BR112023022284A2 - Composições de irna de protease transmembrana, serina 6 (tmprss6) e métodos de uso da mesma - Google Patents

Composições de irna de protease transmembrana, serina 6 (tmprss6) e métodos de uso da mesma

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Publication number
BR112023022284A2
BR112023022284A2 BR112023022284A BR112023022284A BR112023022284A2 BR 112023022284 A2 BR112023022284 A2 BR 112023022284A2 BR 112023022284 A BR112023022284 A BR 112023022284A BR 112023022284 A BR112023022284 A BR 112023022284A BR 112023022284 A2 BR112023022284 A2 BR 112023022284A2
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Brazil
Prior art keywords
tmprss6
methods
serine
transmembrane protease
thalassemia
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BR112023022284A
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English (en)
Inventor
M Deaton Aimee
P Garfinkel Benjamin
D Mcininch James
Michael Gansner John
K Schlegel Mark
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Alnylam Pharmaceuticals Inc
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Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of BR112023022284A2 publication Critical patent/BR112023022284A2/pt

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    • C12N15/1137—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 enzymes
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    • C12Y304/21109—Matriptase (3.4.21.109)

Abstract

composições de irna de protease transmembrana, serina 6 (tmprss6) e métodos de uso da mesma. a presente invenção refere-se a agentes de rnai, por exemplo, agentes de rna de fita dupla (dsrna), direcionados ao gene da protease transmembrana, serina 6 (tmprss6). a invenção também se refere a métodos de utilização de tais agentes de rnai para inibir a expressão de um gene tmprss6 e a métodos de prevenção e tratamento de um distúrbio associado a tmprss6, por exemplo, um distúrbio associado à sobrecarga de ferro e/ou um distúrbio de eritropoiese ineficaz, por exemplo, hemocromatose hereditária, ß-talassemia (por exemplo, ß-talassemia maior e ß-talassemia intermiédia), policitemia vera, síndrome mielodisplásica, anemias diseritropoiéticas congênitas, deficiência de piruvato quinase, porfiria eritropoiética, doença de parkinson, doença de alzheimer ou ataxia de friedreich.
BR112023022284A 2021-04-26 2022-04-25 Composições de irna de protease transmembrana, serina 6 (tmprss6) e métodos de uso da mesma BR112023022284A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163179607P 2021-04-26 2021-04-26
US202163278227P 2021-11-11 2021-11-11
PCT/US2022/026097 WO2022231999A1 (en) 2021-04-26 2022-04-25 Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof

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BR112023022284A2 true BR112023022284A2 (pt) 2023-12-26

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Country Status (10)

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US (1) US11866710B2 (pt)
EP (1) EP4330392A1 (pt)
KR (1) KR20240001207A (pt)
AU (1) AU2022264478A1 (pt)
BR (1) BR112023022284A2 (pt)
CA (1) CA3216106A1 (pt)
CO (1) CO2023014875A2 (pt)
IL (1) IL307926A (pt)
TW (1) TW202309280A (pt)
WO (1) WO2022231999A1 (pt)

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TW202309280A (zh) 2021-04-26 2023-03-01 美商艾拉倫製藥股份有限公司 跨膜絲胺酸蛋白酶６（ＴＭＰＲＳＳ６）ｉＲＮＡ組成物及其使用方法
TW202400193A (zh) 2022-06-24 2024-01-01 丹麥商諾佛 儂迪克股份有限公司 抑制跨膜絲胺酸蛋白酶６（ｔｍｐｒｓｓ６）表現的組成物及方法

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