SG11201808176TA - Gene therapy for treating mucopolysaccharidosis type ii - Google Patents

Gene therapy for treating mucopolysaccharidosis type ii

Info

Publication number
SG11201808176TA
SG11201808176TA SG11201808176TA SG11201808176TA SG11201808176TA SG 11201808176T A SG11201808176T A SG 11201808176TA SG 11201808176T A SG11201808176T A SG 11201808176TA SG 11201808176T A SG11201808176T A SG 11201808176TA SG 11201808176T A SG11201808176T A SG 11201808176TA
Authority
SG
Singapore
Prior art keywords
international
philadelphia
pct
street
treating
Prior art date
Application number
SG11201808176TA
Inventor
Christian Hinderer
James Wilson
Original Assignee
The Trustees Of The Univ Of Pennsyvania
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 The Trustees Of The Univ Of Pennsyvania filed Critical The Trustees Of The Univ Of Pennsyvania
Publication of SG11201808176TA publication Critical patent/SG11201808176TA/en

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
    • 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
    • A61K9/10—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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6827—Hybridisation assays for detection of mutation or polymorphism
    • 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)
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Dispersion Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization M1111101110101011111 HO 11111011101 11111 01010111 00111110 1111 III 11 International Bureau ... .... ..Yjd ...... ... (10) International Publication Number (43) International Publication Date WO 2017/181113 Al 19 October 2017(19.10.2017) WIPO I PCT (51) International Patent Classification: (81) Designated States (unless otherwise indicated, for every A61K 48/00 (2006.01) C12N 9/16 (2006.01) kind of national protection available): AE, AG, AL, AM, C12N 15/86 (2006.01) AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, (21) International Application Number: DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, PCT/US2017/027770 HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, (22) International Filing Date: KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, 14 April 2017 (14.04.2017) MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, (25) Filing Language: English RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, (26) Publication Language: English TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (30) Priority Data: 62/323,194 15 April 2016 (15.04.2016) US (84) Designated States (unless otherwise indicated, for every 62/330,938 3 May 2016 (03.05.2016) US kind of regional protection available): ARIPO (BW, GH, 62/337,163 16 May 2016 (16.05.2016) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 62/367,780 28 July 2016 (28.07.2016) US TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, 62/452,494 31 January 2017 (31.01.2017) US TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, (71) Applicant: THE TRUSTEES OF THE UNIVERSITY LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, OF PENNSYVANIA [US/US]; 3160 Chestnut Street, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, Suite 200, Philadelphia, PA 19104 (US). GW, KM, ML, MR, NE, SN, TD, TG). (72) Inventors: HINDERER, Christian; 1930 Chestnut Street, Published: Apt 7B, Philadelphia, PA 19103 (US). WILSON, James, — with international search report (Art. 21(3)) M.; 1831 Delancey Street, Philadelphia, PA 19103 (US). — before the expiration of the time limit for amending the Agents: KODROFF, Cathy, A. Howson Howson (74) et al.; & claims and to be republished in the event of receipt of LLP, 350 Sentry Parkway, Building 620, Suite 210, Blue amendments (Rule 48.2(h)) Bell, PA 19422 (US). — with sequence listing part of description (Rule 5.2(a)) 1-1 M 11 11 11 GC 11 IN 11 © (54) Title: GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE II ei 0 (57) : A suspension useful for AAV9-mediated intrathecal and/or systemic delivery of an expression cassette containing a h1DS gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating Hunter syndrome and the symptoms associated with Hunter syndrome.
SG11201808176TA 2016-04-15 2017-04-14 Gene therapy for treating mucopolysaccharidosis type ii SG11201808176TA (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201662323194P 2016-04-15 2016-04-15
US201662330938P 2016-05-03 2016-05-03
US201662337163P 2016-05-16 2016-05-16
US201662367780P 2016-07-28 2016-07-28
US201762452494P 2017-01-31 2017-01-31
PCT/US2017/027770 WO2017181113A1 (en) 2016-04-15 2017-04-14 Gene therapy for treating mucopolysaccharidosis type ii

Publications (1)

Publication Number Publication Date
SG11201808176TA true SG11201808176TA (en) 2018-10-30

Family

ID=60042827

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11201808176TA SG11201808176TA (en) 2016-04-15 2017-04-14 Gene therapy for treating mucopolysaccharidosis type ii
SG10201912761UA SG10201912761UA (en) 2016-04-15 2017-04-14 Gene therapy for treating mucopolysaccharidosis type ii

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG10201912761UA SG10201912761UA (en) 2016-04-15 2017-04-14 Gene therapy for treating mucopolysaccharidosis type ii

