DK3292875T3 - Compositions and methods for treating diseases - Google Patents

Compositions and methods for treating diseases Download PDF

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Publication number
DK3292875T3
DK3292875T3 DK17184780.9T DK17184780T DK3292875T3 DK 3292875 T3 DK3292875 T3 DK 3292875T3 DK 17184780 T DK17184780 T DK 17184780T DK 3292875 T3 DK3292875 T3 DK 3292875T3
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Prior art keywords
compositions
methods
treating diseases
diseases
treating
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Application number
DK17184780.9T
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English (en)
Inventor
Barry John Byrne
Darin J Falk
Christina Pacak
Lara Robert Deruisseau
Cathryn Mah
David D Fuller
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Univ Florida
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Priority claimed from US13/527,350 external-priority patent/US20120322861A1/en
Application filed by Univ Florida filed Critical Univ Florida
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Publication of DK3292875T3 publication Critical patent/DK3292875T3/da

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16641—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16644—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Obesity (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hematology (AREA)
  • Dermatology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Mycology (AREA)
DK17184780.9T 2012-06-19 2013-06-19 Compositions and methods for treating diseases DK3292875T3 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US13/527,350 US20120322861A1 (en) 2007-02-23 2012-06-19 Compositions and Methods for Treating Diseases
EP13807500.7A EP2861263B1 (en) 2012-06-19 2013-06-19 Compositions and methods for treating diabetes

Publications (1)

Publication Number Publication Date
DK3292875T3 true DK3292875T3 (en) 2020-08-03

Family

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Application Number Title Priority Date Filing Date
DK17184780.9T DK3292875T3 (en) 2012-06-19 2013-06-19 Compositions and methods for treating diseases

Country Status (24)

Country Link
US (2) US10912804B2 (da)
EP (4) EP2861263B1 (da)
JP (1) JP2015523998A (da)
KR (1) KR20150020250A (da)
CN (1) CN104619354A (da)
AU (1) AU2013277215A1 (da)
BR (1) BR112014031788A2 (da)
CA (1) CA2875523A1 (da)
CL (1) CL2014003464A1 (da)
CY (1) CY1123485T1 (da)
DK (1) DK3292875T3 (da)
ES (1) ES2813558T3 (da)
HK (1) HK1209633A1 (da)
HR (1) HRP20201224T1 (da)
HU (1) HUE051632T2 (da)
IL (1) IL236298A0 (da)
LT (1) LT3292875T (da)
MX (1) MX2014015985A (da)
PL (1) PL3292875T3 (da)
PT (1) PT3292875T (da)
RS (1) RS61055B1 (da)
RU (1) RU2015101276A (da)
SI (1) SI3292875T1 (da)
WO (1) WO2013192317A2 (da)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
CA2875523A1 (en) 2012-06-19 2013-12-27 University Of Florida Research Foundation, Inc. Compositions and methods for treating diseases
SG11201509419QA (en) * 2013-05-15 2015-12-30 Univ Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
MX2018005332A (es) * 2015-11-06 2018-11-09 Crispr Therapeutics Ag Materiales y metodos para tratamiento de la enfermedad de almacenamiento de glucogeno tipo 1a.
EP3293259A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3510149A1 (en) 2016-09-12 2019-07-17 Genethon Acid-alpha glucosidase variants and uses thereof
MA50546A (fr) 2017-06-07 2020-09-16 Regeneron Pharma Compositions et méthodes pour l'internalisation d'enzymes
BR112020005249A2 (pt) 2017-09-22 2020-09-24 The Trustees Of The University Of Pennsylvania terapia de gene para tratar mucopolissacaridose tipo ii
JP6567012B2 (ja) * 2017-09-28 2019-08-28 キヤノン株式会社 情報処理装置、情報処理方法、およびプログラム
JP7134444B2 (ja) * 2018-01-26 2022-09-12 国立大学法人徳島大学 テイ－サックス病及びザンドホッフ病治療用の新規アデノ随伴ウイルスビリオン
US20210162073A1 (en) * 2018-02-05 2021-06-03 Audentes Therapeutics, Inc. Transcription regulatory elements and uses thereof
WO2019154939A1 (en) * 2018-02-07 2019-08-15 Genethon Hybrid regulatory elements
CN114828858A (zh) * 2019-10-25 2022-07-29 奥登茨治疗公司 用于治疗糖原贮积症的组合物和方法

