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MA35935B1 - Oligonucléotides pour moduler l'expression de gènes et leurs utilisations - Google Patents

Oligonucléotides pour moduler l'expression de gènes et leurs utilisations

Info

Publication number
MA35935B1
MA35935B1 MA37342A MA37342A MA35935B1 MA 35935 B1 MA35935 B1 MA 35935B1 MA 37342 A MA37342 A MA 37342A MA 37342 A MA37342 A MA 37342A MA 35935 B1 MA35935 B1 MA 35935B1
Authority
MA
Morocco
Prior art keywords
oligonucleotides
gene expression
modulate gene
modulating
relates
Prior art date
Application number
MA37342A
Other languages
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 MA35935B1 publication Critical patent/MA35935B1/fr

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    • C—CHEMISTRY; METALLURGY
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    • 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
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    • 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
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    • 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
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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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
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
    • C12N2310/30—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
    • C12N2310/30—Chemical structure
    • 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
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    • 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
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/30—Chemical structure
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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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  • Plant Pathology (AREA)
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  • Ophthalmology & Optometry (AREA)
  • Hematology (AREA)
  • Heart & Thoracic Surgery (AREA)
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Abstract

La présente invention concerne des oligonucléotides pour moduler l'expression d'un gène, en particulier pour moduler un gène responsable d'une pathologie d'origine génétique, tumorale ou virale. En outre, la présente invention concerne l'utilisation desdits oligonucléotides, éventuellement chimiquement modifiés, pour le traitement et/ou le diagnostic desdites maladies.
MA37342A 2012-02-24 2014-09-10 Oligonucléotides pour moduler l'expression de gènes et leurs utilisations MA35935B1 (fr)

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 (fr) 2012-02-24 2013-02-21 Oligonucléotides pour moduler l'expression de gènes et leurs utilisations

Publications (1)

Publication Number Publication Date
MA35935B1 true MA35935B1 (fr) 2014-12-01

Family

ID=45999941

Family Applications (1)

Application Number Title Priority Date Filing Date
MA37342A MA35935B1 (fr) 2012-02-24 2014-09-10 Oligonucléotides pour moduler l'expression de gènes et leurs utilisations

Country Status (28)

Country Link
US (2) US10023867B2 (fr)
EP (1) EP2817407B1 (fr)
JP (2) JP6333739B2 (fr)
KR (1) KR101992925B1 (fr)
CN (2) CN115216475A (fr)
AR (1) AR090129A1 (fr)
AU (2) AU2013223694B2 (fr)
BR (1) BR112014020885B1 (fr)
CA (1) CA2864060C (fr)
CY (1) CY1120084T1 (fr)
DK (1) DK2817407T3 (fr)
ES (1) ES2651147T3 (fr)
HK (1) HK1201878A1 (fr)
HU (1) HUE034846T2 (fr)
IN (1) IN2014MN01493A (fr)
IT (1) ITMI20120275A1 (fr)
LT (1) LT2817407T (fr)
MA (1) MA35935B1 (fr)
NO (1) NO2817407T3 (fr)
PL (1) PL2817407T3 (fr)
PT (1) PT2817407T (fr)
RS (1) RS56631B1 (fr)
RU (1) RU2648140C2 (fr)
SG (2) SG11201404852WA (fr)
SI (1) SI2817407T1 (fr)
SM (1) SMT201800042T1 (fr)
TN (1) TN2014000327A1 (fr)
WO (1) WO2013124807A2 (fr)

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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
CN104540947A (zh) 2012-05-16 2015-04-22 Rana医疗有限公司 用于调节smn基因家族表达的组合物和方法
WO2013173608A1 (fr) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions et méthodes pour moduler l'expression de mecp2
WO2013173599A1 (fr) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions et méthodes pour moduler l'expression de la famille multigénique de l'hémoglobine
US10174323B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating ATP2A2 expression
KR20160074368A (ko) 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Utrn 발현을 조절하기 위한 조성물 및 방법
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
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Publication number Publication date
US10023867B2 (en) 2018-07-17
RU2014138198A (ru) 2016-04-10
CA2864060A1 (fr) 2013-08-29
SG11201404852WA (en) 2014-10-30
SI2817407T1 (en) 2018-01-31
JP6333739B2 (ja) 2018-05-30
AU2013223694B2 (en) 2019-01-03
AU2018278972A1 (en) 2019-01-17
ES2651147T3 (es) 2018-01-24
EP2817407B1 (fr) 2017-09-06
KR101992925B1 (ko) 2019-06-25
BR112014020885A2 (pt) 2018-01-09
HK1201878A1 (en) 2015-09-11
CY1120084T1 (el) 2018-12-12
CN115216475A (zh) 2022-10-21
EP2817407A2 (fr) 2014-12-31
AU2013223694A1 (en) 2014-09-25
PT2817407T (pt) 2017-12-07
LT2817407T (lt) 2018-01-25
RS56631B1 (sr) 2018-03-30
US10752900B2 (en) 2020-08-25
WO2013124807A2 (fr) 2013-08-29
NO2817407T3 (fr) 2018-02-03
JP2015513398A (ja) 2015-05-14
KR20140130514A (ko) 2014-11-10
ITMI20120275A1 (it) 2013-08-25
CA2864060C (fr) 2020-03-10
RU2648140C2 (ru) 2018-03-22
TN2014000327A1 (en) 2015-12-21
US20160040166A1 (en) 2016-02-11
HUE034846T2 (en) 2018-03-28
JP2018078893A (ja) 2018-05-24
DK2817407T3 (en) 2017-12-18
IN2014MN01493A (fr) 2015-04-17
WO2013124807A3 (fr) 2013-12-27
AR090129A1 (es) 2014-10-22
SMT201800042T1 (it) 2018-03-08
CN104169421A (zh) 2014-11-26
PL2817407T3 (pl) 2018-03-30
SG10201606946YA (en) 2016-10-28
US20180282731A1 (en) 2018-10-04
BR112014020885B1 (pt) 2020-10-27

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