Krystal Biotech, Inc.
COMPOSITIONS AND METHODS FOR DELIVERING CFTR POLYPEPTIDES
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Abstract:
The present disclosure provides recombinant nucleic acids comprising one or more polynucleotides encoding a cystic fibrosis transmembrane conductance regulator (CFTR) polypeptide (e.g., a human CFTR polypeptide); viruses comprising the recombinant nucleic acids; compositions and formulations comprising the recombinant nucleic acids and/or viruses; methods of their use (e.g., for the treatment of a chronic lung disease, such as cystic fibrosis); and articles of manufacture or kits thereof.
Status:
Application
Type:
Utility
Filling date:
7 Feb 2020
Issue date:
13 Aug 2020