- 专利标题： Polynucleotides encoding α-galactosidase A for the treatment of Fabry disease
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申请号： US16570351申请日： 2019-09-13
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公开(公告)号： US11649461B2公开(公告)日： 2023-05-16
- 发明人: Paolo Martini , Stephen G. Hoge , Kerry Benenato , Vladimir Presnyak , Iain Mcfadyen , Ellalahewage Sathyajith Kumarasinghe , Xuling Zhu , Lin Tung Guey , Staci Sabnis
- 申请人： ModernaTX, Inc.
- 申请人地址： US MA Cambridge
- 专利权人： ModernaTX, Inc.
- 当前专利权人： ModernaTX, Inc.
- 当前专利权人地址： US MA Cambridge
- 代理机构： Cooley LLP
- 代理商 Amy Mandragouras; Ariana D. Harris
- 主分类号： A61K48/00
- IPC分类号： A61K48/00 ; C12N15/67 ; A61K9/51 ; C12N9/40 ; A61P3/00 ; A61K38/47
摘要：
The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
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