AU2018335752B2 - Gene therapy for treating Mucopolysaccharidosis type ii - Google Patents

Gene therapy for treating Mucopolysaccharidosis type ii Download PDF

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Publication number
AU2018335752B2
AU2018335752B2 AU2018335752A AU2018335752A AU2018335752B2 AU 2018335752 B2 AU2018335752 B2 AU 2018335752B2 AU 2018335752 A AU2018335752 A AU 2018335752A AU 2018335752 A AU2018335752 A AU 2018335752A AU 2018335752 B2 AU2018335752 B2 AU 2018335752B2
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raav
suspension
vector
mps
hids
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AU2018335752A1 (en
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Christian HINDERER
James M. Wilson
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
    • 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
    • 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)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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
    • A61K9/10—Dispersions; Emulsions
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
AU2018335752A 2017-09-22 2018-09-21 Gene therapy for treating Mucopolysaccharidosis type ii Active AU2018335752B2 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762561769P 2017-09-22 2017-09-22
US201762562179P 2017-09-22 2017-09-22
US62/561,769 2017-09-22
US62/562,179 2017-09-22
US201762573841P 2017-10-18 2017-10-18
US62/573,841 2017-10-18
PCT/US2018/052129 WO2019060662A1 (en) 2017-09-22 2018-09-21 GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II

Publications (2)

Publication Number Publication Date
AU2018335752A1 AU2018335752A1 (en) 2020-03-12
AU2018335752B2 true AU2018335752B2 (en) 2025-04-10

Family

ID=63915352

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2018335752A Active AU2018335752B2 (en) 2017-09-22 2018-09-21 Gene therapy for treating Mucopolysaccharidosis type ii

Country Status (11)

Country Link
US (3) US11819539B2 (https=)
EP (1) EP3684938A1 (https=)
JP (2) JP7449223B2 (https=)
KR (2) KR20250161673A (https=)
AU (1) AU2018335752B2 (https=)
BR (1) BR112020005249A2 (https=)
CA (1) CA3076036A1 (https=)
IL (1) IL273427B2 (https=)
SG (1) SG11202002457RA (https=)
TW (1) TWI835747B (https=)
WO (1) WO2019060662A1 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL305449B2 (en) * 2016-04-15 2026-01-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous type ii diabetes
TWI835747B (zh) * 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
JP7684296B2 (ja) * 2019-11-22 2025-05-27 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法
MX2022007135A (es) 2019-12-10 2022-09-19 Takeda Pharmaceuticals Co Vectores de virus adenoasociados para el tratamiento del síndrome de hunter.
EP4100419A4 (en) * 2020-02-07 2024-05-29 Denali Therapeutics Inc. METHODS OF TREATING HUNTER SYNDROME
WO2021207077A1 (en) * 2020-04-06 2021-10-14 Homology Medicines, Inc. Adeno-associated virus compositions for ids gene transfer and methods of use thereof
CN114480454B (zh) * 2020-10-27 2024-03-29 华东理工大学 用于治疗粘多糖贮积症ⅱ型的腺相关病毒载体、药物及其应用
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
BR112023016075A2 (pt) 2021-02-10 2023-11-21 Regenxbio Inc Método para tratar hepatoesplenomegalia, método de distribuição de um raav que codifica hids, método para tratar um sujeito humano, método de tratamento de um sujeito humano, método para determinar a eficácia do tratamento, método para identificar um sujeito como tendo mps ii
US20240366789A1 (en) * 2021-04-01 2024-11-07 The University Of North Carolina At Chapel Hill Aav-ids vectors for treatment of mucopolysaccharidosis ii
WO2022221421A2 (en) * 2021-04-13 2022-10-20 Capsida, Inc. Aav compositions with high brain expression for treating mucopolysaccharidosis ii
CN115820740A (zh) * 2021-09-16 2023-03-21 北京锦篮基因科技有限公司 用于治疗ⅱ型粘多糖贮积症的重组腺相关病毒载体及其应用
WO2025075963A1 (en) * 2023-10-02 2025-04-10 Regenxbio Inc. Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016187017A1 (en) * 2015-05-15 2016-11-24 Mcivor R Scott Adeno-associated for therapeutic delivery to central nervous system
WO2017181113A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
WO2018093925A1 (en) * 2016-11-15 2018-05-24 Laoharawee Kanut Method for improving neurological function in mpsi and mpsii and other neurological disorders

