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CY1119491T1 - Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου alas1 - Google Patents

Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου alas1

Info

Publication number
CY1119491T1
CY1119491T1 CY20171100971T CY171100971T CY1119491T1 CY 1119491 T1 CY1119491 T1 CY 1119491T1 CY 20171100971 T CY20171100971 T CY 20171100971T CY 171100971 T CY171100971 T CY 171100971T CY 1119491 T1 CY1119491 T1 CY 1119491T1
Authority
CY
Cyprus
Prior art keywords
methods
compositions
inhalation
gene expression
alas1 gene
Prior art date
Application number
CY20171100971T
Other languages
English (en)
Inventor
Brian Bettencourt
Kevin Fitzgerald
William Querbes
Makiko YASUDA
Robert J. Desnick
Original Assignee
Alnylam Pharmaceuticals, Inc.
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=48326393&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CY1119491(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Alnylam Pharmaceuticals, Inc. filed Critical Alnylam Pharmaceuticals, Inc.
Publication of CY1119491T1 publication Critical patent/CY1119491T1/el

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    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
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    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
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    • A61K47/60Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
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    • C12N15/1137Non-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
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    • C12Y203/01Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
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Abstract

Η εφεύρεση αναφέρεται σε συνθέσεις δίκλωνου ριβονουκλεϊκού οξέος (dsRNA) που στοχεύουν το γονίδιο ALAS1 και σε μεθόδους χρήσης τέτοιων συνθέσεων dsRNA ώστε να μεταβάλλεται (π.χ. να αναστέλλεται) η έκφραση του ALAS1.
CY20171100971T 2012-04-10 2017-09-13 Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου alas1 CY1119491T1 (el)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261622288P 2012-04-10 2012-04-10
US13/835,613 US9133461B2 (en) 2012-04-10 2013-03-15 Compositions and methods for inhibiting expression of the ALAS1 gene
PCT/US2013/036006 WO2013155204A2 (en) 2012-04-10 2013-04-10 Compositions and methods for inhibiting expression of the alas1 gene

Publications (1)

Publication Number Publication Date
CY1119491T1 true CY1119491T1 (el) 2018-03-07

Family

ID=48326393

Family Applications (1)

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CY20171100971T CY1119491T1 (el) 2012-04-10 2017-09-13 Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου alas1

Country Status (28)

Country Link
US (9) US9133461B2 (el)
EP (3) EP3868883A1 (el)
JP (4) JP6422854B2 (el)
KR (3) KR20220106851A (el)
CN (2) CN109112131B (el)
AR (1) AR090641A1 (el)
AU (4) AU2013245949A1 (el)
BR (1) BR112014025020A2 (el)
CA (2) CA2868290A1 (el)
CL (2) CL2014002725A1 (el)
CO (1) CO7121351A2 (el)
CY (1) CY1119491T1 (el)
DK (1) DK2836595T3 (el)
ES (1) ES2640260T3 (el)
HK (2) HK1201876A1 (el)
HR (1) HRP20171369T1 (el)
HU (1) HUE034311T2 (el)
IL (5) IL293818A (el)
LT (1) LT2836595T (el)
ME (1) ME02850B (el)
MX (3) MX368723B (el)
PL (1) PL2836595T3 (el)
PT (1) PT2836595T (el)
RS (1) RS56428B1 (el)
RU (2) RU2019111696A (el)
SI (1) SI2836595T1 (el)
WO (1) WO2013155204A2 (el)
ZA (1) ZA201408111B (el)

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US9133461B2 (en) * 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
KR20240147701A (ko) 2013-05-01 2024-10-08 아이오니스 파마수티컬즈, 인코포레이티드 Hbv 및 ttr 발현을 조절하는 조성물 및 방법
CA2921839A1 (en) 2013-08-28 2015-03-05 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (pkk) expression
MY197646A (en) * 2013-10-04 2023-06-30 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of the alas1 gene
US9994846B2 (en) * 2013-10-25 2018-06-12 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-21 activity
CA2935220A1 (en) 2013-12-27 2015-07-02 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna
CN106103718B (zh) 2014-02-11 2021-04-02 阿尔尼拉姆医药品有限公司 己酮糖激酶(KHK)iRNA组合物及其使用方法
RU2734658C2 (ru) 2014-05-01 2020-10-21 Ионис Фармасьютикалз, Инк. Композиции и способы модулирования экспрессии ангиопоэтин-подобного белка 3
KR102149571B1 (ko) * 2014-05-01 2020-08-31 아이오니스 파마수티컬즈, 인코포레이티드 성장 호르몬 수용체 발현을 조절하기 위한 조성물 및 방법
EP3137091B1 (en) * 2014-05-01 2020-12-02 Ionis Pharmaceuticals, Inc. Conjugates of modified antisense oligonucleotides and their use for modulating pkk expression
SG11201701166UA (en) * 2014-08-20 2017-03-30 Alnylam Pharmaceuticals Inc Modified double-stranded rna agents
JOP20200115A1 (ar) * 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
WO2016061487A1 (en) * 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
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