IL313082B1 - Transthyretin (TTR) iRNA compositions and methods of using them for treating or preventing TTR-related diseases - Google Patents

Transthyretin (TTR) iRNA compositions and methods of using them for treating or preventing TTR-related diseases

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Publication number
IL313082B1
IL313082B1 IL313082A IL31308224A IL313082B1 IL 313082 B1 IL313082 B1 IL 313082B1 IL 313082 A IL313082 A IL 313082A IL 31308224 A IL31308224 A IL 31308224A IL 313082 B1 IL313082 B1 IL 313082B1
Authority
IL
Israel
Prior art keywords
double stranded
rnai agent
ttr
nucleotides
salt
Prior art date
Application number
IL313082A
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English (en)
Hebrew (he)
Other versions
IL313082A (en
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 IL313082A publication Critical patent/IL313082A/en
Publication of IL313082B1 publication Critical patent/IL313082B1/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
    • 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
    • A—HUMAN NECESSITIES
    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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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
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic 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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    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—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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    • C12Q2600/00—Oligonucleotides characterized by their use
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IL313082A 2015-07-31 2016-07-28 Transthyretin (TTR) iRNA compositions and methods of using them for treating or preventing TTR-related diseases IL313082B1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562199563P 2015-07-31 2015-07-31
US201662287518P 2016-01-27 2016-01-27
PCT/US2016/044359 WO2017023660A1 (en) 2015-07-31 2016-07-28 TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES

Publications (2)

Publication Number Publication Date
IL313082A IL313082A (en) 2024-07-01
IL313082B1 true IL313082B1 (en) 2026-02-01

Family

ID=56682264

Family Applications (4)

Application Number Title Priority Date Filing Date
IL313082A IL313082B1 (en) 2015-07-31 2016-07-28 Transthyretin (TTR) iRNA compositions and methods of using them for treating or preventing TTR-related diseases
IL296476A IL296476B2 (en) 2015-07-31 2016-07-28 TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES
IL325313A IL325313A (en) 2015-07-31 2016-07-28 Transthyretin (TTR) iRNA compositions and methods of using them for treating or preventing TTR-related diseases
IL256614A IL256614B2 (en) 2015-07-31 2017-12-27 Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated diseases

Family Applications After (3)

Application Number Title Priority Date Filing Date
IL296476A IL296476B2 (en) 2015-07-31 2016-07-28 TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES
IL325313A IL325313A (en) 2015-07-31 2016-07-28 Transthyretin (TTR) iRNA compositions and methods of using them for treating or preventing TTR-related diseases
IL256614A IL256614B2 (en) 2015-07-31 2017-12-27 Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated diseases

Country Status (35)

Country Link
US (5) US10208307B2 (2)
EP (1) EP3329002B1 (2)
JP (4) JP6896703B2 (2)
KR (2) KR20240074895A (2)
CN (2) CN108138182B (2)
BR (1) BR112018000542B1 (2)
CA (2) CA3301698A1 (2)
CL (1) CL2018000198A1 (2)
CY (1) CY1124545T1 (2)
DK (1) DK3329002T3 (2)
EA (1) EA201890394A1 (2)
ES (1) ES2842300T3 (2)
FI (1) FIC20230006I1 (2)
FR (1) FR23C1008I2 (2)
HK (1) HK1256683A1 (2)
HR (1) HRP20202082T1 (2)
HU (2) HUE051998T2 (2)
IL (4) IL313082B1 (2)
LT (2) LT3329002T (2)
LU (1) LUC00297I2 (2)
MA (1) MA43335B1 (2)
MX (2) MX2018000981A (2)
MY (1) MY195720A (2)
NL (1) NL301216I2 (2)
NO (1) NO2023007I1 (2)
PL (1) PL3329002T3 (2)
PT (1) PT3329002T (2)
RS (1) RS61297B1 (2)
SG (1) SG10201912341SA (2)
SI (1) SI3329002T1 (2)
SM (1) SMT202100020T1 (2)
TW (4) TWI807302B (2)
UA (1) UA126276C2 (2)
WO (1) WO2017023660A1 (2)
ZA (2) ZA201800571B (2)

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RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
PL2563920T3 (pl) 2010-04-29 2017-08-31 Ionis Pharmaceuticals, Inc. Modulacja ekspresji transtyretyny
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
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EP3730618A1 (en) 2011-11-18 2020-10-28 Alnylam Pharmaceuticals, Inc. Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
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PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
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JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
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