Family Cites Families (131)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6407061B1 (en) 1989-12-05 2002-06-18 Chiron Corporation Method for administering insulin-like growth factor to the brain
WO1991007947A1 (en) 1989-12-05 1991-06-13 Ramsey Foundation Neurologic agents for nasal administration to the brain
US5624898A (en) 1989-12-05 1997-04-29 Ramsey Foundation Method for administering neurologic agents to the brain
AU649897B2 (en) 1991-11-14 1994-06-02 Women's And Children's Hospital Synthetic alpha-L-iduronidase and genetic sequences encoding same
FR2716893B1 (fr) 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
US6190659B1 (en) 1996-09-17 2001-02-20 The Rockefeller University Bacterial plasmin binding protein and methods of use thereof
AU8672198A (en) 1997-07-31 1999-02-22 Chiron Corporation Method enabling readministration of aav vector via immunosuppression of host
DE69929600T2 (de) 1998-05-27 2006-09-07 Avigen Inc., Alameda Konvektion-erhöhte verabreichung aadc-kodierende aav vektoren
US7273618B2 (en) 1998-12-09 2007-09-25 Chiron Corporation Method for administering agents to the central nervous system
ES2252993T3 (es) 1998-12-09 2006-05-16 Chiron Corporation Administracion de agentes neurotroficos al sistema nervioso central.
AU2173400A (en) 1998-12-09 2000-06-26 Chiron Corporation Method for administering agents to the central nervous system
PT1916258E (pt) 1999-08-09 2014-07-29 Genzyme Corp Aumento da expressão de uma sequência nucleotídica heteróloga de cadeia simples a partir de vectores virais recombinantes por concepção da sequência de maneira que esta forme pares de bases intracadeia
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
US6585971B1 (en) 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
US6569661B1 (en) 1999-11-12 2003-05-27 Biomarin Pharmaceutical Inc. Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
AU1775901A (en) 1999-11-17 2001-05-30 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
EP1237567B1 (en) 1999-12-09 2005-08-03 Chiron Corporation Method for administering a cytokine to the central nervous system and the lymphatic system
US7084126B1 (en) 2000-05-01 2006-08-01 Healthpartners Research Foundation Methods and compositions for enhancing cellular function through protection of tissue components
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
AU2001267337A1 (en) 2000-06-30 2002-01-14 Maxygen Aps Peptide extended glycosylated polypeptides
AU7194101A (en) 2000-07-18 2002-01-30 Univ Duke Treatment of glycogen storage disease type ii
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
EP1370282A2 (en) 2000-10-13 2003-12-17 Chiron Corporation Method for treating ischemic events affecting the central nervous system
US20020169102A1 (en) 2001-04-03 2002-11-14 Frey William H. Intranasal delivery of agents for regulating development of implanted cells in the CNS
WO2002086105A1 (en) 2001-04-20 2002-10-31 Chiron Corporation Delivery of polynucleotide agents to the central nervous sysstem
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
EP1487474A4 (en) 2002-02-25 2006-11-29 Chiron Corp INTRANASAL ADMINISTRATION OF MC4-R AGONISTS
US7485314B2 (en) 2002-05-06 2009-02-03 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Induction of antigen specific immunologic tolerance
AU2003302724A1 (en) 2002-08-13 2004-07-09 Mcivor, Scott, R. Methods of using vectors to treat metabolic disorders
AU2003272728A1 (en) 2002-09-27 2004-04-19 Children's Medical Center Corporation Methods and compositions for treatment of neurological disorder
US8512710B2 (en) 2002-11-22 2013-08-20 Ansun Biopharma, Inc. Class of therapeutic protein based molecules
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
EP1618201A1 (en) 2003-05-01 2006-01-25 University of Florida Research Foundation, Inc. Vp2 - modified raav vectors and uses thereof
US20050026823A1 (en) 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
US7442372B2 (en) 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
WO2005062881A2 (en) 2003-12-24 2005-07-14 Transgenrx, Inc. Gene therapy using transposon-based vectors
US7618615B2 (en) 2004-08-13 2009-11-17 Healthpartners Research Foundation Methods for providing neuroprotection for the animal central nervous system against neurodegeneration caused by ischemia
US7776312B2 (en) 2004-08-13 2010-08-17 Healthpartners Research Foundation Method of treating Alzheimer's disease comprising administering deferoxamine (DFO) to the upper one-third of the nasal cavity
US20090136505A1 (en) 2005-02-23 2009-05-28 Johanna Bentz Intranasal Administration of Active Agents to the Central Nervous System
