BR112021024764A2 - Oligonucleotídeo antissenso capaz de alterar o splicing de pré-mrna de dux4 - Google Patents

Oligonucleotídeo antissenso capaz de alterar o splicing de pré-mrna de dux4

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Publication number
BR112021024764A2
BR112021024764A2 BR112021024764A BR112021024764A BR112021024764A2 BR 112021024764 A2 BR112021024764 A2 BR 112021024764A2 BR 112021024764 A BR112021024764 A BR 112021024764A BR 112021024764 A BR112021024764 A BR 112021024764A BR 112021024764 A2 BR112021024764 A2 BR 112021024764A2
Authority
BR
Brazil
Prior art keywords
dux4
antisense oligonucleotide
mrna splicing
oligonucleotide capable
altering
Prior art date
Application number
BR112021024764A
Other languages
English (en)
Inventor
Akifumi Nakamura
Hiroaki Mitsuhashi
Makoto Koizumi
Takahiro Katagiri
Original Assignee
Daiichi Sankyo Co Ltd
Tokai Univ Educational System
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 Daiichi Sankyo Co Ltd, Tokai Univ Educational System filed Critical Daiichi Sankyo Co Ltd
Publication of BR112021024764A2 publication Critical patent/BR112021024764A2/pt

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    • 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
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    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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Abstract

oligonucleotídeo antissenso capaz de alterar o splicing de pré-mrna de dux4. a presente invenção refere-se a um método para tratar a distrofia muscular fascio-escápulo-umeral. um oligonucleotídeo que pode alterar o splicing do gene de dux4 de dux4-fl para dux4-s e que é composto por 15 a 30 nucleotídeos e compreende uma sequência de nucleotídeos complementar a uma região que está entre os nucleotídeos nos. 502 a 556 ou 578 a 612 do mrna de dux4-fl, que compreende a sequência de nucleotídeos representada pela seq id no: 1, em que a terminação 5' e/ou a terminação 3' podem ser quimicamente modificadas; ou sal farmaceuticamente aceitável do mesmo. um fármaco (por exemplo, um agente terapêutico para a distrofia muscular fascio-escápulo-umeral) que compreende o oligonucleotídeo ou sal farmaceuticamente aceitável do mesmo.
BR112021024764A 2019-07-12 2020-07-10 Oligonucleotídeo antissenso capaz de alterar o splicing de pré-mrna de dux4 BR112021024764A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2019129735 2019-07-12
PCT/JP2020/026950 WO2021010301A1 (ja) 2019-07-12 2020-07-10 ＤＵＸ４ ｐｒｅ－ｍＲＮＡのスプライシングを変化させるアンチセンスオリゴヌクレオチド

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BR112021024764A2 true BR112021024764A2 (pt) 2022-04-19

Family

ID=74210830

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Country Status (11)

Country Link
US (1) US20220364086A1 (pt)
EP (1) EP3998108A1 (pt)
JP (1) JPWO2021010301A1 (pt)
KR (1) KR20220032004A (pt)
CN (1) CN114026234A (pt)
AU (1) AU2020313255A1 (pt)
BR (1) BR112021024764A2 (pt)
CA (1) CA3142925A1 (pt)
IL (1) IL288596A (pt)
TW (1) TW202117015A (pt)
WO (1) WO2021010301A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4127175A2 (en) 2020-04-02 2023-02-08 Mirecule, Inc. Targeted inhibition using engineered oligonucleotides

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH0787982A (ja) 1993-01-29 1995-04-04 Sankyo Co Ltd 修飾オリゴデオキシリボヌクレオチド
US6096881A (en) 1997-05-30 2000-08-01 Hybridon, Inc. Sulfur transfer reagents for oligonucleotide synthesis
EP2341058A3 (en) 1997-09-12 2011-11-23 Exiqon A/S Oligonucleotide Analogues
NZ513402A (en) 1999-02-12 2003-06-30 Sankyo Co Novel nucleosides and oligonucleotide analogues
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
JP6270742B2 (ja) 2013-01-10 2018-01-31 塩野義製薬株式会社 架橋型核酸誘導体の製造方法
EP3420109A4 (en) * 2016-02-26 2019-11-06 Research Institute at Nationwide Children's Hospital RECOMBINANT VIRUS PRODUCTS AND METHOD OF INDUCING DUX4 EXON SKIPPING
MA45290A (fr) 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
US11518995B2 (en) * 2017-09-19 2022-12-06 Children's National Medical Center Gapmers and methods of using the same for the treatment of muscular dystrophy
JP2019129735A (ja) 2018-01-30 2019-08-08 株式会社ＧＩＳｕｐｐｌｙ 有害動物の捕獲支援システム

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Publication number Publication date
CA3142925A1 (en) 2021-01-21
KR20220032004A (ko) 2022-03-15
CN114026234A (zh) 2022-02-08
WO2021010301A1 (ja) 2021-01-21
US20220364086A1 (en) 2022-11-17
JPWO2021010301A1 (pt) 2021-01-21
IL288596A (en) 2022-02-01
AU2020313255A1 (en) 2022-01-06
EP3998108A1 (en) 2022-05-18
TW202117015A (zh) 2021-05-01

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