SG11201908771YA - Modified oligonucleotides and therapeutic uses thereof - Google Patents

Modified oligonucleotides and therapeutic uses thereof

Info

Publication number
SG11201908771YA
SG11201908771YA SG11201908771YA SG11201908771YA SG 11201908771Y A SG11201908771Y A SG 11201908771YA SG 11201908771Y A SG11201908771Y A SG 11201908771YA SG 11201908771Y A SG11201908771Y A SG 11201908771YA
Authority
SG
Singapore
Prior art keywords
international
california
road
san diego
torrey pines
Prior art date
Application number
Inventor
Alexander Roloff
Nathan C Gianneschi
Cassandra E Callmann
Matthew P Thompson
Paul A Bertin
Original Assignee
Univ California
Vybyl Holdings Inc
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 Univ California, Vybyl Holdings Inc filed Critical Univ California
Publication of SG11201908771YA publication Critical patent/SG11201908771YA/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/68—Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/54—Medicinal 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
    • A61K47/542—Carboxylic acids, e.g. a fatty acid or an amino acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/62—Medicinal 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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/643—Albumins, e.g. HSA, BSA, ovalbumin or a Keyhole Limpet Hemocyanin [KHL]
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization O V SID o Ho SID Em VII IE International Bureau (10) International Publication Number WO 2018/175592 Al (43) International Publication Date 27 September 2018 (27.09.2018) WIPO I PCT (51) International Patent Classification: A61K 47/54 (2017.01) A61K 9/00 (2006.01) A61K 48/00 (2006.01) A61P 35/00 (2006.01) A61K 47/42 (2006.01) (21) International Application Number: PCT/US2018/023578 (22) International Filing Date: 21 March 2018 (21.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/475,185 22 March 2017 (22.03.2017) US (71) Applicants: THE REGENTS OF THE UNIVERSI- TY OF CALIFORNIA [US/US]; 1111 Franklin Street, 5th Floor, Oakland, California 94607 (US). VY- BYL HOLDINGS, INC. [US/US]; 2501 Davey Road, Woodridge, Illinois 60517 (US). (72) Inventors: ROLOFF, Alexander; 10300 North Torrey Pines Road, San Diego, California 92037 (US). GIAN- NESCHI, Nathan C.; 10300 North Torrey Pines Road, San Diego, California 92037 (US). CALLMANN, Cassandra E.; 10300 North Torrey Pines Road, San Diego, Califor- nia 92037 (US). THOMPSPN, Matthew P.; 10300 North Torrey Pines Road, San Diego, California 92037 (US). BERTIN, Paul A.; 2501 Davey Road, Woodridge, Illinois 60517 (US). (74) Agent: DAILEY, Robert S.; Kilpatrick Townsend & Stockton LLP, 1001 West Fourth Street, Winston Salem, North Carolina 27101 (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, (54) Title: MODIFIED OLIGONUCLEOTIDES AND THERAPEUTIC USES THEREOF 0 OH oligonucleotide 8 0 FIG. 1 (57) : The present disclosure generally provides nucleotide-based compounds useful for treating various diseases, including cancer. In some aspects, the disclosure provides oligonucleotides that are chemically modified to include an engineered fatty-acid residue, for example, to assist with improving the half-life of such compounds or assisting with cell penetration (e.g., penetration into tumor cells). In some aspects, the disclosure provides compositions that include such modified nucleotides and a protein, such as albumin or mimetics thereof. The disclosure provides various uses of the compounds and compositions. [Continued on next page] WO 2018/175592 Al MIDEDIM01101 DIDIREIDIOMOMIIII111111111111111111111111111111111 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))
SG11201908771Y 2017-03-22 2018-03-21 Modified oligonucleotides and therapeutic uses thereof SG11201908771YA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762475185P 2017-03-22 2017-03-22
PCT/US2018/023578 WO2018175592A1 (en) 2017-03-22 2018-03-21 Modified oligonucleotides and therapeutic uses thereof

Publications (1)

Publication Number Publication Date
SG11201908771YA true SG11201908771YA (en) 2019-10-30

Family

ID=63586170

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201908771Y SG11201908771YA (en) 2017-03-22 2018-03-21 Modified oligonucleotides and therapeutic uses thereof

Country Status (9)

Country Link
US (1) US20200046846A1 (en)
EP (1) EP3600439A4 (en)
JP (1) JP2020514383A (en)
KR (1) KR20190123351A (en)
CN (1) CN110636865A (en)
AU (1) AU2018237139A1 (en)
CA (1) CA3057292A1 (en)
SG (1) SG11201908771YA (en)
WO (1) WO2018175592A1 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4213883A1 (en) * 2020-09-16 2023-07-26 Astrazeneca AB Oligonucleotides conjugated to fatty acids
CA3231700A1 (en) * 2021-09-10 2023-03-16 Guardian Therapeutics, Llc Fatty acid conjugates of nucleic acids

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0821001A4 (en) * 1995-03-31 2002-07-17 Drug Delivery System Inst Ltd Amidite derivatives and oligonucleotide derivatives
US8969543B2 (en) * 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
US20090306178A1 (en) * 2006-03-27 2009-12-10 Balkrishen Bhat Conjugated double strand compositions for use in gene modulation
KR101224828B1 (en) * 2009-05-14 2013-01-22 (주)바이오니아 SiRNA conjugate and preparing method thereof
GB0910723D0 (en) * 2009-06-22 2009-08-05 Sylentis Sau Novel drugs for inhibition of gene expression
US9315810B2 (en) * 2011-06-03 2016-04-19 National University Corporation Hokkaido University Oligonucleotide derivative, oligonucleotide derivative-containing pharmaceutical composition for treatment and pharmaceutical composition for diagnosis, and oligonucleotide derivative for regulation of miRNA function
RU2599449C1 (en) * 2011-12-15 2016-10-10 Байонир Корпорейшн New oligonucleotides conjugates and use thereof
EP3019200B1 (en) * 2013-07-11 2022-03-23 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
KR102302634B1 (en) * 2013-09-13 2021-09-14 더 스크립스 리서치 인스티튜트 Modified therapeutic agents and compositions thereof
US10039809B2 (en) * 2013-12-18 2018-08-07 The California Institute For Biomedical Research Modified therapeutic agents, stapled peptide lipid conjugates, and compositions thereof
JP6773677B2 (en) * 2015-03-17 2020-10-21 アローヘッド ファーマシューティカルズ インコーポレイテッド Improved disulfide-containing alkyne conjugate
MA41794A (en) * 2015-03-18 2018-01-23 The California Institute For Biomedical Res MODIFIED THERAPEUTIC AGENTS AND ASSOCIATED COMPOSITIONS
US10633653B2 (en) * 2015-08-14 2020-04-28 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
CA2998528A1 (en) * 2015-09-22 2017-03-30 The Regents Of The University Of California Modified cytotoxins and their therapeutic use

Also Published As

Publication number Publication date
EP3600439A4 (en) 2021-01-13
WO2018175592A1 (en) 2018-09-27
JP2020514383A (en) 2020-05-21
EP3600439A1 (en) 2020-02-05
CA3057292A1 (en) 2018-09-27
CN110636865A (en) 2019-12-31
US20200046846A1 (en) 2020-02-13
AU2018237139A1 (en) 2019-10-17
KR20190123351A (en) 2019-10-31

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