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CY2020029I2 - 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
CY2020029I2
CY2020029I2 CY2020029C CY2020029C CY2020029I2 CY 2020029 I2 CY2020029 I2 CY 2020029I2 CY 2020029 C CY2020029 C CY 2020029C CY 2020029 C CY2020029 C CY 2020029C CY 2020029 I2 CY2020029 I2 CY 2020029I2
Authority
CY
Cyprus
Prior art keywords
compositions
methods
gene expression
alas1 gene
inhibiting alas1
Prior art date
Application number
CY2020029C
Other languages
English (en)
Other versions
CY2020029I1 (el
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
Application filed by Alnylam Pharmaceuticals, Inc. filed Critical Alnylam Pharmaceuticals, Inc.
Publication of CY2020029I1 publication Critical patent/CY2020029I1/el
Publication of CY2020029I2 publication Critical patent/CY2020029I2/el

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/04Centrally acting analgesics, e.g. opioids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/14Type of nucleic acid interfering N.A.
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/30Chemical structure
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    • C12N2310/3521Methyl
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    • C12N2310/30Chemical structure
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    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
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    • C12Q2600/00Oligonucleotides characterized by their use
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    • C12YENZYMES
    • C12Y203/00Acyltransferases (2.3)
    • C12Y203/01Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • C12Y203/010375-Aminolevulinate synthase (2.3.1.37)

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CY2020029C 2013-10-04 2020-08-18 Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου alas1 CY2020029I2 (el)

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

Publications (2)

Publication Number Publication Date
CY2020029I1 CY2020029I1 (el) 2020-11-25
CY2020029I2 true CY2020029I2 (el) 2020-11-25

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CY20201100482T CY1122975T1 (el) 2013-10-04 2020-05-27 Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου alas1
CY2020029C CY2020029I2 (el) 2013-10-04 2020-08-18 Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου alas1

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Country Status (38)

Country Link
US (3) US10119143B2 (el)
EP (2) EP3052628B1 (el)
JP (3) JP6613227B2 (el)
KR (3) KR20220159478A (el)
CN (1) CN105980559B (el)
AU (3) AU2014331604B2 (el)
BR (2) BR112016007226B1 (el)
CA (2) CA2925357C (el)
CL (2) CL2016000772A1 (el)
CR (1) CR20160195A (el)
CY (2) CY1122975T1 (el)
DK (1) DK3052628T3 (el)
DO (2) DOP2016000073A (el)
EA (1) EA036477B1 (el)
ES (1) ES2804510T3 (el)
GT (1) GT201600066A (el)
HK (1) HK1221738A1 (el)
HR (1) HRP20200822T1 (el)
HU (2) HUE049227T2 (el)
IL (3) IL302726A (el)
LT (2) LT3052628T (el)
MX (2) MX2016004319A (el)
MY (2) MY197646A (el)
NL (1) NL301061I2 (el)
NO (1) NO2020029I1 (el)
NZ (2) NZ757749A (el)
PE (2) PE20161130A1 (el)
PH (1) PH12016500574A1 (el)
PL (1) PL3052628T3 (el)
PT (1) PT3052628T (el)
SG (2) SG10201910929QA (el)
SI (1) SI3052628T1 (el)
SM (1) SMT202000271T1 (el)
TN (1) TN2016000114A1 (el)
TW (3) TWI768330B (el)
UA (1) UA124961C2 (el)
WO (1) WO2015051318A1 (el)
ZA (2) ZA201602931B (el)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
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
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
SG10201913786SA (en) 2016-11-23 2020-03-30 Alnylam Pharmaceuticals Inc Modified rna agents with reduced off-target effect
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
JP2023515466A (ja) * 2020-02-21 2023-04-13 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 Httの発現を低下させる方法
WO2023060064A1 (en) 2021-10-06 2023-04-13 Alnylam Pharmaceuticals, Inc. Renal injury biomarkers as biomarkers for acute hepatic porphyria (ahp)
WO2024148236A1 (en) * 2023-01-06 2024-07-11 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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