Country Status (11)

Country Link
US (2) US11253612B2 (en)
EP (1) EP3452104A1 (en)
JP (2) JP7171439B2 (en)
KR (2) KR20230125339A (en)
AU (1) AU2017250298A1 (en)
BR (1) BR112018071156A2 (en)
CA (1) CA3019427A1 (en)
IL (2) IL305449A (en)
MA (1) MA44874A (en)
SG (2) SG11201808176TA (en)
WO (1) WO2017181113A1 (en)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL307936A (en) 2017-04-14 2023-12-01 Regenxbio Inc Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells
BR112020005249A2 (en) * 2017-09-22 2020-09-24 The Trustees Of The University Of Pennsylvania gene therapy to treat mucopolysaccharidosis type ii
JP2021500857A (en) 2017-10-02 2021-01-14 デナリ セラピューティクス インコーポレイテッドＤｅｎａｌｉ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． Fusion protein containing enzymes for enzyme replacement therapy
CA3082136A1 (en) * 2017-11-08 2019-05-16 Avexis, Inc. Means and method for preparing viral vectors and uses of same
KR20220112283A (en) 2019-12-10 2022-08-10 다케다 야쿠힌 고교 가부시키가이샤 Adeno-associated viral vectors for the treatment of HUNTER disease
US20230088992A1 (en) 2020-01-29 2023-03-23 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells
AU2021252515A1 (en) * 2020-04-06 2022-10-27 Homology Medicines, Inc. Adeno-associated virus compositions for IDS gene transfer and methods of use thereof
US20230304034A1 (en) 2020-05-12 2023-09-28 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2022040530A2 (en) * 2020-08-21 2022-02-24 Capsida, Inc. Virus compositions for treating mucopolysaccharidosis ii
KR20230145386A (en) 2021-02-10 2023-10-17 리젠엑스바이오 인크. Treatment of mucopolysaccharidosis II using recombinant human iduronate-2-sulfatase (IDS)
WO2022212616A1 (en) * 2021-04-01 2022-10-06 The University Of North Carolina At Chapel Hill Aav-ids vectors for treatment of mucopolysaccharidosis ii
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023137233A2 (en) * 2022-01-17 2023-07-20 Danmarks Tekniske Universitet Compositions and methods for editing genomes

Family Cites Families (99)

