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PE20221578A1 - Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas - Google Patents

Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas

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Publication number
PE20221578A1
PE20221578A1 PE2022001900A PE2022001900A PE20221578A1 PE 20221578 A1 PE20221578 A1 PE 20221578A1 PE 2022001900 A PE2022001900 A PE 2022001900A PE 2022001900 A PE2022001900 A PE 2022001900A PE 20221578 A1 PE20221578 A1 PE 20221578A1
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PE
Peru
Prior art keywords
nucleotide
nucleotides
sina
modified
fluoro
Prior art date
Application number
PE2022001900A
Other languages
English (en)
Inventor
Leonid Beigelman
Vivek Kumar Rajwanshi
Markus Hossbach
Rajendra K Pandey
Jin Hong
Laxman Eltepu
Saul Martinez Montero
Costa N Tilani S De
Original Assignee
Aligos Therapeutics 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.)
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Publication date
Application filed by Aligos Therapeutics Inc filed Critical Aligos Therapeutics Inc
Publication of PE20221578A1 publication Critical patent/PE20221578A1/es

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    • 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
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    • 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
    • C12N15/1131—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 viruses
    • A—HUMAN NECESSITIES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • A61P31/20—Antivirals for DNA viruses
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/31—Chemical structure of the backbone
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Abstract

Referida a una molecula de acido nucleico interferente corto (siNA) que comprende: (a) una cadena de sentido que comprende una primera secuencia de nucleotidos que es al menos alrededor de 60 % a 100 % identica a un ARN que corresponde a un gen diana, en donde la primera secuencia de nucleotidos: (i) tiene de 15 a 30 nucleotidos de longitud; y (ii) comprende 15 o mas nucleotidos modificados que se seleccionan independientemente de un nucleotido 2'-O-metilo y un nucleotido 2'-fluoro, en donde al menos un nucleotido modificado es un nucleotido 2'-O-metilo y el nucleotido en la posicion 3, 5, 7, 8, 9, 10, 11, 12, 14, 17 y/o 19 del extremo 5' de la primera secuencia de nucleotidos es un nucleotido 2'-fluoro; y (b) una cadena antisentido que comprende una segunda secuencia de nucleotidos que es al menos alrededor de 60 % a 100 % complementaria con respecto al ARN que corresponde al gen diana, en donde la segunda secuencia de nucleotidos: (i) tiene de 15 a 30 nucleotidos de longitud; y (ii) comprende 15 o mas nucleotidos modificados independientemente seleccionados de un nucleotido 2'-O-metilo y un nucleotido 2'-fluoro, en donde al menos un nucleotido modificado es un nucleotido 2'-O-metilo y al menos un nucleotido modificado es un nucleotido 2'-fluoro. Las moleculas de siNA pueden ser de cadena doble y comprender nucleotidos modificados que se seleccionan de nucleotidos 2'-O-metilo y nucleotidos 2'- fluoro. En la presente se describen ademas moleculas de siNA que comprenden (a) un bloqueador de fosforilacion, un resto conjugado o un casquete del extremo 5' estabilizado; y (b) un acido nucleico interferente corto (siNA).
PE2022001900A 2020-03-06 2021-03-05 Moleculas de acido nucleico interferente corto (sina, por sus siglas en ingles) modificadas y usos de estas PE20221578A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062986150P 2020-03-06 2020-03-06
US202063109196P 2020-11-03 2020-11-03
PCT/US2021/021199 WO2021178885A1 (en) 2020-03-06 2021-03-05 Modified short interfering nucleic acid (sina) molecules and uses thereof

Publications (1)

Publication Number Publication Date
PE20221578A1 true PE20221578A1 (es) 2022-10-06

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US (4) US20220364096A1 (es)
EP (1) EP4121533A1 (es)
JP (1) JP2023515664A (es)
KR (1) KR20220163960A (es)
CN (1) CN115485384A (es)
AU (1) AU2021230601A1 (es)
BR (1) BR112022017287A2 (es)
CA (1) CA3170466A1 (es)
CL (1) CL2022002379A1 (es)
CO (1) CO2022014258A2 (es)
GE (2) GEP20257742B (es)
IL (1) IL296045A (es)
MX (1) MX2022011012A (es)
PE (1) PE20221578A1 (es)
PH (1) PH12022552163A1 (es)
TW (1) TW202208625A (es)
WO (1) WO2021178885A1 (es)

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US12129469B2 (en) 2024-10-29
CL2022002379A1 (es) 2023-07-28
AU2021230601A2 (en) 2022-10-20
PH12022552163A1 (en) 2024-01-29
GEP20257742B (en) 2025-03-10
WO2021178885A1 (en) 2021-09-10
MX2022011012A (es) 2022-10-07
TW202208625A (zh) 2022-03-01
IL296045A (en) 2022-10-01
US20220177888A1 (en) 2022-06-09
US11549110B2 (en) 2023-01-10
BR112022017287A2 (pt) 2022-11-16
CA3170466A1 (en) 2021-09-10
AU2021230601A1 (en) 2022-10-06
KR20220163960A (ko) 2022-12-12
US20230365970A1 (en) 2023-11-16
US20220364096A1 (en) 2022-11-17
US20230332153A1 (en) 2023-10-19
JP2023515664A (ja) 2023-04-13
EP4121533A1 (en) 2023-01-25
CN115485384A (zh) 2022-12-16
GEAP202416053A (en) 2024-10-10
CO2022014258A2 (es) 2022-12-30

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