CY1120084T1 - Oλiγονουκλεοτiδiα για τη ρυθμιση της γονιδιακης εκφρασης και οι χρησεις αυτων - Google Patents

Oλiγονουκλεοτiδiα για τη ρυθμιση της γονιδιακης εκφρασης και οι χρησεις αυτων

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Publication number
CY1120084T1
CY1120084T1 CY20171101270T CY171101270T CY1120084T1 CY 1120084 T1 CY1120084 T1 CY 1120084T1 CY 20171101270 T CY20171101270 T CY 20171101270T CY 171101270 T CY171101270 T CY 171101270T CY 1120084 T1 CY1120084 T1 CY 1120084T1
Authority
CY
Cyprus
Prior art keywords
oligonucleotide
regulation
gene expression
oligonucleotides
regulating
Prior art date
Application number
CY20171101270T
Other languages
Greek (el)
English (en)
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 CY1120084T1 publication Critical patent/CY1120084T1/el

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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
    • 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
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
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    • 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
    • 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
    • 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
    • C12N2310/11—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
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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
    • 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
    • C12N2310/321—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
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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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    • C12N2310/00—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
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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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Oncology (AREA)
  • Neurology (AREA)
  • Plant Pathology (AREA)
  • Inorganic Chemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Endocrinology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pulmonology (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Reproductive Health (AREA)
  • Psychology (AREA)
CY20171101270T 2012-02-24 2017-12-05 Oλiγονουκλεοτiδiα για τη ρυθμιση της γονιδιακης εκφρασης και οι χρησεις αυτων CY1120084T1 (el)

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
CY1120084T1 true CY1120084T1 (el) 2018-12-12

Family

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Family Applications (1)

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CY20171101270T CY1120084T1 (el) 2012-02-24 2017-12-05 Oλiγονουκλεοτiδiα για τη ρυθμιση της γονιδιακης εκφρασης και οι χρησεις αυτων

Country Status (28)

Country Link
US (2) US10023867B2 (enExample)
EP (1) EP2817407B1 (enExample)
JP (2) JP6333739B2 (enExample)
KR (1) KR101992925B1 (enExample)
CN (2) CN115216475A (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) SG10201606946YA (enExample)
SI (1) SI2817407T1 (enExample)
SM (1) SMT201800042T1 (enExample)
TN (1) TN2014000327A1 (enExample)
WO (1) WO2013124807A2 (enExample)

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JP2015518712A (ja) 2012-05-16 2015-07-06 ラナ セラピューティクス インコーポレイテッド Ｍｅｃｐ２発現を調節するための組成物及び方法
JP2015518713A (ja) 2012-05-16 2015-07-06 ラナ セラピューティクス インコーポレイテッド Ｕｔｒｎ発現を調節するための組成物及び方法
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CN104583401A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节atp2a2表达的组合物和方法
EP2850188A4 (en) 2012-05-16 2016-01-20 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING THE EXPRESSION OF THE HEMOGLOBIN GENE FAMILIES
WO2013173638A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating smn gene family expression
CA3047076A1 (en) * 2016-12-13 2018-06-21 Am Sciences Inc Pharmaceutical composition for preventing or treating hepatitis b
US20250205341A1 (en) * 2022-03-22 2025-06-26 Bion Inc. Protein delivery composition containing bile salt and cationic peptide and use thereof

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SG11201404852WA (en) 2014-10-30
CA2864060C (en) 2020-03-10
NO2817407T3 (enExample) 2018-02-03
US10752900B2 (en) 2020-08-25
EP2817407A2 (en) 2014-12-31
KR20140130514A (ko) 2014-11-10
SMT201800042T1 (it) 2018-03-08
EP2817407B1 (en) 2017-09-06
JP2018078893A (ja) 2018-05-24
AU2013223694B2 (en) 2019-01-03
AU2013223694A1 (en) 2014-09-25
TN2014000327A1 (en) 2015-12-21
CA2864060A1 (en) 2013-08-29
KR101992925B1 (ko) 2019-06-25
CN104169421A (zh) 2014-11-26
PL2817407T3 (pl) 2018-03-30
IN2014MN01493A (enExample) 2015-04-17
AR090129A1 (es) 2014-10-22
CN115216475A (zh) 2022-10-21
AU2018278972A1 (en) 2019-01-17
JP6333739B2 (ja) 2018-05-30
SI2817407T1 (en) 2018-01-31
BR112014020885A2 (pt) 2018-01-09
SG10201606946YA (en) 2016-10-28
DK2817407T3 (en) 2017-12-18
RS56631B1 (sr) 2018-03-30
WO2013124807A3 (en) 2013-12-27
HUE034846T2 (en) 2018-03-28
JP2015513398A (ja) 2015-05-14
RU2014138198A (ru) 2016-04-10
HK1201878A1 (en) 2015-09-11
US10023867B2 (en) 2018-07-17
WO2013124807A2 (en) 2013-08-29
ITMI20120275A1 (it) 2013-08-25
ES2651147T3 (es) 2018-01-24
LT2817407T (lt) 2018-01-25
US20180282731A1 (en) 2018-10-04
RU2648140C2 (ru) 2018-03-22
MA35935B1 (fr) 2014-12-01
BR112014020885B1 (pt) 2020-10-27
US20160040166A1 (en) 2016-02-11
PT2817407T (pt) 2017-12-07

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