治療
US20030225260A1 (en) 2002-04-30 2003-12-04 Snyder Richard O. Production of recombinant AAV virions
AU2003220115A1 (en) 2002-05-20 2003-12-12 Board Of Regents, The University Of Texas System Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone, and other tissues
WO2003101202A1 (en) 2002-05-31 2003-12-11 Osiris Therapeutics, Inc. Intraperitoneal delivery of genetically engineered mesenchymal stem cells
US20030224477A1 (en) 2002-05-31 2003-12-04 Heartlein Michael W. Optimized promoter constructs
JP2006515747A (ja) 2002-11-01 2006-06-08 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
EP2314676A1 (en) 2002-11-26 2011-04-27 Genentech, Inc. Compositions and methods for the treatment of immune related diseases
US20040248262A1 (en) 2003-01-22 2004-12-09 Koeberl Dwight D. Constructs for expressing lysomal polypeptides
PT2444102E (pt) 2003-01-31 2015-09-17 Sinai School Medicine Terapia combinada para o tratamento de distúrbios de deficiência proteica
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
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
PL1620133T3 (pl) 2003-05-01 2016-05-31 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
WO2004098532A2 (en) 2003-05-05 2004-11-18 Genzyme Corporation Methods of reducing an immune response
SI2332972T1 (en) 2003-06-24 2018-05-31 Genzyme Corporation New beta-actin and rpS21 promoters and their use
WO2005019258A2 (en) 2003-08-11 2005-03-03 Genentech, Inc. Compositions and methods for the treatment of immune related diseases
AU2004296848A1 (en) 2003-12-04 2005-06-23 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
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
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
ES2887076T3 (es) 2005-05-02 2021-12-21 Genzyme Corp Terapia génica para trastornos neurometabólicos
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
EP1961816B1 (en) 2005-11-18 2015-09-02 Tokyo Metropolitan Institute of Medical Science Novel highly functional enzyme having modified substrate-specificity
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
LT1986661T (lt) 2006-02-08 2018-12-10 Genzyme Corporation Nimano-piko a tipo ligos genų terapija
MX361816B (es) 2006-02-09 2018-12-17 Genzyme Corp Suministro intraventricular lento.
FI3457135T3 (fi) 2006-05-16 2025-03-08 Amicus Therapeutics Inc Fabryn taudin hoitovaihtoehtoja
EP2121005A1 (en) 2006-12-21 2009-11-25 Academisch Ziekenhuis bij de Universiteit van Amsterdam Treatment of fabry disease
US8759501B2 (en) 2007-01-18 2014-06-24 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
WO2008121826A2 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of fabry disease using pharmacological chaperones
WO2008134628A2 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases 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
PL3252161T3 (pl) 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych
US20100291060A1 (en) 2007-08-29 2010-11-18 Shire Human Genetic Therapies, Inc Subcutaneous administration of alpha-galactosidase 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
WO2009097129A1 (en) 2008-01-29 2009-08-06 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
HUE026543T2 (hu) 2008-02-12 2016-06-28 Amicus Therapeutics Inc Eljárás betegségek gyógyászati chaperonnal történõ kezelésére adott válasz elõrejelzésére
US8568709B2 (en) * 2008-03-20 2013-10-29 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
PL2659904T3 (pl) 2008-06-26 2016-01-29 Orphazyme Aps Zastosowanie Hsp70 jako regulatora aktywności enzymatycznej
EP2323652A2 (en) 2008-08-06 2011-05-25 Summit Corporation Plc Treatment of lysosomal storage disorders and other proteostatic diseases
PT2465542E (pt) 2008-12-16 2015-04-16 Genzyme Corp Conjugados oligossacárido-proteína
EP2396343B1 (en) 2009-02-11 2017-05-17 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
WO2011041897A1 (en) 2009-10-06 2011-04-14 Angiochem Inc. Compositions and methods for the transport of therapeutic agents
PT2493487T (pt) 2009-10-27 2016-11-08 Erytech Pharma Composição para induzir uma tolerância imunitária específica
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
ES2754398T3 (es) 2009-11-27 2020-04-17 Genzyme Corp Eliglustat (Genz 112638) como inhibidor de glucosilceramida-sintasa para uso en un método de tratamiento de la enfermedad de fabry o de gaucher, comprendiendo el método ajustar la dosis terapéutica individual al metabolismo P-450 del paciente
US9169494B2 (en) 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
HUE030959T2 (en) 2010-03-02 2017-06-28 Protalix Ltd Stabilized alpha-galactosidase and its uses
TWI688395B (zh) 2010-03-23 2020-03-21 英翠克頌公司 條件性表現治療性蛋白質之載體、包含該載體之宿主細胞及彼等之用途
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
EP3103469B1 (en) 2010-06-25 2020-12-09 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
US20130224757A1 (en) 2010-08-19 2013-08-29 Novozymes A/S Induced sporulation screening method
SG189108A1 (en) 2010-09-29 2013-05-31 Oxyrane Uk Ltd 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
AU2011356137A1 (en) 2011-01-20 2013-08-15 Protalix Ltd. Nucleic acid construct for expression of alpha-galactosidase in plants and plant cells
SG10201604757RA (en) * 2011-03-11 2016-08-30 Amicus Therapeutics Inc Dosing regimens for the treatment of fabry disease
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
JP6184945B2 (ja) 2011-06-08 2017-08-23 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 ｍＲＮＡ送達のための脂質ナノ粒子組成物および方法
