CA2864060C - Oligonucleotides for modulating gene expression and uses thereof - Google Patents

Oligonucleotides for modulating gene expression and uses thereof Download PDF

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Publication number
CA2864060C
CA2864060C CA2864060A CA2864060A CA2864060C CA 2864060 C CA2864060 C CA 2864060C CA 2864060 A CA2864060 A CA 2864060A CA 2864060 A CA2864060 A CA 2864060A CA 2864060 C CA2864060 C CA 2864060C
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seq
oligonucleotides
oligonucleotide
gene
oligonucleotide according
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French (fr)
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CA2864060A1 (en
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Roberto Tonelli
Leonardo Venturelli
Andrea Tortori
Luca Montemurro
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Biogenera SpA
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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
    • C12N15/1135—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
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/555—Heterocyclic compounds containing heavy metals, e.g. hemin, hematin, melarsoprol
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    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
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    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
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    • A61P31/12—Antivirals
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    • A61P35/00—Antineoplastic agents
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    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • A—HUMAN NECESSITIES
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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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
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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
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    • 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
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3513—Protein; Peptide
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Molecular Biology (AREA)
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  • 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)
CA2864060A 2012-02-24 2013-02-21 Oligonucleotides for modulating gene expression and uses thereof Active CA2864060C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
ITMI2012A000275 2012-02-24
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 (2)

Publication Number Publication Date
CA2864060A1 CA2864060A1 (en) 2013-08-29
CA2864060C true CA2864060C (en) 2020-03-10

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CA2864060A Active CA2864060C (en) 2012-02-24 2013-02-21 Oligonucleotides for modulating gene expression and uses thereof

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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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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
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SMT201800042T1 (it) 2018-03-08
BR112014020885A2 (pt) 2018-01-09
IN2014MN01493A (enExample) 2015-04-17
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
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LT2817407T (lt) 2018-01-25
AR090129A1 (es) 2014-10-22
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SG10201606946YA (en) 2016-10-28
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JP2018078893A (ja) 2018-05-24
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US20180282731A1 (en) 2018-10-04
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