Invention Grant
- Patent Title: Polynucleotides encoding citrin for the treatment of Citrullinemia type 2
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Application No.: US16302360Application Date: 2017-05-18
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Publication No.: US10993918B2Publication Date: 2021-05-04
- Inventor: Paolo Martini , Stephen Hoge , Kerry Benenato , Vladimir Presnyak , Iain McFadyen , Ellalahewage Sathyajith Kumarasinghe , Jingsong Cao , 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: Fish & Richardson P.C.
- International Application: PCT/US2017/033421 WO 20170518
- International Announcement: WO2017/201349 WO 20171123
- Main IPC: A61K9/51
- IPC: A61K9/51 ; C07K14/47 ; A61P13/00 ; A61K9/00 ; A61K38/00
Abstract:
The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. 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 Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.
Public/Granted literature
- US20190175517A1 POLYNUCLEOTIDES ENCODING CITRIN FOR THE TREATMENT OF CITRULLINEMIA TYPE 2 Public/Granted day:2019-06-13
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