SG11201602631XA - Compositions and methods for inhibiting expression of the alas1 gene - Google Patents

Compositions and methods for inhibiting expression of the alas1 gene

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Publication number
SG11201602631XA
SG11201602631XA SG11201602631XA SG11201602631XA SG11201602631XA SG 11201602631X A SG11201602631X A SG 11201602631XA SG 11201602631X A SG11201602631X A SG 11201602631XA SG 11201602631X A SG11201602631X A SG 11201602631XA SG 11201602631X A SG11201602631X A SG 11201602631XA
Authority
SG
Singapore
Prior art keywords
compositions
methods
inhibiting expression
alas1 gene
alas1
Prior art date
Application number
SG11201602631XA
Other languages
English (en)
Inventor
Brian Bettencourt
Kevin Fitzgerald
William Querbes
Robert J Desnick
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 SG11201602631XA publication Critical patent/SG11201602631XA/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
    • A—HUMAN NECESSITIES
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    • 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
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • 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
    • A61P25/04—Centrally acting analgesics, e.g. opioids
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    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
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    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
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    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3533—Halogen
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/118—Prognosis of disease development
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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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SG11201602631XA 2013-10-04 2014-10-03 Compositions and methods for inhibiting expression of the alas1 gene SG11201602631XA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361887288P 2013-10-04 2013-10-04
US201461983720P 2014-04-24 2014-04-24
PCT/US2014/059160 WO2015051318A1 (fr) 2013-10-04 2014-10-03 Compositions et méthodes permettant d'inhiber l'expression du gène alas1

Publications (1)

Publication Number Publication Date
SG11201602631XA true SG11201602631XA (en) 2016-05-30

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SG11201602631XA SG11201602631XA (en) 2013-10-04 2014-10-03 Compositions and methods for inhibiting expression of the alas1 gene
SG10201910929QA SG10201910929QA (en) 2013-10-04 2014-10-03 Compositions and methods for inhibiting expression of the alas1 gene

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Country Status (37)

Country Link
US (3) US10119143B2 (fr)
EP (2) EP3052628B1 (fr)
JP (3) JP6613227B2 (fr)
KR (3) KR20220159478A (fr)
CN (1) CN105980559B (fr)
AU (3) AU2014331604B2 (fr)
BR (2) BR122020001264B1 (fr)
CA (2) CA2925357C (fr)
CL (2) CL2016000772A1 (fr)
CR (1) CR20160195A (fr)
CY (2) CY1122975T1 (fr)
DK (1) DK3052628T3 (fr)
DO (2) DOP2016000073A (fr)
EA (1) EA036477B1 (fr)
ES (1) ES2804510T3 (fr)
GT (1) GT201600066A (fr)
HK (1) HK1221738A1 (fr)
HR (1) HRP20200822T1 (fr)
HU (2) HUE049227T2 (fr)
IL (3) IL282747B2 (fr)
LT (2) LT3052628T (fr)
MX (2) MX2016004319A (fr)
MY (2) MY197646A (fr)
NL (1) NL301061I2 (fr)
NO (1) NO2020029I1 (fr)
NZ (2) NZ757749A (fr)
PE (2) PE20161130A1 (fr)
PH (1) PH12016500574A1 (fr)
PL (1) PL3052628T3 (fr)
PT (1) PT3052628T (fr)
SG (2) SG11201602631XA (fr)
SI (1) SI3052628T1 (fr)
TN (1) TN2016000114A1 (fr)
TW (3) TWI768330B (fr)
UA (1) UA124961C2 (fr)
WO (1) WO2015051318A1 (fr)
ZA (2) ZA201602931B (fr)

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Publication number Priority date Publication date Assignee Title
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
UA124961C2 (uk) * 2013-10-04 2021-12-22 Елнілем Фармасьютикалз, Інк. ДВОНИТКОВА РИБОНУКЛЕЇНОВА КИСЛОТА (dsRNA) ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ALAS1
JP7289610B2 (ja) 2014-08-20 2023-06-12 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 修飾二本鎖ｒｎａ剤
EP3207138B1 (fr) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Agents polynucléotidiques de ciblage d'acide aminolévulinique synthase-1 (alas1) et utilisations de ceux-ci
WO2017048843A1 (fr) 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Compositions et méthodes permettant d'inhiber l'expression du gène alas1
MX2019005816A (es) 2016-11-23 2019-10-07 Alnylam Pharmaceuticals Inc Agentes de arn modificados con efecto reducido fuera de la diana.
US20200368268A1 (en) 2018-01-08 2020-11-26 Novartis Ag Immune-enhancing rnas for combination with chimeric antigen receptor therapy
IL295625A (en) * 2020-02-21 2022-10-01 Ionis Pharmaceuticals Inc Methods to download htt expression
EP4413378A1 (fr) 2021-10-06 2024-08-14 Alnylam Pharmaceuticals, Inc. Biomarqueurs de lésion rénale en tant que biomarqueurs pour la porphyrie hépatique aiguë (pha)
WO2024148236A1 (fr) * 2023-01-06 2024-07-11 Memorial Sloan-Kettering Cancer Center Procédés pour améliorer l'efficacité d'une thérapie par interférence arn par ciblage d'alas1/alas2
CN118460654A (zh) * 2024-07-10 2024-08-09 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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