SG11201909868YA - Compositions and methods of treating huntington's disease - Google Patents

Compositions and methods of treating huntington's disease

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Publication number
SG11201909868YA
SG11201909868YA SG11201909868YA SG11201909868YA SG 11201909868Y A SG11201909868Y A SG 11201909868YA SG 11201909868Y A SG11201909868Y A SG 11201909868YA SG 11201909868Y A SG11201909868Y A SG 11201909868YA
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SG
Singapore
Prior art keywords
cambridge
street
international
sidney
disease
Prior art date
Application number
Inventor
Dinah Sah
Fen Chen
Pengcheng Zhou
Xin Wang
Yanquan Shu
Jinzhao Hou
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Voyager Therapeutics Inc
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Publication date
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of SG11201909868YA publication Critical patent/SG11201909868YA/en

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
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    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • 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
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2320/00—Applications; Uses
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
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    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14123—Virus like particles [VLP]
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    • 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
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    • 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

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 08 November 2018 (08.11.2018) WIPO I PCT omit VIII °nolo III tom 1110 01110111110 mil (10) International Publication Number WO 2018/204803 Al (51) International Patent Classification: C12N 15/09 (2006.01) C12N 15/86 (2006.01) C12N 15/11 (2006.01) C12N 15/861 (2006.01) C12N 15/113 (2010.01) (21) International Application Number: PCT/US2018/031117 (22) International Filing Date: 04 May 2018 (04.05.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/501,786 05 May 2017 (05.05.2017) US 62/507,921 18 May 2017 (18.05.2017) US 62/520,086 15 June 2017 (15.06.2017) US (71) Applicant: VOYAGER THERAPEUTICS, INC. [US/US]; 75 Sidney Street, Cambridge, MA 02139 (US). (72) Inventors: SAH, Dinah, Wen-Yee; 75 Sidney Street, Cam- bridge, MA 02139 (US). CHEN, Fen; 75 Sidney Street, Cambridge, MA 02139 (US). ZHOU, Pengcheng; 75 Sid- ney Street, Cambridge, MA 02139 (US). WANG, Xin; 75 Sidney Street, Cambridge, MA 02139 (US). SHU, Yan- quan; 75 Sidney Street, Cambridge, MA 02139 (US). HOU, Jinzhao; 75 Sidney Street, Cambridge, MA 02139 (US). (74) Agent: WARD, Donna, T. et al.; DTWARD, PC, 142A Main Street, Groton, MA 01824 (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, 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, KM, ML, MR, NE, SN, TD, TG). Published: with international search report (Art. 21(3)) before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) with sequence listing part of description (Rule 5.2(a)) (54) Title: COMPOSITIONS AND METHODS OF TREATING HUNTINGTON'S DISEASE FIG. 1 O cc 100 120 130 110 120 1 O O (57) : The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
SG11201909868Y 2017-05-05 2018-05-04 Compositions and methods of treating huntington's disease SG11201909868YA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762501786P 2017-05-05 2017-05-05
US201762507921P 2017-05-18 2017-05-18
US201762520086P 2017-06-15 2017-06-15
PCT/US2018/031117 WO2018204803A1 (en) 2017-05-05 2018-05-04 Compositions and methods of treating huntington's disease

Publications (1)

Publication Number Publication Date
SG11201909868YA true SG11201909868YA (en) 2019-11-28

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SG11201909868Y SG11201909868YA (en) 2017-05-05 2018-05-04 Compositions and methods of treating huntington's disease

Country Status (9)

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US (1) US11752181B2 (en)
EP (1) EP3619308A4 (en)
JP (2) JP2020518259A (en)
CN (1) CN111108198A (en)
AU (1) AU2018261003A1 (en)
CA (1) CA3061368A1 (en)
MX (1) MX2019013172A (en)
SG (1) SG11201909868YA (en)
WO (1) WO2018204803A1 (en)

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