NZ581205A (en) 2005-02-23 2011-06-30 Alza Corp Intranasal administration of active agents to the central nervous system
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
CA2620202C (en) 2005-08-26 2016-10-04 The Board Of Trustees Of The Leland Stanford Junior University Therapy procedure for drug delivery for trigeminal pain
JP5706602B2 (ja) 2006-02-08 2015-04-22 ジェンザイム・コーポレーション ニーマンピック病ａ型に対する遺伝子治療
DK1988823T3 (en) 2006-02-09 2018-12-03 Genzyme Corp SLOW INTRAVENTRICULAR ADMINISTRATION
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
WO2007127163A2 (en) 2006-04-24 2007-11-08 Geron Corporation Cns-tumor treatment method and composition
EP1915986A1 (en) 2006-10-23 2008-04-30 BIOPHARM GESELLSCHAFT ZUR BIOTECHNOLOGISCHEN ENTWICKLUNG VON PHARMAKA mbH Lipid growth factor formulations
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
EP2132309A4 (en) 2007-02-23 2011-01-05 Univ Florida COMPOSITIONS AND METHODS OF TREATING GLYCOLOGICAL MEMORY DISEASES
PL3252161T3 (pl) 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych
EP2152240A4 (en) 2007-06-08 2012-05-09 Healthpartners Res Foundation PHARMACEUTICAL COMPOSITIONS AND METHOD FOR REINFORCED TARGETING OF THERAPEUTIC COMPOUNDS TO THE CENTRAL NERVOUS SYSTEM
US8283160B2 (en) 2007-09-11 2012-10-09 Frey Ii William H Methods, pharmaceutical compositions and articles of manufacture for administering therapeutic cells to the animal central nervous system
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009075815A1 (en) 2007-12-07 2009-06-18 Duke University Immunomodulating gene therapy
DK2242840T3 (da) 2008-01-29 2019-10-21 Applied Genetic Tech Corporation Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension
WO2009120978A2 (en) 2008-03-27 2009-10-01 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of bdnf and compositions therfor
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
WO2010071832A1 (en) 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
US7989502B2 (en) 2009-02-06 2011-08-02 Sri International Intranasal delivery of modafinil
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
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US20110070241A1 (en) 2009-06-30 2011-03-24 Duke University Methods for modulating immune responses to aav gene therapy vectors
US8622993B2 (en) 2009-12-18 2014-01-07 Healthpartners Research Foundation Device and method for delivering therapeutic substances to the maxillary sinus of a patient
CN102791130B (zh) * 2010-03-03 2015-02-25 泰华制药工业有限公司 使用拉喹莫德治疗狼疮肾炎
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
NZ605873A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of arylsulfatase a
NZ605874A (en) 2010-06-25 2015-02-27 Shire Human Genetic Therapies Methods and compositions for cns delivery of heparan n-sulfatase
CN103179980B (zh) 2010-06-25 2016-09-28 夏尔人类遗传性治疗公司 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物
RS62620B1 (sr) 2010-06-25 2021-12-31 Shire Human Genetic Therapies Metode i kompozicije za isporuku cns akrilsulfataze a
SMT201600385T1 (it) 2010-06-25 2017-03-08 Shire Human Genetic Therapies Trasporto di agenti terapeutici all’snc
WO2012101671A1 (en) 2011-01-25 2012-08-02 Jcr Pharmaceuticals Co., Ltd. Method for production of human recombinant iduronate 2-sulfatase
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
DK2675902T3 (da) 2011-02-17 2019-06-03 Univ Pennsylvania Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel
ES2745470T3 (es) 2011-03-31 2020-03-02 Univ Iowa Res Found Partícula de AAV2 que comprende una proteína de la cápside de AAV2 y un vector que comprende un ácido nucleico que codifica una tripeptidilo peptidasa 1 (TPP1) para el tratamiento de lipofuscinosis ceroide infantil tardía (LINCL) en un mamífero no roedor mediante inyección intraventricular o administración icv
ES2702496T3 (es) 2011-04-21 2019-03-01 Nationwide Childrens Hospital Inc Productos de virus recombinante y procedimientos para la inhibición de la expresión de la miotilina
US10196636B2 (en) 2011-04-21 2019-02-05 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of myotilin
US8609088B2 (en) 2011-05-10 2013-12-17 Regents Of The University Of Minnesota Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
EP2709670A4 (en) 2011-05-18 2015-01-21 Childrens Hosp Medical Center TARGETED TRANSFER OF PROTEINS OVER THE BLOOD BRAIN BARRIER
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
US9387236B2 (en) 2011-06-10 2016-07-12 Prothera Inc. Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases
AU2012286994B2 (en) 2011-07-25 2017-08-10 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of DUX4
JP6329483B2 (ja) 2011-10-12 2018-05-23 シナジェバ・バイオファーマ・コーポレイションＳｙｎａｇｅｖａ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐ． 組換えヒトｎａｇｌｕタンパク質およびその利用
