SG11201804994QA - Adeno-associated viral vectors for treating mucolipidosis type ii - Google Patents

Adeno-associated viral vectors for treating mucolipidosis type ii

Info

Publication number
SG11201804994QA
SG11201804994QA SG11201804994QA SG11201804994QA SG11201804994QA SG 11201804994Q A SG11201804994Q A SG 11201804994QA SG 11201804994Q A SG11201804994Q A SG 11201804994QA SG 11201804994Q A SG11201804994Q A SG 11201804994QA SG 11201804994Q A SG11201804994Q A SG 11201804994QA
Authority
SG
Singapore
Prior art keywords
international
mucolipidosis type
vectors
iii
pct
Prior art date
Application number
SG11201804994QA
Inventor
Nelson Yew
Dong-Kyu Jin
Original Assignee
Genzyme Corp
Samsung Life Public Welfare Foundation
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 Genzyme Corp, Samsung Life Public Welfare Foundation filed Critical Genzyme Corp
Publication of SG11201804994QA publication Critical patent/SG11201804994QA/en

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
    • A01K67/027—New breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knockout animals
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/10—Laxatives
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/12—Antidiarrhoeals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/16—Central respiratory analeptics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1288—Transferases for other substituted phosphate groups (2.7.8)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/08—Transferases for other substituted phosphate groups (2.7.8)
    • C12Y207/08017—UDP-N-acetylglucosamine--lysosomal-enzyme N-acetylglucosaminephosphotransferase (2.7.8.17)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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
    • A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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
    • 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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • 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
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 1111111101111011101010111110101111101110101101101110110111001111101111011111 International Bureau 0.. .... .. (10) International Publication Number ...... ... (43) International Publication Date WO 2017/106313 Al 22 June 2017(22.06.2017) WIPO I PCT (51) International Patent Classification: AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, C12N 15/86 (2006.01) C12N 9/12 (2006.01) BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, A01K 67/027 (2006.01) A61K 48/00 (2006.01) DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, (21) International Application Number: KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, PCT/US2016/066611 MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (22) International Filing Date: NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 14 December 2016 (14.12.2016) RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, (25) Filing Language: English ZA, ZM, ZW. (26) Publication Language: English (84) Designated States (unless otherwise indicated, for every (30) Priority Data: kind of regional protection available): ARIPO (BW, GH, 62/267,502 15 December 2015 (15.12.2015) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, (71) Applicants: GENZYME CORPORATION [US/US]; TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, 500 Kendall Street, Cambridge, MA 02142 (US). SAM- DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, SUNG LIFE PUBLIC WELFARE FOUNDATION LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, [KR/KR]; 48, Itaewon-ro 55-gil, Yongsan-gu, Seoul (KR). SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, (72) Inventors: YEW, Nelson; c/o Sanofi, 55 Corporate Drive, GW, KM, ML, MR, NE, SN, TD, TG). Bridgewater, NJ 08807 (US). JIN, Dong-Kyu; c/o Sam- Declarations under Rule 4.17: — sung Life Public Welfare Foundation, 48, Itaewon-ro 55- — of inventorship (Rule 4.17(N)) gil, Yongsan-gu, Seoul (KR). — Published: Agents: DONAHUE, Brian, A. et al.; Morrison & Foer- (74) _ ster LLP, 755 Page Mill Road, Palo Alto, CA 94304-1018 — with international search report (Art. 21(3)) = (U S) . — with sequence listing part of description (Rule 5.2(a)) = Designated States (unless otherwise indicated, for every = (81) kind of national protection available): AE, AG, AL, AM, = = = = = = = = = = = = 1-1 M 11 M 0 1-1 (54) Title: ADENO-ASSOCIATED VIRAL VECTORS FOR TREATING MUCOLIPIDOSIS TYPE II IN (57) : Provided herein are recombinant adeno-associated virus (rAAV) vectors comprising nucleic acid encoding N-acetyl- 1-1 c:::: ) glucosamine-1 -phosphate transferase, alpha and beta subunits (GNPTAB) and at least one AAV inverted terminal repeat (ITR). In ei some embodiments, the rAAV vectors may be included in a rAAV particle, which may be contained in pharmaceutical compositions O and kits. These vectors, particles, compositions, and kits may find use, inter alia, in methods and uses related to treating mucol - ipidosis type II (ML II) or mucolipidosis type III (ML III) in a mammal, or related to increasing body size, bone mineral content, and/or bone mineral density in a mammal with mucolipidosis type II (ML II) or mucolipidosis type III (ML III).
SG11201804994QA 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii SG11201804994QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267502P 2015-12-15 2015-12-15
PCT/US2016/066611 WO2017106313A1 (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii

Publications (1)

Publication Number Publication Date
SG11201804994QA true SG11201804994QA (en) 2018-07-30

Family

ID=57737987

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11201804994QA SG11201804994QA (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii
SG10201912935WA SG10201912935WA (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG10201912935WA SG10201912935WA (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii

Country Status (13)

