LT2817407T - Oligonukleotidai, skirti genų raiškos moduliavimui, ir jų panaudojimas - Google Patents

Oligonukleotidai, skirti genų raiškos moduliavimui, ir jų panaudojimas

Info

Publication number
LT2817407T
LT2817407T LTEP13716415.8T LT13716415T LT2817407T LT 2817407 T LT2817407 T LT 2817407T LT 13716415 T LT13716415 T LT 13716415T LT 2817407 T LT2817407 T LT 2817407T
Authority
LT
Lithuania
Prior art keywords
oligonucleotides
gene expression
modulating gene
modulating
expression
Prior art date
Application number
LTEP13716415.8T
Other languages
English (en)
Lithuanian (lt)
Inventor
Roberto Tonelli
Leonardo VENTURELLI
Andrea TORTORI
Luca MONTEMURRO
Original Assignee
Biogenera S.P.A.
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 Biogenera S.P.A. filed Critical Biogenera S.P.A.
Publication of LT2817407T publication Critical patent/LT2817407T/lt

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    • 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
    • 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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47—Quinolines; Isoquinolines
    • A61K31/475—Quinolines; Isoquinolines having an indole ring, e.g. yohimbine, reserpine, strychnine, vinblastine
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/555—Heterocyclic compounds containing heavy metals, e.g. hemin, hematin, melarsoprol
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7048—Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin, digitoxin or digoxin
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K33/00—Medicinal preparations containing inorganic active ingredients
    • A61K33/24—Heavy metals; Compounds thereof
    • A61K33/243—Platinum; Compounds thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/32—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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Health & Medical Sciences (AREA)
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  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Oncology (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Inorganic Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Pulmonology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Reproductive Health (AREA)
  • Hematology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Communicable Diseases (AREA)
LTEP13716415.8T 2012-02-24 2013-02-21 Oligonukleotidai, skirti genų raiškos moduliavimui, ir jų panaudojimas LT2817407T (lt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
IT000275A ITMI20120275A1 (it) 2012-02-24 2012-02-24 Oligonucleotidi per la modulazione dell'espressione genica e loro usi
PCT/IB2013/051410 WO2013124807A2 (en) 2012-02-24 2013-02-21 Oligonucleotides for modulating gene expression and uses thereof

Publications (1)

Publication Number Publication Date
LT2817407T true LT2817407T (lt) 2018-01-25

Family

ID=45999941

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP13716415.8T LT2817407T (lt) 2012-02-24 2013-02-21 Oligonukleotidai, skirti genų raiškos moduliavimui, ir jų panaudojimas

Country Status (28)

Country Link
US (2) US10023867B2 (enExample)
EP (1) EP2817407B1 (enExample)
JP (2) JP6333739B2 (enExample)
KR (1) KR101992925B1 (enExample)
CN (2) CN104169421A (enExample)
AR (1) AR090129A1 (enExample)
AU (2) AU2013223694B2 (enExample)
BR (1) BR112014020885B1 (enExample)
CA (1) CA2864060C (enExample)
CY (1) CY1120084T1 (enExample)
DK (1) DK2817407T3 (enExample)
ES (1) ES2651147T3 (enExample)
HK (1) HK1201878A1 (enExample)
HU (1) HUE034846T2 (enExample)
IN (1) IN2014MN01493A (enExample)
IT (1) ITMI20120275A1 (enExample)
LT (1) LT2817407T (enExample)
MA (1) MA35935B1 (enExample)
NO (1) NO2817407T3 (enExample)
PL (1) PL2817407T3 (enExample)
PT (1) PT2817407T (enExample)
RS (1) RS56631B1 (enExample)
RU (1) RU2648140C2 (enExample)
SG (2) SG11201404852WA (enExample)
SI (1) SI2817407T1 (enExample)
SM (1) SMT201800042T1 (enExample)
TN (1) TN2014000327A1 (enExample)
WO (1) WO2013124807A2 (enExample)

