发明申请
- 专利标题： TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF CODON-OPTIMIZED mRNA ENCODING CFTR
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申请号： US17449679申请日： 2021-10-01
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公开(公告)号： US20220160827A1公开(公告)日： 2022-05-26
- 发明人: Michael Heartlein , Frank DeRosa , Alan Kimura , Jonathan Abysalh , Anusha Dias , Shrirang Karve , Zarna Patel
- 申请人： Translate Bio, Inc.
- 申请人地址： US MA Lexington
- 专利权人： Translate Bio, Inc.
- 当前专利权人： Translate Bio, Inc.
- 当前专利权人地址： US MA Lexington
- 主分类号： A61K38/17
- IPC分类号： A61K38/17 ; A61P11/12 ; A61K9/127 ; A61K9/00 ; A61K48/00
摘要：
The present invention provides, among other things, methods of treating cystic fibrosis, comprising a step of administering to a subject in need of treatment a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA encoding the CFTR protein comprises a polynucleotide sequence at least 80% identical to SEQ ID NO: 1, wherein the mRNA is at a concentration of at least 0.4 mg/mL, and wherein the step of administering comprises inhalation.
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