Country Link
US (1) US20200261598A1 (en)
EP (1) EP3390644A1 (en)
JP (2) JP7347932B2 (en)
KR (1) KR20180098294A (en)
CN (1) CN108603199A (en)
AU (2) AU2016372035B2 (en)
BR (1) BR112018012015A2 (en)
CA (1) CA3008268A1 (en)
IL (1) IL259964B (en)
MX (1) MX2018007330A (en)
RU (1) RU2742612C2 (en)
SG (2) SG11201804994QA (en)
WO (1) WO2017106313A1 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020008835A2 (en) 2017-11-07 2020-10-20 The University Of North Carolina At Chapel Hill optimized aga genes and expression cassettes and their use
BR112021009733A2 (en) * 2018-11-21 2022-01-04 Stridebio Inc Recombinant virus vectors and nucleic acids for their production

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999055378A1 (en) * 1998-04-27 1999-11-04 Temple University - Of The Commonwealth System Of Higher Education Method of treating endotoxemia
US6537785B1 (en) * 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
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
SG168422A1 (en) 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
US20060073123A1 (en) * 2002-04-30 2006-04-06 Jie Mi Adenovirus vectors for immunotherapy
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
EP2277996B1 (en) 2003-05-21 2014-09-03 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
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
DK2158322T3 (en) * 2007-06-06 2017-08-28 Genzyme Corp GENTERAPY AGAINST LYSOSOMAL STORAGE DISEASES
EP2443233B1 (en) 2009-06-16 2016-06-01 Genzyme Corporation Improved methods for purification of recombinant aav vectors
KR102007044B1 (en) * 2010-06-25 2019-08-02 샤이어 휴먼 지네틱 테라피즈 인크. CNS delivery of therapeutic agents
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
MX353930B (en) 2011-02-17 2018-02-02 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer.
MX2015012454A (en) * 2013-03-15 2016-04-25 Philadelphia Children Hospital Vectors comprising stuffer/filler polynucleotide sequences and methods of use.
CA2927366A1 (en) * 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
CA2946593A1 (en) * 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy

Also Published As

Publication number Publication date
JP7347932B2 (en) 2023-09-20
MX2018007330A (en) 2019-03-14
AU2023204116A1 (en) 2023-07-13
IL259964B (en) 2022-09-01
IL259964A (en) 2018-07-31
AU2016372035A1 (en) 2018-08-02
BR112018012015A2 (en) 2018-12-04
JP2022046635A (en) 2022-03-23
JP2019500034A (en) 2019-01-10
RU2742612C2 (en) 2021-02-09
CA3008268A1 (en) 2017-06-22
WO2017106313A1 (en) 2017-06-22
SG10201912935WA (en) 2020-02-27
AU2016372035B2 (en) 2023-03-30
RU2018124448A3 (en) 2020-08-14
RU2018124448A (en) 2020-01-17
EP3390644A1 (en) 2018-10-24
KR20180098294A (en) 2018-09-03
CN108603199A (en) 2018-09-28
US20200261598A1 (en) 2020-08-20

Similar Documents

Publication Publication Date Title
SG11201901221YA (en) Methods for detecting aav
SG11201907298WA (en) Methods and compositions for gene transfer across the vasculature
SG11201906297QA (en) Nucleic acids encoding crispr-associated proteins and uses thereof
SG11201808990QA (en) Compositions for topical application of compounds
SG11201803701YA (en) Methods and compositions for gene editing in hematopoietic stem cells
SG11201900028VA (en) Crispr/cas9-based compositions and methods for treating cancer
SG11201805451TA (en) Immune effector cell therapies with enhanced efficacy
SG11201811432WA (en) Rna for cancer therapy
SG11201810171SA (en) Kras g12c inhibitors
SG11201804070XA (en) Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
SG11201903825SA (en) Engineered artificial antigen presenting cells for tumor infiltrating lymphocyte expansion
SG11201903514VA (en) Novel cd47 monoclonal antibodies and uses thereof
SG11201803983UA (en) Transformable tagging compositions, methods, and processes incorporating same
SG11201805645QA (en) Lsd1 inhibitors
SG11201811600PA (en) Pseudotyped oncolytic viral delivery of therapeutic polypeptides
SG11201808196UA (en) Neoantigens and methods of their use
SG11201809700YA (en) Gdf15 fusion proteins and uses thereof
SG11201909432SA (en) Compounds for increasing genome editing efficiency
SG11202000145XA (en) Methods and systems for conditionally regulating gene expression
SG11201808422QA (en) Gene therapy for treating hemophilia a
SG11201906392WA (en) Nucleoside-modified rna for inducing an immune response against zika virus
SG11201907846VA (en) Therapeutic rna
SG11201903450YA (en) Methods for treating muscle wasting and bone disease using novel hybrid actriib ligand trap proteins
SG11201904535VA (en) Prevention and treatment of bone and cartilage damage or disease
SG11201408679XA (en) Methods for treating tyrosine-kinase-inhibitor-resistant malignancies in patients with genetic polymorphisms or ahi1 dysregulations or mutations employing dianhydrogalactitol, diacetyldianhydrogalactitol, dibromodulcitol, or analogs or derivatives thereof