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.
Claims18
A method of providing prophylactic, palliative, or therapeutic relief of one or more signs or symptoms of cystic fibrosis in a subject in need thereof, the method comprising administering to the subject an effective amount of a pharmaceutical composition comprising:
- (a) a herpes virus comprising a recombinant herpes virus genome, wherein the recombinant herpes virus genome comprises one or more polynucleotides encoding a CFTR polypeptide;
- and
- (b) a pharmaceutically acceptable excipient,
- wherein the one or more polynucleotides encoding a CFTR polypeptide are operably linked to a promoter,
- wherein the one or more polynucleotides encoding a CFTR polypeptide are expressed from the recombinant herpes virus genome to effect a measurable improvement in or prevention of the one or more signs or symptoms of cystic fibrosis, and
- wherein the pharmaceutical composition is administered to the subject using a nebulizer.
A method of reducing or inhibiting progressive lung destruction in a subject in need thereof, the method comprising administering to the subject an effective amount of a pharmaceutical composition comprising:
- (a) a herpes virus comprising a recombinant herpes virus genome, wherein the recombinant herpes virus genome comprises one or more polynucleotides encoding a CFTR polypeptide;
- and
- (b) a pharmaceutically acceptable excipient, wherein the one or more polynucleotides encoding a CFTR polypeptide are operably linked to a promoter,
- wherein the one or more polynucleotides encoding a CFTR polypeptide are expressed from the recombinant herpes virus genome to reduce or inhibit the progressive lung destruction, and
- wherein the pharmaceutical composition is administered to the subject using a nebulizer.
Dependencies are inferred from the claim text.