SG11201910015SA - Gene therapy for neuronal ceroid lipofuscinoses - Google Patents

Gene therapy for neuronal ceroid lipofuscinoses

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Publication number
SG11201910015SA
SG11201910015SA SG11201910015SA SG11201910015SA SG 11201910015S A SG11201910015S A SG 11201910015SA SG 11201910015S A SG11201910015S A SG 11201910015SA SG 11201910015S A SG11201910015S A SG 11201910015SA
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SG
Singapore
Prior art keywords
international
street
philadelphia
pct
aav
Prior art date
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Inventor
James Wilson
Nathan Katz
Christian Hinderer
Juliette Hordeaux
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Univ Pennsylvania
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Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of SG11201910015SA publication Critical patent/SG11201910015SA/en

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    • 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)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485—Exopeptidases (3.4.11-3.4.19)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
    • C12N2710/16141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16143—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
    • C12N2750/14141—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
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 15 November 2018 (15.11.2018) WIPO I PCT ~~ll~~~~~~~~ 011101VIIIVIIIVIII olo oi olo Imo ow (10) International Publication Number WO 2018/209205 Al (51) International Patent Classification: A61K 31/436 (2006.01) A61K 38/46 (2006.01) A61K 38/13 (2006.01) Published: — with international search report (Art. 21(3)) with sequence listing part of description (Rule 5.2(a)) (21) International Application Number: PCT/US2018/032278 (22) International Filing Date: 11 May 2018 (11.05.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/504,817 11 May 2017 (11.05.2017) US 62/599,816 18 December 2017 (18.12.2017) US 62/652,006 03 April 2018 (03.04.2018) US (71) Applicant: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA [US/US]; 3160 Chestnut Street, Suite 200, Philadelphia, PA 19104 (US). (72) Inventors: WILSON, James, M.; 1831 Delancey Street, Philadelphia, PA 19103 (US). KATZ, Nathan; 1111 Hope Street, Unit 3, Stamford, CT 06907 (US). HINDERER, Christian; 212 Brown Street, Apt. 8A, Philadelphia, PA 19123 (US). HORDEAUX, Juliette; 712 S. Mildred Street, Philadelphia, PA 19147 (US). (74) Agent: SCHALLER, Colleen, M. et al.; Howson & How- son LLP, 350 Sentry Parkway, Building 620, Suite 210, Blue Bell, PA 19422 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, 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, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 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, ZA, ZM, ZW. = (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, 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, LV, 1-1 MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, 11 KM, ML, MR, NE, SN, TD, TG). O ! (54) Title: GENE THERAPY FOR NEURONAL CEROID LIPOFUSCINOSES (57) : Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant O adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome \" comprising (a) an AAV 5' inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human 0 TPP1; (d) an AAV 3' ITR.
SG11201910015S 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses SG11201910015SA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762504817P 2017-05-11 2017-05-11
US201762599816P 2017-12-18 2017-12-18
US201862652006P 2018-04-03 2018-04-03
PCT/US2018/032278 WO2018209205A1 (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses

Publications (1)

Publication Number Publication Date
SG11201910015SA true SG11201910015SA (en) 2019-11-28

Family

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SG11201910015S SG11201910015SA (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses
SG10201912401QA SG10201912401QA (en) 2017-05-11 2018-05-11 Gene therapy for neuronal ceroid lipofuscinoses

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Country Status (10)

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US (2) US11591614B2 (en)
EP (1) EP3621612A4 (en)
JP (2) JP7273730B2 (en)
KR (1) KR20200023280A (en)
AU (1) AU2018265531A1 (en)
BR (1) BR112019023303A2 (en)
CA (1) CA3061655A1 (en)
RU (1) RU2019139555A (en)
SG (2) SG11201910015SA (en)
WO (1) WO2018209205A1 (en)

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CA3076036A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
MX2021005517A (en) * 2018-11-14 2021-06-18 Regenxbio Inc Gene therapy for neuronal ceroid lipofuscinoses.
CN113993553A (en) * 2019-04-08 2022-01-28 费城儿童医院 Treatment of lysosomal storage diseases of the eye by administration of an AAV expressing TPP1
SG11202111195VA (en) * 2019-04-12 2021-11-29 Encoded Therapeutics Inc Compositions and methods for administration of therapeutics
BR112022022212A2 (en) 2020-05-12 2022-12-13 Univ Pennsylvania COMPOSITIONS FOR SPECIFIC DRG REDUCTION OF TRANSGENE EXPRESSION
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression

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Publication number Publication date
CA3061655A1 (en) 2018-11-15
RU2019139555A3 (en) 2021-10-12
US20230383312A1 (en) 2023-11-30
AU2018265531A1 (en) 2019-11-14
RU2019139555A (en) 2021-06-11
EP3621612A4 (en) 2021-06-16
EP3621612A1 (en) 2020-03-18
BR112019023303A2 (en) 2020-06-16
WO2018209205A9 (en) 2019-11-21
WO2018209205A1 (en) 2018-11-15
KR20200023280A (en) 2020-03-04
SG10201912401QA (en) 2020-02-27
JP2020519292A (en) 2020-07-02
JP2023099113A (en) 2023-07-11
US20200239908A1 (en) 2020-07-30
US11591614B2 (en) 2023-02-28
JP7273730B2 (en) 2023-05-15

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