Invention Application
- Patent Title: POLYNUCLEOTIDES ENCODING METHYLMALONYL-CoA MUTASE
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Application No.: US16515256Application Date: 2019-07-18
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Publication No.: US20200078314A1Publication Date: 2020-03-12
- Inventor: Paolo Martini , Vladimir Presnyak , Kerry Benenato , Stephen Hoge , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe
- Applicant: ModernaTX, Inc.
- Applicant Address: US MA Cambridge
- Assignee: ModernaTX, Inc.
- Current Assignee: ModernaTX, Inc.
- Current Assignee Address: US MA Cambridge
- Main IPC: A61K9/51
- IPC: A61K9/51 ; C12N15/52 ; A61K48/00 ; A61K38/52 ; A61P43/00 ; A61P3/00 ; C12N9/90 ; A61K9/127

Abstract:
The disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human methylmalonyl-CoA mutase precursor, human methylmalonyl-CoA mutase (MCM) mature form, or functional fragments thereof. In some embodiments, the disclosure includes methods of treating methylmalonic acidemia in a subject in need thereof comprising administering an mRNA encoding an MCM polypeptide.
Public/Granted literature
- US11504337B2 Polynucleotides encoding methylmalonyl-CoA mutase Public/Granted day:2022-11-22
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