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ITMI20120275A1 (it) * 2012-02-24 2013-08-25 Biogenera Societa A Responsabilita Limitata Oligonucleotidi per la modulazione dell'espressione genica e loro usi
US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
CN104540946A (zh) 2012-05-16 2015-04-22 Rana医疗有限公司 用于调节utrn表达的组合物和方法
CN104583402A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节mecp2表达的组合物和方法
JP2015523854A (ja) 2012-05-16 2015-08-20 ラナ セラピューティクス インコーポレイテッド Smn遺伝子ファミリー発現を調節するための組成物及び方法
EA201492120A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии atp2a2
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
JP6811338B2 (ja) * 2016-12-13 2021-01-13 エーエム サイエンシーズ インコーポレイテッド B型肝炎の予防または治療用医薬組成物
EP4497432A4 (en) * 2022-03-22 2025-07-30 Bion Inc PROTEIN DELIVERY COMPOSITION CONTAINING BILE SALT AND CATIONIC PEPTIDE AND USE THEREOF

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CA2300938A1 (en) * 1997-08-20 1999-02-25 Somagenics, Inc. Antisense and antigene therapeutics with improved binding properties and methods for their use
WO2001061030A2 (en) * 2000-02-14 2001-08-23 Bollon Arthur P LIBRARIES OF OPTIMUM SUBSEQUENCE REGIONS OF mRNA AND GENOMIC DNA FOR CONTROL OF GENE EXPRESSION
JP2004524371A (ja) * 2001-04-13 2004-08-12 ザ・ポピュレイション・カウンシル,インコーポレイテッド 核レセプターを介するペプチド核酸の細胞核内導入
US20030087861A1 (en) * 2001-05-17 2003-05-08 Iversen Patrick L Combined approach to treatment of cancer using a c-myc antisense oligomer
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JP2011511051A (ja) * 2008-02-05 2011-04-07 アンティソマ リサーチ リミテッド 生物学的物質およびそれらの使用
KR20110091796A (ko) * 2008-12-04 2011-08-12 오피케이오 큐알엔에이, 엘엘씨 종양 억제 유전자에 대한 천연 안티센스 전사체의 억제에 의해 종양 억제 유전자 관련된 질환의 치료
CN102058535B (zh) * 2010-12-21 2013-05-01 中国人民解放军总医院 叶酸受体靶向的新型脂质体
ITMI20120275A1 (it) * 2012-02-24 2013-08-25 Biogenera Societa A Responsabilita Limitata Oligonucleotidi per la modulazione dell'espressione genica e loro usi

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SMT201800042T1 (it) 2018-03-08
BR112014020885A2 (pt) 2018-01-09
IN2014MN01493A (enExample) 2015-04-17
CA2864060C (en) 2020-03-10
JP6333739B2 (ja) 2018-05-30
US10023867B2 (en) 2018-07-17
NO2817407T3 (enExample) 2018-02-03
MA35935B1 (fr) 2014-12-01
HUE034846T2 (en) 2018-03-28
AU2018278972A1 (en) 2019-01-17
SI2817407T1 (en) 2018-01-31
TN2014000327A1 (en) 2015-12-21
CA2864060A1 (en) 2013-08-29
PT2817407T (pt) 2017-12-07
ITMI20120275A1 (it) 2013-08-25
KR101992925B1 (ko) 2019-06-25
CN104169421A (zh) 2014-11-26
CY1120084T1 (el) 2018-12-12
BR112014020885B1 (pt) 2020-10-27
WO2013124807A3 (en) 2013-12-27
PL2817407T3 (pl) 2018-03-30
DK2817407T3 (en) 2017-12-18
US10752900B2 (en) 2020-08-25
EP2817407A2 (en) 2014-12-31
HK1201878A1 (en) 2015-09-11
CN115216475A (zh) 2022-10-21
AU2013223694B2 (en) 2019-01-03
SG11201404852WA (en) 2014-10-30
ES2651147T3 (es) 2018-01-24
AU2013223694A1 (en) 2014-09-25
RU2014138198A (ru) 2016-04-10
AR090129A1 (es) 2014-10-22
RU2648140C2 (ru) 2018-03-22
WO2013124807A2 (en) 2013-08-29
SG10201606946YA (en) 2016-10-28
EP2817407B1 (en) 2017-09-06
JP2018078893A (ja) 2018-05-24
JP2015513398A (ja) 2015-05-14
US20180282731A1 (en) 2018-10-04
RS56631B1 (sr) 2018-03-30
KR20140130514A (ko) 2014-11-10
US20160040166A1 (en) 2016-02-11

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