JP6320931B2 (ja) 2011-12-22 2018-05-09 セントジーン アイピー ゲーエムベーハー リソソーム酵素を再構成する能力を有する化合物ならびにアンブロキソールおよび／またはアンブロキソールの誘導体の組み合わせ
AU2013216920B2 (en) 2012-02-07 2016-09-22 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
ES2762873T3 (es) 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
AU2013243949A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
AU2013243954A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
WO2013156552A1 (en) 2012-04-18 2013-10-24 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
US20130323221A1 (en) 2012-05-30 2013-12-05 Biostrategies LC Plant lectins as carriers of associated drug substances into animal and human cells
EP3816281A1 (en) 2012-07-11 2021-05-05 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
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
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
WO2014160092A1 (en) 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
JP6567494B2 (ja) 2013-03-14 2019-08-28 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 ４’−チオ−修飾ヌクレオチドを有するリボ核酸及び関連方法
US9447433B2 (en) 2013-03-15 2016-09-20 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
CA2902210C (en) 2013-03-15 2021-07-20 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
EP3622821A1 (en) 2013-05-15 2020-03-18 Regents of the University of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
EP3008191A2 (en) 2013-06-13 2016-04-20 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
DK3024497T3 (da) 2013-07-26 2021-04-12 Univ Iowa Res Found Fremgangsmåder og præparater til behandling af hjernesygdomme
MX372849B (es) 2013-08-13 2020-07-06 Univ Northwestern Partículas conjugadas con péptidos.
TWI642782B (zh) 2013-10-23 2018-12-01 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
WO2015073988A1 (en) 2013-11-15 2015-05-21 Trustees Of The University Of Pennsylvania Compositions and methods for suppression of inhibitor formation against factor viii in hemophilia a patients
CN105979945A (zh) 2013-12-11 2016-09-28 建新公司 葡糖神经酰胺合酶抑制剂
RU2685269C2 (ru) 2013-12-23 2019-04-17 БКН Пептидес, С.А. Аналоги бикалутамида или (s)-бикалутамид в качестве активирующих экзоцитоз соединений для применения в лечении расстройства лизосомного накопления или гликогеноза
US10357542B2 (en) 2014-02-04 2019-07-23 New York University Progranulin (PGRN) and its derivatives for diagnosis and treatment of lysosomal storage diseases
AU2015252797C1 (en) 2014-05-02 2021-04-22 Genzyme Corporation AAV vectors for retinal and CNS gene therapy
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
WO2016037162A1 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector immune responses
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
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
US11149278B2 (en) 2014-12-12 2021-10-19 Curevac Ag Artificial nucleic acid molecules for improved protein expression
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
US20170360900A1 (en) 2014-12-22 2017-12-21 Codexis, Inc. Human alpha-galactosidase variants
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
EP3247808B1 (en) 2015-01-21 2021-05-05 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
SG11201706445SA (en) 2015-02-10 2017-09-28 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
US20180135074A1 (en) 2015-02-19 2018-05-17 University Of Florida Research Foundation, Incorporated Recombinant aav vectors for gene therapy of human hematopoietic disorders
KR20170116159A (ko) 2015-02-19 2017-10-18 다니스코 유에스 인크. 향상된 단백질 발현
ES2899783T3 (es) 2015-03-17 2022-03-14 eleva GmbH Proteínas lisosómicas glucosiladas, método de producción y utilizaciones
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
WO2016172155A1 (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
SMT202200022T1 (it) 2015-05-15 2022-03-21 Regenxbio Inc Virus adeno-associato per veicolazione terapeutica al sistema nervoso centrale
EP3303377B1 (en) 2015-06-05 2022-11-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for transient gene therapy with enhanced stability
JP2018519827A (ja) 2015-07-01 2018-07-26 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 逆配向のヒトユビキチンｃプロモーターを含むレトロウイルスベクター
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
DK3782639T3 (da) 2015-12-08 2022-08-29 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
US11124546B2 (en) 2015-12-14 2021-09-21 The University Of North Carolina At Chapel Hill Modified capsid proteins for enhanced delivery of parvovirus vectors
GB201522243D0 (en) 2015-12-16 2016-01-27 Ucl Business Plc Treatment
WO2017112733A1 (en) 2015-12-23 2017-06-29 Danisco Us Inc. Enhanced protein production and methods thereof
CA3009799A1 (en) 2016-01-04 2017-07-13 Cour Pharmaceuticals Development Company Inc. Particles encapsulating fusion proteins containing linked epitopes
EP3402531A4 (en) 2016-01-11 2019-08-14 Medgenics Medical Israel Ltd COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISEASES OF THE CENTRAL NERVOUS SYSTEM
AR107483A1 (es) 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
SI3411484T1 (sl) 2016-02-05 2023-12-29 Emory University Injiciranje enojnega ali samokomplementarnega adeno-povezanega virusa 9 v cerebrospinalno tekočino
US20180126003A1 (en) 2016-05-04 2018-05-10 Curevac Ag New targets for rna therapeutics
US11078247B2 (en) 2016-05-04 2021-08-03 Curevac Ag RNA encoding a therapeutic protein