US9821149B2 (en) 2011-10-14 2017-11-21 Healthpartners Research & Education Methods for using ultrasound for enhancing efficiency and targeting of intranasal administration of therapeutic compounds to the central nervous system
JP6188703B2 (ja) 2011-10-27 2017-08-30 サンガモ セラピューティクス， インコーポレイテッド Ｈｐｒｔ遺伝子座を修飾するための方法および組成物
AU2012346448B2 (en) 2011-12-02 2017-09-14 Armagen, Inc. Methods and compositions for increasing arylsulfatase A activity in the CNS
CA2863964C (en) 2012-02-07 2021-10-26 Global Bio Therapeutics Usa, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
CN104203120A (zh) 2012-03-23 2014-12-10 泰尔茂株式会社 穿刺器具以及穿刺装置
RS61055B1 (sr) 2012-06-19 2020-12-31 Univ Florida Kompozicije i postupci za lečenje bolesti
WO2013190059A1 (en) 2012-06-21 2013-12-27 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
AU2013296425B2 (en) 2012-08-01 2018-06-07 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
US12024568B2 (en) 2012-09-13 2024-07-02 Cornell University Treatment of brain cancers using central nervous system mediated gene transfer of monoclonal antibodies
HUE039334T2 (hu) 2012-11-27 2018-12-28 Biomarin Pharm Inc Célzott terápiás lizoszómális enzim fúziós fehérjék és azok alkalmazásai
WO2014125647A1 (ja) 2013-02-18 2014-08-21 富士通オプティカルコンポーネンツ株式会社 光受信装置
SI2984166T1 (sl) 2013-03-15 2020-09-30 The Trustees Of The University Of Pennsylvania Sestavki za zdravljenje MPSI
KR102413498B1 (ko) 2013-04-20 2022-06-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 엑손 2-표적 U7snRNA 폴리뉴클레오티드 작제물의 재조합형 아데노 부속 바이러스 전달
MX380973B (es) 2013-05-01 2025-03-12 Genzyme Corp Composiciones y metodos para tratar la atrofia muscular espinal.
CN105377039A (zh) 2013-05-15 2016-03-02 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
EP3024497B1 (en) 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2015021443A1 (en) 2013-08-08 2015-02-12 Global Bio Therapeutics Usa, Inc. Clamp device for minimally invasive procedures and uses thereof
LT3702466T (lt) 2013-08-27 2023-02-27 Research Institute At Nationwide Children's Hospital Produktai ir būdai, skirti šoninės amiotrofinės sklerozės gydymui
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
CN120174012A (zh) 2013-10-24 2025-06-20 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
US20170049887A1 (en) 2014-04-25 2017-02-23 University Of Florida Research Foundation, Inc. Methods of permitting a subject to receive multiple doses of recombinant adeno-associated virus
WO2015196150A2 (en) 2014-06-20 2015-12-23 Wisconsin Alumni Research Foundation (Warf) Mutations that confer genetic stability to additional genes in influenza viruses
BR112017005892A2 (pt) 2014-09-24 2017-12-12 Hope City variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
CN107249646B (zh) 2014-12-16 2021-06-29 内布拉斯加大学董事会 用于青少年巴滕病的基因疗法
US10538589B2 (en) 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
EP3101125A1 (en) 2015-06-05 2016-12-07 Laboratorios Del Dr. Esteve, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
EP3322297B1 (en) 2015-07-13 2024-12-04 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
HK1257801A1 (zh) * 2015-10-28 2019-11-01 宾夕法尼亚州大学信托人 用於基因治疗的腺伴随病毒载体的鞘内给药
CA3010738A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
EP3411488A1 (en) * 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
PL3411484T3 (pl) 2016-02-05 2024-02-19 Emory University Wstrzykiwanie jednoniciowego lub samokomplementarnego wirusa adenosatelitarnego 9 do płynu mózgowego
US10561894B2 (en) 2016-03-18 2020-02-18 Icon Health & Fitness, Inc. Treadmill with removable supports
CA3049915A1 (en) 2017-01-31 2018-08-09 Stephen YOO Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
KR102719222B1 (ko) 2017-05-11 2024-10-21 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 신경 세로이드 지질갈색소증에 대한 유전자 요법
MA50016A (fr) 2017-07-06 2020-07-08 Univ Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
IL280198B2 (en) 2018-07-18 2026-01-01 Regenxbio Inc Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus)
US20240084917A1 (en) 2019-10-07 2024-03-14 Wartsila Services Switzerland Ltd. Step lift control of hydraulically actuated popped valves
WO2021150570A1 (en) 2020-01-22 2021-07-29 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016187017A1 (en) * 2015-05-15 2016-11-24 Mcivor R Scott Adeno-associated for therapeutic delivery to central nervous system
WO2017181113A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
WO2018093925A1 (en) * 2016-11-15 2018-05-24 Laoharawee Kanut Method for improving neurological function in mpsi and mpsii and other neurological disorders

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