Family Cites Families (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US6451600B1 (en) 1989-12-22 2002-09-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
WO1995011640A1 (en) 1993-10-26 1995-05-04 Howmedica Inc. Prosthesis with integral proximal spacer
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
EP1390490B1 (en) * 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
EP3339430A1 (en) 2001-12-17 2018-06-27 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses thereof
JP2005518807A (ja) 2002-03-04 2005-06-30 トランジェーヌ、ソシエテ、アノニム 筋細胞中の目的とする遺伝子の発現の持続のための発現カセット
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
WO2003092598A2 (en) * 2002-04-30 2003-11-13 University Of Florida Treatment for pompe disease
AU2003302724A1 (en) * 2002-08-13 2004-07-09 Mcivor, Scott, R. Methods of using vectors to treat metabolic disorders
ATE521701T1 (de) 2003-01-22 2011-09-15 Univ Duke Verbesserte konstrukte zur expression lysosomaler polypeptide
EP1668110A2 (en) * 2003-08-21 2006-06-14 Q Therapeutics, Inc. Genetically modified somatic cells for sustained secretion of lysosomal proenzymes deficient in lysosomal storage disorders
WO2005049850A2 (en) * 2003-11-14 2005-06-02 University Of Washington Compositions and methods for systemic nucleic acid sequence delivery
EP1708756A2 (en) 2003-12-12 2006-10-11 Wisconsin Alumni Research Foundation Treatment of mammals by sirna delivery into mammalian nerve cells
WO2005118611A2 (en) * 2004-01-23 2005-12-15 The Trustees Of The University Of Pennsylvania Microutrophin and uses thereof
JP4914224B2 (ja) * 2004-02-10 2012-04-11 バイオマリン ファーマシューティカル インコーポレイテッド 酸性αグルコシダーゼおよびそのフラグメント
US20060078542A1 (en) 2004-02-10 2006-04-13 Mah Cathryn S Gel-based delivery of recombinant adeno-associated virus vectors
EP1812556A4 (en) * 2004-10-05 2011-02-02 Univ Georgia NEURONAL PRECURSORS OF FEEDER-FREE HUMAN EMBRYONIC STEM CELL CULTURE
CA2591544A1 (en) 2004-12-15 2006-06-22 The University Of North Carolina At Chapel Hill Chimeric vectors
CA2604052C (en) 2005-04-06 2014-07-08 Adamas Pharmaceuticals, Inc. Methods and compositions for the treatment of cns-related conditions
EP2186823A1 (en) 2005-11-14 2010-05-19 Merial Limited Gene therapy for renal failure
US7867484B2 (en) * 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
LT1988823T (lt) 2006-02-09 2018-12-10 Genzyme Corporation Lėtas intraventrikuliarinis pateikimas
US20100305068A1 (en) * 2006-11-09 2010-12-02 Foldrx Pharmaceuticals, Inc. Compounds and methods for modulating protein trafficking
CN101636200A (zh) * 2006-11-13 2010-01-27 齐斯特治疗公司 用于治疗庞贝氏症的方法
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
CA2680063A1 (en) 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions and methods for treating glycogen storage diseases
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
JP2012526997A (ja) * 2009-05-14 2012-11-01 オートミール バイオテクノロジーズ グループ， エル．エル．シー． 自発的に生じる疾患のためのプラットフォーム技術
BRPI1010024A2 (pt) * 2009-06-05 2019-09-24 Link Medicine Corp derivados de aminopirrolidinona e uso dos mesmos
WO2011123803A1 (en) 2010-04-02 2011-10-06 Shire Human Genetic Therapies, Inc. Systems and methods for managing treatment of an orphan disease
KR20190060881A (ko) 2010-04-23 2019-06-03 유니버시티 오브 플로리다 리서치 파운데이션, 인크. 레베르 선천성 흑내장-1(lca1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법
US8679478B2 (en) * 2010-10-04 2014-03-25 Duke University Methods of lysosomal storage disease therapy
US20120141420A1 (en) * 2010-11-30 2012-06-07 Sygnis Bioscience Gmbh & Co. Kg Targeted delivery of g-csf for the treatment of amyotrophic lateral sclerosis
EP2852388A4 (en) 2012-05-23 2016-01-13 Univ Johns Hopkins COMPOUNDS AND METHOD FOR USE THEREOF FOR THE TREATMENT OF NEURODEEGENERATIVE DISEASES
CA2875523A1 (en) 2012-06-19 2013-12-27 University Of Florida Research Foundation, Inc. Compositions and methods for treating diseases

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US10912804B2 (en) 2021-02-09
CY1123485T1 (el) 2022-03-24
SI3292875T1 (sl) 2020-08-31
CN104619354A (zh) 2015-05-13
EP2861263A4 (en) 2016-04-13
IL236298A0 (en) 2015-02-26
AU2013277215A1 (en) 2015-02-05
CA2875523A1 (en) 2013-12-27
EP4124346A3 (en) 2023-04-19
JP2015523998A (ja) 2015-08-20
HUE051632T2 (hu) 2021-03-29
MX2014015985A (es) 2015-07-21
WO2013192317A2 (en) 2013-12-27
PL3292875T3 (pl) 2020-11-16
EP2861263A2 (en) 2015-04-22
RU2015101276A (ru) 2016-08-10
RS61055B1 (sr) 2020-12-31
KR20150020250A (ko) 2015-02-25
LT3292875T (lt) 2020-08-25
EP3292875B1 (en) 2020-05-27
BR112014031788A2 (pt) 2017-06-27
EP4124346A2 (en) 2023-02-01
US20210145906A1 (en) 2021-05-20
EP2861263B1 (en) 2017-12-06
PT3292875T (pt) 2020-08-31
HRP20201224T1 (hr) 2020-11-13
EP3818991A3 (en) 2021-07-14
ES2813558T3 (es) 2021-03-24
EP3818991A2 (en) 2021-05-12
EP3292875A1 (en) 2018-03-14
US20170028002A1 (en) 2017-02-02
HK1209633A1 (en) 2016-04-08
CL2014003464A1 (es) 2015-06-12

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