* 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
AU6909091A (en) 1989-12-05 1991-06-26 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
FR2716893B1 (en) * 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Recombinant viruses, their preparation and their therapeutic use.
US6190659B1 (en) 1996-09-17 2001-02-20 The Rockefeller University Bacterial plasmin binding protein and methods of use thereof
CA2297490A1 (en) 1997-07-31 1999-02-11 Chiron Corporation Method enabling readministration of aav vector via immunosuppression of host
PT1080202E (en) 1998-05-27 2006-05-31 Avigen Inc DISTRIBUTION OF AAV VECTORS ENCODING AADC INTENSIFIED BY CONVECTION
DE69923998T2 (en) 1998-12-09 2006-04-27 Chiron Corp., Emeryville USE OF A NEUROLOGICAL AGENT FOR THE PRODUCTION OF A MEDICAMENT FOR THE TREATMENT OF DISEASES OF THE CENTRAL NERVOUS SYSTEM
US7273618B2 (en) 1998-12-09 2007-09-25 Chiron Corporation Method for administering agents to the central nervous system
ATE310501T1 (en) 1998-12-09 2005-12-15 Chiron Corp ADMINISTRATION OF NEUROTROPIC AGENTS INTO THE CENTRAL NERVOUS SYSTEM
ES2308989T3 (en) 1999-08-09 2008-12-16 Targeted Genetics Corporation INCREASE IN THE EXPRESSION OF A NUCLEOTIDIC SEQUENCE HETEROLOGY FROM RECOMBINANT VIRECT VECTORS CONTAINING A SEQUENCE FORMING INTRACATENARY BASE PAIRS.
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
WO2001036603A2 (en) 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
CA2393688A1 (en) 1999-12-09 2001-06-14 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
EP3449934B1 (en) 2000-07-18 2020-05-20 Duke University 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
EP1385937A4 (en) 2001-04-20 2005-11-09 Chiron Corp Delivery of polynucleotide agents to the central nervous sysstem
ATE520707T1 (en) 2001-11-13 2011-09-15 Univ Pennsylvania METHOD FOR THE DETECTION AND/OR IDENTIFICATION OF ADENO-ASSOCIATED VIRUS (AAV) SEQUENCES AND ISOLATION OF NEW SEQUENCES IDENTIFIED THEREFROM
EP2359869B1 (en) 2001-12-17 2018-12-26 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor
WO2003072056A2 (en) 2002-02-25 2003-09-04 Chiron Corporation 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
US8512710B2 (en) 2002-11-22 2013-08-20 Ansun Biopharma, Inc. Class of therapeutic protein based molecules
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
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
WO2005062881A2 (en) 2003-12-24 2005-07-14 Transgenrx, Inc. Gene therapy using transposon-based vectors
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
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
CN101184499B (en) 2005-02-23 2012-03-28 阿尔扎公司 Intranasal administration of active agents to the central nervous system
US20090136505A1 (en) 2005-02-23 2009-05-28 Johanna Bentz Intranasal Administration of Active Agents to the Central Nervous System
EP3409296A1 (en) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating device
JP2009506076A (en) 2005-08-26 2009-02-12 ザ・ボード・オブ・トラスティーズ・オブ・ザ・レランド・スタンフォード・ジュニア・ユニバーシティ Therapeutic procedures for drug delivery for trigeminal neuralgia
BRPI0707590A2 (en) 2006-02-08 2011-05-10 Genzyme Corp niemann-pick gene therapy for type a disease
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
US8153604B2 (en) 2006-04-24 2012-04-10 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
EP2132309A4 (en) 2007-02-23 2011-01-05 Univ Florida Compositions and methods for treating glycogen storage diseases
SI2158322T1 (en) 2007-06-06 2017-10-30 Genzyme Corporation Gene therapy for lysosomal storage diseases
JP2011504163A (en) 2007-06-08 2011-02-03 ヘルスパートナーズ リサーチ ファウンデーション Pharmaceutical compositions and methods for enhancing 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
US9265843B2 (en) 2008-03-27 2016-02-23 The Ohio State University Treatment of metabolic-related disorders using hypothalamic gene transfer of BDNF and compositions therefor
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides 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
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide 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
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
SG10201908848RA (en) 2010-03-29 2019-10-30 Univ Pennsylvania Pharmacologically induced transgene ablation system
CA3066596A1 (en) 2010-04-23 2011-10-27 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
PT3626258T (en) 2010-06-25 2021-10-19 Shire Human Genetic Therapies Methods and compositions for cns delivery of iduronate-2-sulfatase
WO2011163649A2 (en) 2010-06-25 2011-12-29 Shire Human Genetic Therapies, Inc Methods and compositions for cns delivery of iduronate-2-sulfatase
RU2626514C2 (en) 2010-06-25 2017-07-28 Шир Хьюман Дженетик Терапис, Инк. Medical agents delivery to the central nervous system
US9320711B2 (en) 2010-06-25 2016-04-26 Shire Human Genetic Therapies, Inc. Methods and compositions for CNS delivery of heparan N-sulfatase
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
PL2675902T3 (en) 2011-02-17 2019-08-30 The Trustees Of The University Of Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
CA2832151C (en) 2011-03-31 2021-06-15 University Of Iowa Research Foundation Methods and compositions for treating brain diseases
ES2702496T3 (en) 2011-04-21 2019-03-01 Nationwide Childrens Hospital Inc Recombinant virus products and procedures for the inhibition of miotilin expression
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
US10227387B2 (en) 2011-05-18 2019-03-12 Children's Hospital Medical Center Targeted delivery of proteins across 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
WO2013016352A1 (en) 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
CA2852027A1 (en) 2011-10-12 2013-04-18 Synageva Biopharma Corp. Recombinant human naglu protein and uses thereof
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
WO2013081706A1 (en) 2011-12-02 2013-06-06 Armagen Technologies, Inc. Methods and compositions for increasing arylsulfatase a activity in the cns
WO2013119880A1 (en) 2012-02-07 2013-08-15 Global Bio Therapeutics Usa, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
US20150182637A1 (en) 2012-06-21 2015-07-02 Association Institut De Myologie Widespread gene delivery of gene therapy vectors
PT2879719T (en) 2012-08-01 2018-10-08 Ohio State Innovation Foundation Intrathecal delivery of recombinant adeno-associated virus 9
PL2925776T3 (en) 2012-11-27 2018-11-30 Biomarin Pharmaceutical Inc. Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof
WO2014125647A1 (en) 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 Photoreceptor device
JP6591956B2 (en) 2013-03-15 2019-10-16 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Compositions and methods for treating MPS1
JP6461917B2 (en) 2013-04-20 2019-01-30 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 Delivery of exon 2 targeted U7 snRNA polynucleotide constructs by recombinant adeno-associated virus
WO2014178863A1 (en) 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
CA2912678C (en) * 2013-05-15 2023-10-10 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
ES2859605T3 (en) 2013-07-26 2021-10-04 Univ Iowa Res Found Procedures and compositions for treating diseases of the brain
PT3702466T (en) 2013-08-27 2023-01-24 Res Inst Nationwide Childrens Hospital Products and methods for treatment of amyotrophic lateral sclerosis
ES2739288T3 (en) 2013-09-13 2020-01-30 California Inst Of Techn Selective recovery
WO2015060722A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
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
ES2856090T3 (en) 2014-09-24 2021-09-27 Hope City Adeno-associated virus vector variants for high-efficiency genomic editing and their methods
EP3233131A1 (en) 2014-12-16 2017-10-25 Board of Regents of the University of Nebraska Gene therapy for juvenile batten disease
HUE057795T2 (en) 2015-05-15 2022-06-28 Regenxbio Inc Adeno-associated virus for therapeutic delivery to central nervous system
EP3101125A1 (en) 2015-06-05 2016-12-07 Laboratorios Del Dr. Esteve, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
LT3411484T (en) 2016-02-05 2023-11-27 Emory University Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
US10561894B2 (en) 2016-03-18 2020-02-18 Icon Health & Fitness, Inc. Treadmill with removable supports
CA3044089A1 (en) 2016-11-15 2018-05-24 Kelly M. PODETZ-PEDERSEN Method for improving neurological function in mpsi and mpsii and other neurological disorders

