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:

23 Jun 2021

Issue date:

14 Oct 2021