Invention Grant
- Patent Title: Polynucleotides encoding α-galactosidase A for the treatment of Fabry disease
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Application No.: US16570351Application Date: 2019-09-13
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Publication No.: US11649461B2Publication Date: 2023-05-16
- Inventor: Paolo Martini , Stephen G. Hoge , Kerry Benenato , Vladimir Presnyak , Iain Mcfadyen , Ellalahewage Sathyajith Kumarasinghe , Xuling Zhu , Lin Tung Guey , Staci Sabnis
- Applicant: ModernaTX, Inc.
- Applicant Address: US MA Cambridge
- Assignee: ModernaTX, Inc.
- Current Assignee: ModernaTX, Inc.
- Current Assignee Address: US MA Cambridge
- Agency: Cooley LLP
- Agent Amy Mandragouras; Ariana D. Harris
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C12N15/67 ; A61K9/51 ; C12N9/40 ; A61P3/00 ; A61K38/47
Abstract:
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.
Public/Granted literature
- US20200149052A1 POLYNUCLEOTIDES ENCODING ALPHA-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE Public/Granted day:2020-05-14
Information query
