SG11201804729RA - Methods and compositions for treating a serpinc1-associated disorder - Google Patents

Methods and compositions for treating a serpinc1-associated disorder

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Publication number
SG11201804729RA
SG11201804729RA SG11201804729RA SG11201804729RA SG11201804729RA SG 11201804729R A SG11201804729R A SG 11201804729RA SG 11201804729R A SG11201804729R A SG 11201804729RA SG 11201804729R A SG11201804729R A SG 11201804729RA SG 11201804729R A SG11201804729R A SG 11201804729RA
Authority
SG
Singapore
Prior art keywords
international
street
cambridge
floor
compositions
Prior art date
Application number
SG11201804729RA
Inventor
Akin Akinc
Benny Sorensen
Pushkal Garg
Gabriel Robbie
Original Assignee
Genzyme 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 Genzyme Corp filed Critical Genzyme Corp
Publication of SG11201804729RA publication Critical patent/SG11201804729RA/en

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
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    • C12N2310/3533Halogen
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/35Special therapeutic applications based on a specific dosage / administration regimen

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) CORRECTED VERSION (19) World Intellectual Property Organization International Bureau .9 . si wiel y) (43) International Publication Date sawe 15 June 2017(15.06.2017) WIPO I PCT ID Hit (10) WO 111111111111111111111111111111 International III 2017/100236 Publication 111111111111111111111111111111t111111111111111 Number A8 (51) International Patent Classification: (81) Designated States (unless otherwise indicated, for every A61K 31/713 (2006.01) A61P 7/04 (2006.01) kind of national protection available): AE, AG, AL, AM, C12N 15/113 (2010.01) 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, (21) International Application Number: DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, PCT/US2016/065245 HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, (22) International Filing Date: KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, 7 December 2016 (07.12.2016) 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, (25) Filing Language: English SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, (26) Publication Language: English TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (30) Priority Data: 62/264,013 7 December 2015 (07.12.2015) US (84) Designated States (unless otherwise indicated, for every 62/315,228 30 March 2016 (30.03.2016) US kind of regional protection available): ARIPO (BW, GH, 62/366,304 25 July 2016 (25.07.2016) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, 62/429,241 2 December 2016 (02.12.2016) US TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, (71) Applicant: ALNYLAM PHARMACEUTICALS, INC. DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, [US/US]; 300 Third Street, 3rd Floor, Cambridge, MA LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, 02142 (US). TR), BJ, SM, OAPI (BF, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). (72) Inventors: AKINC, Akin; 300 Third Street, 3rd Floor, Cambridge, MA 02142 (US). SORENSEN, Benny; 300 Published: Third Street, 3rd Floor, Cambridge, MA 02142 (US). — with international search report (Art 21(3)) GARG, Pushkal; 300 Third Street, 3rd Floor, Cambridge, MA 02142 ROBBIE, Gabriel; 300 Third Street, 3rd — with sequence listing of description (Rule 5.2(a)) (US). part Floor, Cambridge, MA 02142 (US). (48) Date of publication of this corrected version: (74) Agents: ZACHARAKIS, Maria Laccotripe et al.; Mc- 20 July 2017 Carter & English, LLP, 265 Franklin Street, Boston, MA Information (15) about Correction: 02110 (US). see Notice of 20 July 2017 Ge .4 t M ei O O 1-1 it - - 1-1 0 (54) Title: METHODS AND COMPOSITIONS FOR TREATING A SERPINC1-ASSOCIATED DISORDER ei 0 (57) : The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc l ,1 . gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc 1 and to treat subjects having a Serpinc 1-associated disease, e.g., a bleeding disorder, such as a hemophilia.
SG11201804729RA 2015-12-07 2016-12-07 Methods and compositions for treating a serpinc1-associated disorder SG11201804729RA (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562264013P 2015-12-07 2015-12-07
US201662315228P 2016-03-30 2016-03-30
US201662366304P 2016-07-25 2016-07-25
US201662429241P 2016-12-02 2016-12-02
PCT/US2016/065245 WO2017100236A1 (en) 2015-12-07 2016-12-07 Methods and compositions for treating a serpinc1-associated disorder

Publications (1)

Publication Number Publication Date
SG11201804729RA true SG11201804729RA (en) 2018-07-30

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SG10201913085TA SG10201913085TA (en) 2015-12-07 2016-12-07 Methods and compositions for treating a serpinc1-associated disorder
SG11201804729RA SG11201804729RA (en) 2015-12-07 2016-12-07 Methods and compositions for treating a serpinc1-associated disorder

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Country Status (15)

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US (2) US11091759B2 (en)
EP (2) EP4424828A1 (en)
JP (3) JP6985283B2 (en)
KR (3) KR20210157474A (en)
CN (1) CN108601795A (en)
AU (2) AU2016368289C1 (en)
BR (1) BR112018011450A2 (en)
CA (1) CA3007014A1 (en)
CO (1) CO2018006590A2 (en)
IL (2) IL308717A (en)
MX (1) MX2018006989A (en)
RU (2) RU2754188C2 (en)
SG (2) SG10201913085TA (en)
TW (2) TWI836281B (en)
WO (1) WO2017100236A1 (en)

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