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公开(公告)号：US20240197825A1
公开(公告)日：2024-06-20
申请号：US17761893
申请日：2020-09-17
Applicant: Translate Bio, Inc.
Inventor： Richard Wooster , Frank DeRosa , Lianne Boeglin , Priyaanka Nanduri , Anusha Dias , Khang Anh Tran
CPC classification number: A61K38/177 , A61K48/0033 , A61K48/005
Abstract: The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding an engineered or mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.
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