Compositions and methods for delivering CFTR polypeptides
AssigneeKrystal Biotech, Inc.
InventorsTrevor Parry, Suma Krishnan, Pooja Agarwal
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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.
Claims7
A pharmaceutical composition useful for delivery of one or more polynucleotides encoding a cystic fibrosis transmembrane conductance regulator (CFTR) polypeptide to the airways of a subject comprising:
- (a) a replication defective herpes simplex virus type 1 (HSV-1) comprising a recombinant HSV-1 genome, wherein the recombinant HSV-1 genome comprises the one or more polynucleotides encoding the CFTR polypeptide;
- and
- (b) a pharmaceutically acceptable excipient,
- wherein the pharmaceutical composition is formulated in a vibrating mesh nebulizer,
- wherein the one or more polynucleotides encoding the CFTR polypeptide are operably linked to a promoter,
- wherein the recombinant HSV-1 genome comprises an inactivating mutation in or both copies of the Infected Cell Protein (ICP) 4 herpes simplex virus gene and an inactivating mutation in the ICP22 herpes simplex virus gene, and
- wherein the recombinant HSV-1 genome does not comprise an inactivating mutation in an ICP27 herpes simplex virus gene.
Dependencies are inferred from the claim text.