SG11201809699XA - Modulatory polynucleotides - Google Patents

Modulatory polynucleotides

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Publication number
SG11201809699XA
SG11201809699XA SG11201809699XA SG11201809699XA SG11201809699XA SG 11201809699X A SG11201809699X A SG 11201809699XA SG 11201809699X A SG11201809699X A SG 11201809699XA SG 11201809699X A SG11201809699X A SG 11201809699XA SG 11201809699X A SG11201809699X A SG 11201809699XA
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SG
Singapore
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international
massachusetts
street
cambridge
pct
Prior art date
Application number
SG11201809699XA
Inventor
Dinah Wen-Yee Sah
Jinzhao Hou
Mathieu Nonnenmacher
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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 SG11201809699XA publication Critical patent/SG11201809699XA/en

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    • 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
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    • 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
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    • C12N15/09—Recombinant DNA-technology
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
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    • 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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Abstract

International Patent Classification: C12N 15/113 (2010.01) Cl 2N 15/85 (2006.01) (21) International Application Number: PCT/US2017/033268 (22) International Filing Date: 18 May 2017 (18.05.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/338,137 18 May 2016 (18.05.2016) US 62/485,050 13 April 2017 (13.04.2017) US (71) Applicant: VOYAGER THERAPEUTICS, INC. [US/US]; 75 SIDNEY STREET, CAMBRIDGE, Massa- chusetts 02139 (US). (72) Inventors: SAH, Dinah Wen-Yee; 75 Sidney Street, Cam- bridge, Massachusetts 02139 (US). HOU, Jinzhao; 75 Sid- ney Street, Cambridge, Massachusetts 02139 (US). NON- NENMACHER, Mathieu; 75 Sidney Street, Cambridge, Massachusetts 02139 (US). (74) Agent: WARD, Donna T. et al.; DT WARD, PC, 142A Main Street, Groton, Massachusetts 01450 (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, 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: MODULATORY POLYNUCLEOTIDES FIG. 1 Seed II IIIIIII till f Thermodynamic asymmetry (e.g., loops aed bulges) Passenger strand (sense) Guide strand Dresha cleavage site .CNNC (antisense or miR) Dicer cleavage site Optimized 5U Loop one helix turn Paired G — Unpaired U 13 rit cc O N O Loop motif: UGLIO (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 23 November 2017 (23.11.2017) WIPO I PCT ill~~~~~~~~ 011101tIIl DIDu IDIIIDIDIIl omolommoiflo oimIE (10) International Publication Number WO 2017/201248 Al \" (57) : The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of modulatory polynucleotides.
SG11201809699XA 2016-05-18 2017-05-18 Modulatory polynucleotides SG11201809699XA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662338137P 2016-05-18 2016-05-18
US201762485050P 2017-04-13 2017-04-13
PCT/US2017/033268 WO2017201248A1 (en) 2016-05-18 2017-05-18 Modulatory polynucleotides

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SG11201809699XA true SG11201809699XA (en) 2018-12-28

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US (3) US10584337B2 (en)
EP (1) EP3458588A4 (en)
JP (3) JP7066635B2 (en)
KR (3) KR20240056729A (en)
CN (2) CN117904112A (en)
AU (2) AU2017267665C1 (en)
BR (1) BR112018073384A2 (en)
CA (1) CA3024448A1 (en)
IL (2) IL262784B2 (en)
MX (2) MX2018014154A (en)
RU (1) RU2758488C2 (en)
SG (1) SG11201809699XA (en)
WO (1) WO2017201248A1 (en)
ZA (1) ZA201807401B (en)

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