Invention Publication
- Patent Title: TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF CODON-OPTIMIZED mRNA ENCODING CFTR
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Application No.: US17449679Application Date: 2021-10-01
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Publication No.: US20240299489A9Publication Date: 2024-09-12
- Inventor: Michael Heartlein , Frank DeRosa , Alan Kimura , Jonathan Abysalh , Anusha Dias , Shrirang Karve , Zarna Patel
- Applicant: Translate Bio, Inc.
- Applicant Address: US MA Lexington
- Assignee: Translate Bio, Inc.
- Current Assignee: Translate Bio, Inc.
- Current Assignee Address: US MA Lexington
- The original application number of the division: US15981757 2018.05.16
- Main IPC: A61K38/17
- IPC: A61K38/17 ; A61K9/00 ; A61K9/127 ; A61K48/00 ; A61P11/12
Abstract:
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.
Public/Granted literature
- US20220160827A1 TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF CODON-OPTIMIZED mRNA ENCODING CFTR Public/Granted day:2022-05-26
Information query
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