TN2016000114A1 - Compositions and methods for inhibiting expression of the alas1 gene. - Google Patents

Compositions and methods for inhibiting expression of the alas1 gene.

Info

Publication number
TN2016000114A1
TN2016000114A1 TN2016000114A TN2016000114A TN2016000114A1 TN 2016000114 A1 TN2016000114 A1 TN 2016000114A1 TN 2016000114 A TN2016000114 A TN 2016000114A TN 2016000114 A TN2016000114 A TN 2016000114A TN 2016000114 A1 TN2016000114 A1 TN 2016000114A1
Authority
TN
Tunisia
Prior art keywords
compositions
methods
inhibiting expression
alas1 gene
alas1
Prior art date
Application number
TN2016000114A
Other languages
English (en)
Inventor
Brian Bettencourt
Robert Desnick
Kevin Fitzgerald
William Querbes
Makiko Yasuda
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
Application filed by Alnylam Pharmaceuticals Inc, Icahn School Med Mount Sinai filed Critical Alnylam Pharmaceuticals Inc
Publication of TN2016000114A1 publication Critical patent/TN2016000114A1/en

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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
    • 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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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/3533—Halogen
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    • 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)

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TN2016000114A 2013-10-04 2014-10-03 Compositions and methods for inhibiting expression of the alas1 gene. TN2016000114A1 (en)

Applications Claiming Priority (3)

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US201361887288P 2013-10-04 2013-10-04
US201461983720P 2014-04-24 2014-04-24
PCT/US2014/059160 WO2015051318A1 (en) 2013-10-04 2014-10-03 Compositions and methods for inhibiting expression of the alas1 gene

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TN2016000114A1 true TN2016000114A1 (en) 2017-07-05

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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
TWI768330B (zh) * 2013-10-04 2022-06-21 美國西奈山伊坎醫學院 抑制alas1基因表現的組合物及方法
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
WO2017048843A1 (en) 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the alas1 gene
EP3544617A4 (en) 2016-11-23 2020-08-05 Alnylam Pharmaceuticals, Inc. REDUCED OFF-TARGET MODIFIED RNA AGENTS
TW201930591A (zh) 2018-01-08 2019-08-01 瑞士商諾華公司 用於與嵌合抗原受體療法併用之免疫增強rna
CN115279903A (zh) * 2020-02-21 2022-11-01 Ionis制药公司 用于降低htt表达的方法
JP2024537202A (ja) 2021-10-06 2024-10-10 アルナイラム ファーマシューティカルズ， インコーポレイテッド 急性肝性ポルフィリン症（ａｈｐ）のバイオマーカーとしての腎障害マーカー
EP4646481A1 (en) * 2023-01-06 2025-11-12 Memorial Sloan-Kettering Cancer Center Methods for enhancing the efficacy of rnai therapy by targeting alas1/alas2
CN118460654B (zh) * 2024-07-10 2025-02-14 凯莱英医药集团（天津）股份有限公司 一种Givosiran的制备方法

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