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
R ZIEGLER, MOLECULAR THERAPY, GB, (2004-02-01), vol. 9, no. 2, doi:10.1016/j.ymthe.2003.11.015, ISSN 1525-0016, pages 231 - 240 *

Also Published As

Publication number Publication date
LT3244931T (lt) 2019-02-11
JP2018520646A (ja) 2018-08-02
US11103596B2 (en) 2021-08-31
TW201706412A (zh) 2017-02-16
US20180110878A1 (en) 2018-04-26
US20220016263A1 (en) 2022-01-20
AU2016261453B2 (en) 2019-03-28
EP3244931A1 (en) 2017-11-22
GB201508025D0 (en) 2015-06-24
DK3244931T3 (en) 2019-02-11
CN107980063B (zh) 2021-12-10
TR201820102T4 (tr) 2019-01-21
HRP20190165T1 (hr) 2019-04-05
ES2703814T3 (es) 2019-03-12
CA2985935C (en) 2022-03-22
CA2985935A1 (en) 2016-11-17
CN107980063A (zh) 2018-05-01
PT3244931T (pt) 2019-01-21
AU2016261453A1 (en) 2017-11-23
EP3470088A1 (en) 2019-04-17
US12370268B2 (en) 2025-07-29
EP3244931B1 (en) 2018-10-24
PL3244931T3 (pl) 2019-04-30
KR20180012772A (ko) 2018-02-06
JP6688812B2 (ja) 2020-04-28
WO2016181122A1 (en) 2016-11-17

Similar Documents

Publication Publication Date Title
US12370268B2 (en) Fabry disease gene therapy
US20220331409A1 (en) Factor ix gene therapy
US20250236887A1 (en) Adeno-associated virus (aav) systems for treatment of genetic hearing loss
US12023386B2 (en) Composition for treatment of Crigler-Najjar syndrome
US12331331B2 (en) Polynucleotides
US11077208B2 (en) Wilson's disease gene therapy
CN109762831B (zh) 用于治疗3a型粘多糖贮积症的基因药物构建体
US10426845B2 (en) Diabetes gene therapy
US20250177563A1 (en) Adeno-Associated Viral (AAV)-Mediated Sphingosine-1-Phosphate Lyase (SPL) Expression for Treating Pulmonary Fibrosis

Legal Events

Date Code Title Description
DA2 Applications for amendment section 104

Free format text: THE NATURE OF THE AMENDMENT IS AS SHOWN IN THE STATEMENT(S) FILED 06 JUN 2019

DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS AS SHOWN IN THE STATEMENT FILED 06 JUN 2019

FGA Letters patent sealed or granted (standard patent)
HB Alteration of name in register

Owner name: UCL BUSINESS LTD

Free format text: FORMER NAME(S): UCL BUSINESS PLC

MK14 Patent ceased section 143(a) (annual fees not paid) or expired