JP7689110B2 - 脊髄性筋萎縮症のための併用療法 - Google Patents

脊髄性筋萎縮症のための併用療法 Download PDF

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JP7689110B2
JP7689110B2 JP2022509053A JP2022509053A JP7689110B2 JP 7689110 B2 JP7689110 B2 JP 7689110B2 JP 2022509053 A JP2022509053 A JP 2022509053A JP 2022509053 A JP2022509053 A JP 2022509053A JP 7689110 B2 JP7689110 B2 JP 7689110B2
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アレクサンダー マッキャンベル，
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Biogen MA Inc
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    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A—HUMAN NECESSITIES
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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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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JP2022509053A 2019-08-15 2020-08-14 脊髄性筋萎縮症のための併用療法 Active JP7689110B2 (ja)

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US201962887579P 2019-08-15 2019-08-15
US62/887,579 2019-08-15
PCT/US2020/046546 WO2021030766A1 (en) 2019-08-15 2020-08-14 Combination therapy for spinal muscular atrophy

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JP2022544538A JP2022544538A (ja) 2022-10-19
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JP7689110B2 true JP7689110B2 (ja) 2025-06-05

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US (1) US20220280548A1 (https=)
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Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250206783A1 (en) * 2022-03-21 2025-06-26 Capsigen Inc. Transcription-dependent directed evolution of aav capsids having enhanced tropism
EP4601651A1 (en) * 2022-10-14 2025-08-20 Genentech, Inc. Methods for treating spinal muscular atrophy
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treating spinal muscular atrophy
CN118045206B (zh) * 2024-04-12 2024-07-05 四川至善唯新生物科技有限公司 一种治疗脊髓型肌肉萎缩的药物组合物及其用途

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012530715A (ja) 2009-06-17 2012-12-06 アイシス ファーマシューティカルズ， インコーポレーテッド 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法
WO2014178863A1 (en) 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
JP2018533605A (ja) 2015-11-12 2018-11-15 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 脊髄性筋萎縮症を処置するための組成物

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013068441A1 (en) * 2011-11-11 2013-05-16 Santaris Pharma A/S Compounds for the modulation of smn2 splicing
SG11201407483YA (en) * 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
MX2018007234A (es) * 2015-12-14 2018-11-09 Univ Pennsylvania Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal.
EP3484524B1 (en) * 2016-07-15 2022-11-09 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of smn2
US20210308281A1 (en) * 2018-08-15 2021-10-07 Biogen Ma Inc. Combination therapy for spinal muscular atrophy

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2012530715A (ja) 2009-06-17 2012-12-06 アイシス ファーマシューティカルズ， インコーポレーテッド 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法
WO2014178863A1 (en) 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
JP2018533605A (ja) 2015-11-12 2018-11-15 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 脊髄性筋萎縮症を処置するための組成物

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Journal of Medicinal Chemistry，2018年，Vol.61, No.24，p.11021－11036

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WO2021030766A1 (en) 2021-02-18
JP2025122671A (ja) 2025-08-21
EP4013387A4 (en) 2023-09-27
JP2022544538A (ja) 2022-10-19
WO2021030766A8 (en) 2021-03-18
EP4013387A1 (en) 2022-06-22
US20220280548A1 (en) 2022-09-08

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