ModernaTX, Inc.;Seattle Children's Hospital d/b/a Seattle Children's Research Institute
发明人:
Raj Rajendran,Paolo G. V. Martini,Carol H. Miao
申请号：
US16621514
公开号：
US20200268666A1
申请日:
2018.06.13
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The invention relates to mRNA therapy for the treatment of Hemophilia A. mRNAs for use in the invention, when administered in vivo, encode Factor VIII, isoforms thereof, functional fragments thereof, and fusion proteins comprising Factor VIII. 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 Factor VIII expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient Factor VII I activity in subjects.
