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AR088911A1 - AGENTES DE ARNi, COMPOSICIONES Y METODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR) - Google Patents

AGENTES DE ARNi, COMPOSICIONES Y METODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR)

Info

Publication number
AR088911A1
AR088911A1 ARP120104346A ARP120104346A AR088911A1 AR 088911 A1 AR088911 A1 AR 088911A1 AR P120104346 A ARP120104346 A AR P120104346A AR P120104346 A ARP120104346 A AR P120104346A AR 088911 A1 AR088911 A1 AR 088911A1
Authority
AR
Argentina
Prior art keywords
chain
ttr
nucleotides
yyy
zzz
Prior art date
Application number
ARP120104346A
Other languages
English (en)
Inventor
G Rajeev Kallanthottathil
Kuchimanchi Satyanarayana
Zimmermann Tracy
Manoharan Muthiah
Maier Martin
Charisse Klaus
Original Assignee
Alnylam Pharmaceuticals
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=48430225&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=AR088911(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Alnylam Pharmaceuticals filed Critical Alnylam Pharmaceuticals
Publication of AR088911A1 publication Critical patent/AR088911A1/es

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    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
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    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/30—Chemical structure
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Abstract

Agentes de ARNi, por ejemplo, agentes de ARNi de doble cadena, dirigidos al gen de transtiretina (TTR) y métodos para usar dichos agentes de ARNi para tratar o prevenir enfermedades asociadas a TTR. Reivindicación 1: Un agente de ARNi de doble cadena, caracterizado porque comprende una cadena orientada en el sentido del marco de lectura que es complementaria de una cadena antisentido, en donde dicha cadena antisentido comprende una región que es la parte complementaria de un ARNm que codifica transtiretina (TTR), en donde cada cadena tiene entre aproximadamente 14 y aproximadamente 30 nucleótidos, en donde dicho agente de ARNi de doble cadena está representado por la fórmula: cadena sentido: 5 nₚ-Na-(XXX)ⁱ-Nb-YYY-Nb-(ZZZ)ʲ-Na-nq 3 cadena antisentido: 3 nₚ-Na-(XXX)ₖ-Nb-YYY-Nb(ZZZ)ₗ-Na-nq 5 en donde: i, j, k y l son cada uno de manera independiente 0 ó 1; p, p, q y q son cada uno de manera independiente 0 - 6; cada Na y Na representa de manera independiente una secuencia de oligonucleótidos que comprende 0 - 25 nucleótidos que están modificados o no modificados o combinaciones de los mismos, donde cada secuencia comprende al menos dos nucleótidos modificados de manera diferente; cada Nb y Nb representa de manera independiente una secuencia de oligonucleótidos que comprende 0 - 10 nucleótidos que están modificados o no modificados o combinaciones de los mismos; cada nₚ, nₚ, nq y nq representa de manera independiente un nucleótido saliente; XXX, YYY, ZZZ, XXX, YYY, y ZZZ representan cada uno de manera independiente un motivo de tres modificaciones idénticas en tres nucleótidos consecutivos; las modificaciones de Nb difieren de la modificación de Y y las modificaciones de Nb difieren de la modificación de Y; y en donde la cadena orientada en el sentido del marco de lectura está conjugada a por lo menos un ligando.
ARP120104346A 2011-11-18 2012-11-19 AGENTES DE ARNi, COMPOSICIONES Y METODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR) AR088911A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161561710P 2011-11-18 2011-11-18
US201261615618P 2012-03-26 2012-03-26
US201261680098P 2012-08-06 2012-08-06

Publications (1)

Publication Number Publication Date
AR088911A1 true AR088911A1 (es) 2014-07-16

Family

ID=48430225

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP120104346A AR088911A1 (es) 2011-11-18 2012-11-19 AGENTES DE ARNi, COMPOSICIONES Y METODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR)

Country Status (38)

Country Link
US (5) US9399775B2 (es)
EP (4) EP2780353B1 (es)
JP (4) JP6165158B2 (es)
KR (4) KR20220045091A (es)
CN (2) CN108186667B (es)
AR (1) AR088911A1 (es)
AU (5) AU2012340159B2 (es)
BR (1) BR112014011896B1 (es)
CA (2) CA2856243C (es)
CL (3) CL2014001291A1 (es)
CO (1) CO7020872A2 (es)
CR (1) CR20140232A (es)
CY (1) CY1123693T1 (es)
DK (1) DK3301177T3 (es)
DO (1) DOP2014000107A (es)
EC (1) ECSP14005606A (es)
ES (1) ES2800065T3 (es)
GT (1) GT201400097A (es)
HK (2) HK1200171A1 (es)
HR (1) HRP20200672T1 (es)
HU (1) HUE048622T2 (es)
IL (6) IL284530B (es)
IN (1) IN2014CN03463A (es)
LT (1) LT3301177T (es)
MX (3) MX348575B (es)
MY (1) MY167390A (es)
NI (1) NI201400046A (es)
PE (1) PE20142362A1 (es)
PH (1) PH12014501106B1 (es)
PL (1) PL3301177T3 (es)
PT (1) PT3301177T (es)
RS (1) RS60414B1 (es)
RU (1) RU2678807C2 (es)
SG (2) SG10201912170WA (es)
SI (1) SI3301177T1 (es)
SM (1) SMT202000308T1 (es)
UA (1) UA118649C2 (es)
WO (1) WO2013075035A1 (es)

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KR102318555B1 (ko) 2020-03-19 2021-10-29 한국과학기술연구원 광소자용 역나노콘과 그 제조방법

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