BR112016020618A8 - oligômero antissentido, composição farmacêutica, e, uso de um oligômero antissentido - Google Patents

oligômero antissentido, composição farmacêutica, e, uso de um oligômero antissentido

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Publication number
BR112016020618A8
BR112016020618A8 BR112016020618A BR112016020618A BR112016020618A8 BR 112016020618 A8 BR112016020618 A8 BR 112016020618A8 BR 112016020618 A BR112016020618 A BR 112016020618A BR 112016020618 A BR112016020618 A BR 112016020618A BR 112016020618 A8 BR112016020618 A8 BR 112016020618A8
Authority
BR
Brazil
Prior art keywords
antisense oligomer
pharmaceutical composition
allows
exon
human
Prior art date
Application number
BR112016020618A
Other languages
English (en)
Other versions
BR112016020618A2 (pt
BR112016020618B1 (pt
Inventor
Tatsushi Wakayama
Haruna Seo
Youhei Satou
Shin'ichi Takeda
Tetsuya Nagata
Original Assignee
Nippon Shinyaku Co Ltd
Nat Center Neurology & Psychiatry
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 Nippon Shinyaku Co Ltd, Nat Center Neurology & Psychiatry filed Critical Nippon Shinyaku Co Ltd
Publication of BR112016020618A2 publication Critical patent/BR112016020618A2/pt
Publication of BR112016020618A8 publication Critical patent/BR112016020618A8/pt
Publication of BR112016020618B1 publication Critical patent/BR112016020618B1/pt

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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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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
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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    • C12N2310/321—2'-O-R Modification
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    • C12N2310/32—Chemical structure of the sugar
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    • 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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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
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    • 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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    • 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
    • C12N2310/3535—Nitrogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Abstract

oligômero antissentido, composição farmacêutica e método para tratamento de distrofia muscular, e, uso de um oligômero antissentido. é provida uma droga que permite o salto de éxon 51 altamente eficiente no gene da distrofia humana. a presente invenção provê um oligômero antissentido que permite que o éxon 51 no gene da distrofia humana seja saltado.
BR112016020618-5A 2014-03-12 2015-03-11 Oligômero antissentido, composição farmacêutica, e, uso de um oligômero antissentido BR112016020618B1 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2014-048897 2014-03-12
JP2014048897 2014-03-12
PCT/JP2015/057180 WO2015137409A1 (ja) 2014-03-12 2015-03-11 アンチセンス核酸

Publications (3)

Publication Number Publication Date
BR112016020618A2 BR112016020618A2 (pt) 2017-08-15
BR112016020618A8 true BR112016020618A8 (pt) 2021-07-13
BR112016020618B1 BR112016020618B1 (pt) 2023-04-04

Family

ID=54071850

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Application Number Title Priority Date Filing Date
BR112016020618-5A BR112016020618B1 (pt) 2014-03-12 2015-03-11 Oligômero antissentido, composição farmacêutica, e, uso de um oligômero antissentido

Country Status (30)

Country Link
US (4) US9988629B2 (pt)
EP (2) EP3118311B1 (pt)
JP (1) JP6606488B2 (pt)
KR (1) KR102335801B1 (pt)
CN (2) CN106459955B (pt)
AU (2) AU2015227733B2 (pt)
BR (1) BR112016020618B1 (pt)
CA (1) CA2939948A1 (pt)
CY (1) CY1121322T1 (pt)
DK (1) DK3118311T3 (pt)
ES (1) ES2710802T3 (pt)
HR (1) HRP20190235T1 (pt)
HU (1) HUE042283T2 (pt)
IL (1) IL247663B (pt)
LT (1) LT3118311T (pt)
MX (1) MX2016011538A (pt)
MY (1) MY193708A (pt)
NZ (1) NZ724836A (pt)
PH (1) PH12016501761B1 (pt)
PL (1) PL3118311T3 (pt)
PT (1) PT3118311T (pt)
RS (1) RS58573B1 (pt)
RU (2) RU2730681C2 (pt)
SG (1) SG11201607095RA (pt)
SI (1) SI3118311T1 (pt)
TR (1) TR201901939T4 (pt)
TW (2) TWI672314B (pt)
UA (1) UA120849C2 (pt)
WO (1) WO2015137409A1 (pt)
ZA (1) ZA201605864B (pt)

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US20180179538A1 (en) 2018-06-28
AU2020200679A1 (en) 2020-02-20
EP3118311A4 (en) 2017-10-11
WO2015137409A1 (ja) 2015-09-17
RU2702424C2 (ru) 2019-10-08
US9988629B2 (en) 2018-06-05
JPWO2015137409A1 (ja) 2017-04-06
PH12016501761A1 (en) 2016-11-07
SI3118311T1 (sl) 2019-05-31
UA120849C2 (uk) 2020-02-25
EP3118311A1 (en) 2017-01-18
RS58573B1 (sr) 2019-05-31
LT3118311T (lt) 2019-04-10
CN106459955A (zh) 2017-02-22
EP3118311B1 (en) 2018-12-26
CY1121322T1 (el) 2020-05-29
RU2019130513A (ru) 2019-10-10
JP6606488B2 (ja) 2019-11-13
KR20160132056A (ko) 2016-11-16
ZA201605864B (en) 2018-12-19
MX2016011538A (es) 2017-04-13
US11053497B2 (en) 2021-07-06
IL247663B (en) 2020-06-30
RU2019130513A3 (pt) 2020-03-13
RU2730681C2 (ru) 2020-08-24
EP3514234A1 (en) 2019-07-24
PH12016501761B1 (en) 2016-11-07
AU2015227733A1 (en) 2016-10-20
BR112016020618A2 (pt) 2017-08-15
US20220162604A1 (en) 2022-05-26
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AU2015227733B2 (en) 2019-10-31
CA2939948A1 (en) 2015-09-17
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PL3118311T3 (pl) 2019-09-30
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PT3118311T (pt) 2019-02-15
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SG11201607095RA (en) 2016-10-28
IL247663A0 (en) 2016-11-30
TWI672314B (zh) 2019-09-21
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US20170067048A1 (en) 2017-03-09
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