SG11201808912QA - Targeted nucleic acid conjugate compositions - Google Patents

Targeted nucleic acid conjugate compositions

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Publication number
SG11201808912QA
SG11201808912QA SG11201808912QA SG11201808912QA SG11201808912QA SG 11201808912Q A SG11201808912Q A SG 11201808912QA SG 11201808912Q A SG11201808912Q A SG 11201808912QA SG 11201808912Q A SG11201808912Q A SG 11201808912QA SG 11201808912Q A SG11201808912Q A SG 11201808912QA
Authority
SG
Singapore
Prior art keywords
international
british columbia
burnaby
rule
glenlyon
Prior art date
Application number
SG11201808912QA
Inventor
James Heyes
Richard J Holland
Alan D Martin
Mark Wood
Original Assignee
Arbutus Biopharma Corp
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.)
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Publication date
Application filed by Arbutus Biopharma Corp filed Critical Arbutus Biopharma Corp
Publication of SG11201808912QA publication Critical patent/SG11201808912QA/en

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
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    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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    • A61K47/554Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
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    • C07C235/10Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by nitrogen atoms not being part of nitro or nitroso groups
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  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)

Abstract

WO 17 / 177 3 26 Al (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date WO 2017/177326 Al 19 October 2017 (19.10.2017) WIPO I PCT (51) International Patent Classification: C12N 15/113 (2010.01) CO7D 209/52 (2006.01) A61K 47/54 (2017.01) C07F 9/40 (2006.01) CO7C 217/52 (2006.01) CO7H 15/26 (2006.01) CO7C 235/06 (2006.01) CO7H 21/00 (2006.01) CO7C 237/08 (2006.01) CO7H 21/02 (2006.01) CO7C 237/20 (2006.01) C07J 63/00 (2006.01) CO7D 207/08 (2006.01) C07J 9/00 (2006.01) CO7D 207/12 (2006.01) Cl 2N 15/87 (2006.01) CO7D 209/12 (2006.01) (21) International Application Number: PCT/CA2017/050447 (22) International Filing Date: 11 April 2017 (11.04.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/321,034 11 April 2016 (11.04.2016) US 62/417,156 3 November 2016 (03.11.2016) US 62/438,310 22 December 2016 (22.12.2016) US (71) Applicant: PROTIVA BIOTHERAPEUTICS, INC. [CA/CA]; 100-8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). (72) Inventors; and (71) Applicants : HEYES, James [CA/CA]; 100-8900 Glenly- on Parkway, Burnaby, British Columbia V5J 5J8 (CA). HOLLAND, Richard J. [CA/CA]; 100-8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). MARTIN, Alan D. [CA/CA]; 100-8900 Glenlyon Park- way, Burnaby, British Columbia V5J 5J8 (CA). WOOD, Mark [CA/CA]; 100-8900 Glenlyon Parkway, Burnaby, British Columbia V5J 5J8 (CA). (74) Agent: SMART & BIGGAR; PO Box 11115, #2300- 1055 West Georgia Street, Vancouver, British Columbia V6E 3P3 (CA). (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). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.1700) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: — with international search report (Art. 21(3)) [Continued on next page] (54) Title: TARGETED NUCLEIC ACID CONJUGATE COMPOSITIONS 1 11111 11111111 0 111 0 1 11111 11111 0 10 1111 I 1 1 1 0 10 1110 11111 0 111 0 111 1111 1111111 1111 0 11 1111 HN '10° 0 O H OAc NH AGO O NH X d Figure 1 (57) : The invention provides conjugates that comprise a targeting moiety, a nucleic acid, and optional linking groups as well as synthetic intermediates and synthetic methods useful for preparing the conjugates. The conjugates are useful to target thera- peutic nucleic acids to the liver and to treat liver diseases including hepatitis (e.g. hepatitis B and hepatitis D). WO 2017/177326 Al MIDEDIMOM0101011101010MOIDEHIIIMMEMOVOIMIE before the expiration of the time limit for amending the — with sequence listing part of description (Rule 5.2(a)) claims and to be republished in the event of receipt of amendments (Rule 48.2(h))
SG11201808912QA 2016-04-11 2017-04-11 Targeted nucleic acid conjugate compositions SG11201808912QA (en)

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US201662321034P 2016-04-11 2016-04-11
US201662417156P 2016-11-03 2016-11-03
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