Also Published As

Publication number Publication date
SG10201912761UA (en) 2020-02-27
EP3452104A1 (en) 2019-03-13
US11253612B2 (en) 2022-02-22
WO2017181113A1 (en) 2017-10-19
IL262211B2 (en) 2024-01-01
BR112018071156A2 (en) 2019-03-12
JP2023011821A (en) 2023-01-24
IL262211B1 (en) 2023-09-01
CA3019427A1 (en) 2017-10-19
KR20230125339A (en) 2023-08-29
JP2019518002A (en) 2019-06-27
MA44874A (en) 2019-03-13
AU2017250298A1 (en) 2018-10-11
IL305449A (en) 2023-10-01
US20220125949A1 (en) 2022-04-28
JP7171439B2 (en) 2022-11-15
KR20190008237A (en) 2019-01-23
IL262211A (en) 2018-11-29
US20190070311A1 (en) 2019-03-07

Similar Documents

Publication Publication Date Title
SG11201808176TA (en) Gene therapy for treating mucopolysaccharidosis type ii
SG11201806270XA (en) Gene therapy for treating mucopolysaccharidosis type i
SG11201909949XA (en) Targeted immunotolerance
SG11201908719QA (en) Biomarkers and car t cell therapies with enhanced efficacy
SG11201908391XA (en) Methods for modulating an immune response
SG11201808422QA (en) Gene therapy for treating hemophilia a
SG11201907916TA (en) Compounds and compositions for intracellular delivery of therapeutic agents
SG11201808263VA (en) Smoking device and method for aerosol-generation
SG11201808812RA (en) Novel aav8 mutant capsids and compositions containing same
SG11201909868YA (en) Compositions and methods of treating huntington's disease
SG11201804375WA (en) Compositions and methods for internalizing enzymes
SG11201808799SA (en) Amine-substituted aryl or heteroaryl compounds as ehmt1 and ehmt2 inhibitors
SG11201804729RA (en) Methods and compositions for treating a serpinc1-associated disorder
SG11201810179RA (en) Novel crispr enzymes and systems
SG11201907474UA (en) Compositions useful in treatment of spinal muscular atrophy
SG11201803701YA (en) Methods and compositions for gene editing in hematopoietic stem cells
SG11201901531TA (en) Regulation of gene expression using engineered nucleases
SG11201804814YA (en) Materials and methods for delivering nucleic acids to cochlear and vestibular cells
SG11201809437TA (en) Intracellular delivery of biomolecules to induce tolerance
SG11201807025SA (en) Crispr/cas systems for c-1 fixing bacteria
SG11201807176XA (en) TGFß1-BINDING IMMUNOGLOBULINS AND USE THEREOF
SG11201803593QA (en) Engineered nucleic-acid targeting nucleic acids
SG11201901020RA (en) Anti-siglec-7 antibodies for the treatment of cancer
SG11201806583XA (en) Compositions containing tucaresol or its analogs
SG11201907023UA (en) Method of reducing neutropenia