SG11201811554SA - Antisense oligonucleotides for modulating htra1 expression - Google Patents

Antisense oligonucleotides for modulating htra1 expression

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Publication number
SG11201811554SA
SG11201811554SA SG11201811554SA SG11201811554SA SG11201811554SA SG 11201811554S A SG11201811554S A SG 11201811554SA SG 11201811554S A SG11201811554S A SG 11201811554SA SG 11201811554S A SG11201811554S A SG 11201811554SA SG 11201811554S A SG11201811554S A SG 11201811554SA
Authority
SG
Singapore
Prior art keywords
roche
fremtidsvej
international
horsholm
copenhagen
Prior art date
Application number
SG11201811554SA
Inventor
Roberto Iacone
Peter Hagedorn
Susanne Kammler
Søren Ottosen
Sindri Traustason
Heidi Hudlebusch
Lykke Pedersen
Original Assignee
Hoffmann La Roche
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 Hoffmann La Roche filed Critical Hoffmann La Roche
Publication of SG11201811554SA publication Critical patent/SG11201811554SA/en

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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
    • C12N15/1137—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 against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111011111111101 IIIIIIIII101111101001H0111010111111111111110111111011110111111 Organization International Bureau (10) International Publication Number (43) International Publication Date ......0\"\" WO 2018/002105 Al 04 January 2018 (04.01.2018) W I PO I PCT (51) International Patent Classification: (84) Designated States (unless otherwise indicated, for every C12N 15/113 (2010.01) A61P 27/02 (2006.01) kind of regional protection available): ARIPO (BW, GH, (21) International Application Number: GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, PCT/EP2017/065937 UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, (22) International Filing Date: EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, 28 June 2017 (28.06.2017) 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, (25) Filing Language: English KM, ML, MR, NE, SN, TD, TG). (26) Publication Language: English Published: (30) Priority Data: — with international search report (Art. 21(3)) 16177508.5 01 July 2016 (01.07.2016) EP — with sequence listing part of description (Rule 5.2(a)) 17170129.5 09 May 2017 (09.05.2017) EP (71) Applicant (for all designated States except US): F. HOFF- MANN-LA ROCHE AG [CH/CH]; Grenzacherstrasse 124, 4070 Basel (CH). (71) Applicant (for US only): HOFFMANN-LA ROCHE INC. [US/US]; Overlook at Great Notch, 150 Clove Road, 8th Floor, Suite 8 - Legal Department, Little Falls, New Jer- sey 07424 (US). — = Inventors: IACONE, Roberto; c/o F. Hoffmann-La Roche (72) AG, Grenzacherstrasse 124, 4070 Basel (CH). HAGE- = DORN, Peter; c/o Roche Innovation Center Copenhagen _ A/S, Fremtidsvej 3, 2970 Horsholm (DK). KAMMLER, Susanne; c/o Roche Innovation Center Copenhagen A/S, Fremtidsvej 3,2970 Horsholm (DK). OTTOSEN, Soren; c/ = o Roche Innovation Center Copenhagen A/S, Fremtidsvej 3, 2970 Horsholm (DK). TRAUSTASON, Sindri; c/o Roche = _= Innovation Center Copenhagen A/S, Fremtidsvej 3, 2970 Horsholm (DK). HUDLEBUSCH, Heidi; c/o Roche limo- vation Center Copenhagen A/S, Fremtidsvej 3, 2970 flor- sholm (DK). PEDERSEN, Lykke; c/o Roche Innovation Center Copenhagen A/S, Fremtidsvej 3, 2970 Horsholm = = — = (DK). — _ Agent: TURNER, Mark; Grenzacherstrasse 124, 4070 (74) Basel (CH). = Designated States (unless otherwise indicated, for every = (81) = 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, = — 1-1 kr) 0 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. 1-1 N i © © CO\" (54) Title: ANTISENSE OLIGONUCLEOTIDES FOR MODULATING HTRA1 EXPRESSION 1-1 0 (57) : The present invention relates to antisense oligonucleotides (oligomers) that are complementary to HTRA1, leading N i to modulation of the expression of HTRA1. Modulation of HTRA expression is beneficial for a range of medical disorders, such as 0 macular degeneration, e.g. age-related macular degeneration.
SG11201811554SA 2016-07-01 2017-06-28 Antisense oligonucleotides for modulating htra1 expression SG11201811554SA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16177508 2016-07-01
EP17170129 2017-05-09
PCT/EP2017/065937 WO2018002105A1 (en) 2016-07-01 2017-06-28 Antisense oligonucleotides for modulating htra1 expression

Publications (1)

Publication Number Publication Date
SG11201811554SA true SG11201811554SA (en) 2019-01-30

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SG10202005885VA SG10202005885VA (en) 2016-07-01 2017-06-28 Antisense oligonucleotides for modulating htra1 expression
SG11201811554SA SG11201811554SA (en) 2016-07-01 2017-06-28 Antisense oligonucleotides for modulating htra1 expression

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US (2) US10519450B2 (en)
EP (1) EP3478839A1 (en)
JP (3) JP7060525B2 (en)
KR (1) KR20190025970A (en)
CN (1) CN109415732B (en)
AU (1) AU2017289204A1 (en)
BR (1) BR112018077321A2 (en)
CA (1) CA3026855A1 (en)
CL (1) CL2018003698A1 (en)
CO (1) CO2018013240A2 (en)
CR (1) CR20180606A (en)
IL (1) IL263489A (en)
MA (1) MA45620A (en)
MX (1) MX2018015722A (en)
PE (1) PE20190381A1 (en)
PH (1) PH12018502707A1 (en)
RU (1) RU2019101298A (en)
SG (2) SG10202005885VA (en)
TW (1) TW201803990A (en)
WO (1) WO2018002105A1 (en)
ZA (1) ZA201808291B (en)

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EP3478839A1 (en) 2019-05-08
JP2019522987A (en) 2019-08-22
CR20180606A (en) 2019-02-14
MA45620A (en) 2019-05-08
JP2024029178A (en) 2024-03-05
JP2022106785A (en) 2022-07-20
TW201803990A (en) 2018-02-01
PH12018502707A1 (en) 2019-11-11
CN109415732A (en) 2019-03-01
CN109415732B (en) 2024-01-02
JP7060525B2 (en) 2022-04-26
MX2018015722A (en) 2019-05-27
CO2018013240A2 (en) 2018-12-14
US20180002701A1 (en) 2018-01-04
RU2019101298A3 (en) 2021-01-26
KR20190025970A (en) 2019-03-12
BR112018077321A2 (en) 2019-04-24
US10519450B2 (en) 2019-12-31
JP7416852B2 (en) 2024-01-17
WO2018002105A1 (en) 2018-01-04
SG10202005885VA (en) 2020-07-29
RU2019101298A (en) 2020-08-03
IL263489A (en) 2019-01-31
US20200399641A1 (en) 2020-12-24
PE20190381A1 (en) 2019-03-08
CA3026855A1 (en) 2018-01-04
ZA201808291B (en) 2019-10-30
AU2017289204A1 (en) 2019-01-17
CL2018003698A1 (en) 2019-04-05

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