MX2021009135A - Oligonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa. - Google Patents

Oligonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa.

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Publication number
MX2021009135A
MX2021009135A MX2021009135A MX2021009135A MX2021009135A MX 2021009135 A MX2021009135 A MX 2021009135A MX 2021009135 A MX2021009135 A MX 2021009135A MX 2021009135 A MX2021009135 A MX 2021009135A MX 2021009135 A MX2021009135 A MX 2021009135A
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MX
Mexico
Prior art keywords
therapy
epidermolysis bullosa
oligonucleotide
oligonucleotides
col7a1 exon
Prior art date
Application number
MX2021009135A
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English (en)
Inventor
Elisabeth Marlene Haisma
Marko Potman
Wouter Beumer
Vera Brinks
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Wings Therapeutics Inc
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Publication date
Application filed by Wings Therapeutics Inc filed Critical Wings Therapeutics Inc
Publication of MX2021009135A publication Critical patent/MX2021009135A/es

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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C—CHEMISTRY; METALLURGY
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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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
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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
    • C12N15/1138—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 receptors or cell surface proteins
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3521—Methyl
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
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Abstract

Oligonucleótidos antisentido capaces de prevenir o reducir la inclusión del exón 73 en el mRNA de COL7A humano son caracterizados en varias formas: (a) la secuencia del oligonucleótido incluye cuando mucho dos secuencias de CpG; (b) el oligonucleótido tiene una longitud de no más de 24 nucleótidos; (c) el oligonucleótido es capaz de templarse al elemento (unión SRp40/SC35 / ESE) en el exón 73. Estos oligonucleótidos pueden ser útilmente oligorribonucleótidos con enlaces internucleosídicos modificados, por ejemplo, enlaces de fosforotioato.
MX2021009135A 2015-03-11 2017-09-08 Oligonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa. MX2021009135A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB1504124.7A GB201504124D0 (en) 2015-03-11 2015-03-11 Oligonucleotides

Publications (1)

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MX2021009135A true MX2021009135A (es) 2021-09-10

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MX2017011599A MX2017011599A (es) 2015-03-11 2016-03-11 Oigonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa.
MX2021009135A MX2021009135A (es) 2015-03-11 2017-09-08 Oligonucleotidos que igualan exon 73 de col7a1 para terapia de epidermolisis bullosa.

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US (2) US10563198B2 (es)
EP (2) EP3763816A1 (es)
JP (2) JP6929783B2 (es)
KR (1) KR20170120617A (es)
CN (1) CN107567498B (es)
AU (1) AU2016231067B2 (es)
BR (1) BR112017019225A2 (es)
CA (1) CA2978375A1 (es)
DK (1) DK3268474T3 (es)
EA (1) EA035732B1 (es)
ES (1) ES2803651T3 (es)
GB (1) GB201504124D0 (es)
HR (1) HRP20200944T1 (es)
HU (1) HUE050898T2 (es)
IL (1) IL254173B (es)
MX (2) MX2017011599A (es)
PL (1) PL3268474T3 (es)
PT (1) PT3268474T (es)
SI (1) SI3268474T1 (es)
WO (1) WO2016142538A1 (es)

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WO2024080311A1 (ja) * 2022-10-12 2024-04-18 昌和 栗田 遺伝子導入法、遺伝子治療法、及び組織再生法
CN116555349A (zh) * 2023-01-09 2023-08-08 中吉智药(南京)生物技术有限公司 一种腺相关病毒载体及其构建方法与应用

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Publication number Publication date
JP7188810B2 (ja) 2022-12-13
PT3268474T (pt) 2020-06-17
EP3268474B1 (en) 2020-05-27
US10563198B2 (en) 2020-02-18
EA201791986A1 (ru) 2018-01-31
CN107567498A (zh) 2018-01-09
NZ736083A (en) 2021-08-27
EP3763816A1 (en) 2021-01-13
GB201504124D0 (en) 2015-04-22
IL254173A0 (en) 2017-10-31
US20200399638A1 (en) 2020-12-24
ES2803651T3 (es) 2021-01-28
US20180216106A1 (en) 2018-08-02
EP3268474A1 (en) 2018-01-17
WO2016142538A1 (en) 2016-09-15
KR20170120617A (ko) 2017-10-31
HRP20200944T1 (hr) 2020-09-18
US11352626B2 (en) 2022-06-07
CA2978375A1 (en) 2016-09-15
JP2022000020A (ja) 2022-01-04
BR112017019225A2 (pt) 2018-05-08
JP2018509903A (ja) 2018-04-12
AU2016231067B2 (en) 2022-04-21
AU2016231067A1 (en) 2017-10-26
JP6929783B2 (ja) 2021-09-01
CN107567498B (zh) 2021-09-21
DK3268474T3 (da) 2020-06-15
HUE050898T2 (hu) 2021-01-28
EA035732B1 (ru) 2020-07-31
PL3268474T3 (pl) 2020-08-24
IL254173B (en) 2022-03-01
SI3268474T1 (sl) 2020-07-31
MX2017011599A (es) 2018-06-15

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