RS56428B1 - Sastavi i metode za inhibiranje ekspresije alas1 gena - Google Patents

Sastavi i metode za inhibiranje ekspresije alas1 gena

Info

Publication number
RS56428B1
RS56428B1 RS20170890A RSP20170890A RS56428B1 RS 56428 B1 RS56428 B1 RS 56428B1 RS 20170890 A RS20170890 A RS 20170890A RS P20170890 A RSP20170890 A RS P20170890A RS 56428 B1 RS56428 B1 RS 56428B1
Authority
RS
Serbia
Prior art keywords
compositions
methods
inhibiting expression
alas1 gene
alas1
Prior art date
Application number
RS20170890A
Other languages
English (en)
Inventor
Brian Bettencourt
Kevin Fitzgerald
William Querbes
Makiko Yasuda
Robert J Desnick
Original Assignee
Alnylam Pharmaceuticals Inc
Icahn School Med Mount Sinai
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=RS56428(B1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Alnylam Pharmaceuticals Inc, Icahn School Med Mount Sinai filed Critical Alnylam Pharmaceuticals Inc
Publication of RS56428B1 publication Critical patent/RS56428B1/sr

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    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/56—Medicinal 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
    • A61K47/59—Medicinal 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
    • A61K47/60—Medicinal 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
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    • A61P25/00—Drugs for disorders of the nervous system
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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
    • C12N15/1137—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 enzymes
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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 N.A.
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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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    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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    • C12N2310/3521—Methyl
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    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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    • C12N2320/30—Special therapeutic applications
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    • C12Y—ENZYMES
    • C12Y203/00—Acyltransferases (2.3)
    • C12Y203/01—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/01037—5-Aminolevulinate synthase (2.3.1.37)
RS20170890A 2012-04-10 2013-04-10 Sastavi i metode za inhibiranje ekspresije alas1 gena RS56428B1 (sr)

Applications Claiming Priority (4)

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
EP13721433.4A EP2836595B1 (en) 2012-04-10 2013-04-10 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
RS56428B1 true RS56428B1 (sr) 2018-01-31

Family

ID=48326393

Family Applications (1)

Application Number Title Priority Date Filing Date
RS20170890A RS56428B1 (sr) 2012-04-10 2013-04-10 Sastavi i metode za inhibiranje ekspresije alas1 gena

Country Status (28)

Country Link
US (8) US9133461B2 (sr)
EP (3) EP3868883A1 (sr)
JP (4) JP6422854B2 (sr)
KR (3) KR102423326B1 (sr)
CN (2) CN104540948B (sr)
AR (1) AR090641A1 (sr)
AU (4) AU2013245949A1 (sr)
BR (1) BR112014025020A2 (sr)
CA (2) CA3169218A1 (sr)
CL (2) CL2014002725A1 (sr)
CO (1) CO7121351A2 (sr)
CY (1) CY1119491T1 (sr)
DK (1) DK2836595T3 (sr)
ES (1) ES2640260T3 (sr)
HK (2) HK1201876A1 (sr)
HR (1) HRP20171369T1 (sr)
HU (1) HUE034311T2 (sr)
IL (5) IL281295B (sr)
LT (1) LT2836595T (sr)
ME (1) ME02850B (sr)
MX (3) MX368723B (sr)
PL (1) PL2836595T3 (sr)
PT (1) PT2836595T (sr)
RS (1) RS56428B1 (sr)
RU (2) RU2687223C2 (sr)
SI (1) SI2836595T1 (sr)
WO (1) WO2013155204A2 (sr)
ZA (1) ZA201408111B (sr)

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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
EP3633039A1 (en) * 2013-05-01 2020-04-08 Ionis Pharmaceuticals, Inc. Compositions and methods
US9670492B2 (en) 2013-08-28 2017-06-06 Ionis Pharmaceuticals, Inc. Modulation of prekallikrein (PKK) expression
PE20161130A1 (es) * 2013-10-04 2016-11-25 Icahn School Med Mount Sinai Composiciones y metodos para inhibir la expresion del gen alas1
EP3060664B1 (en) * 2013-10-25 2021-07-07 Sanofi Microrna compounds and methods for modulating mir-21 activity
US9701966B2 (en) 2013-12-27 2017-07-11 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA
CA2938857A1 (en) 2014-02-11 2015-08-20 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
WO2015168618A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
CN106255755B (zh) 2014-05-01 2020-07-24 Ionis制药公司 用于调节血管生成素样蛋白3表达的组合物和方法
HUE052709T2 (hu) * 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
EP3812462A1 (en) 2014-08-20 2021-04-28 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
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
TW201718857A (zh) * 2015-09-14 2017-06-01 艾爾妮蘭製藥公司 用於抑制alas1基因表現之組合物及方法
JP2018531605A (ja) 2015-11-06 2018-11-01 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． アポリポタンパク質（ａ）発現の調節
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
SI3607069T1 (sl) 2017-04-05 2023-02-28 Silence Therapeutics Gmbh Produkti in sestavki
US11324820B2 (en) 2017-04-18 2022-05-10 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (HBV) infection
EP3417860A1 (en) * 2017-06-19 2018-12-26 Asociación Centro de Investigación Cooperativa en Biociencias - CIC bioGUNE Use of ciclopirox as a modulator of the heme group biosynthesis and in the treatment of porphyrias and other diseases
US11479532B2 (en) * 2017-06-23 2022-10-25 University Of South Florida 5-aminolevulinate synthase inhibitors and methods of use thereof
US11866701B2 (en) 2017-11-01 2024-01-09 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
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