METHODS AND COMPOSITIONS FOR TREATING CYSTIC FIBROSIS
The present disclosure provides a method of treating diseases or disorders associated with CFTR protein dysfunction, including Cystic Fibrosis, by administering stable, long-lasting vasoactive intestinal peptide therapeutic agents. These agents include one or more elastin-like peptides and can be administered at a low-dose.
1 - 21 . (canceled)
22 . A method for treating cystic fibrosis comprising subcutaneously administering to a patient in need thereof a pharmaceutical composition comprising the amino acid sequence of SEQ ID NO: 15.
23 . The method of claim 22 , wherein the pharmaceutical composition is formulated for systemic delivery.
24 . The method of claim 22 , wherein the pharmaceutical composition is formulated for sustained release.
25 . The method of claim 22 , wherein the pharmaceutical composition is administered with one or more additional cystic fibrosis therapies or therapies to treat disorders associated with CFTR protein dysfunction.
26 . The method of claim 25 , wherein the one or more additional cystic fibrosis therapies is selected from the group consisting of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) potentiators, CFTR correctors, nonsense mutation readthrough agents, CFTR production correctors, read-through agents, small molecule ion channel agents, osmotic agents, gene therapy, RNA repair, soluble guanylate cyclase stimulators, S-nitrosoglutathione reductase inhibitors, DNase, antibiotics, antifungals, mucolytics, bronchodilators, nitric oxide, anticholinergics, nonsteroidal anti-inflammatory drugs (NSAIDs), membrane stabilizers, corticosteroids, and enzyme replacement therapy.
27 . The method of claim 26 , wherein the CFTR potentiator is selected from the group consisting of ivacaftor (VX-770) and QBW251.
28 . The method of claim 26 , wherein the CFTR corrector is selected from the group consisting of lumacaftor (VX-809) and VX-661.
29 . The method of claim 22 , wherein the patient has at least one or more mutations in a CFTR gene.
30 . The method of claim 29 , wherein the patient is homozygous for the one or more mutations in the CFTR gene.
31 . The method of claim 29 , wherein the patient is heterozygous for the one or more mutations in the CFTR gene.
32 . The method of claim 29 , wherein the patient has an F508del mutation.