Functional porosome manipulation
AssigneePorosome Therapeutics, Inc.
InventorsBhanu Pratap Jena, Won Jin Cho
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Abstract
This disclosure provides a porosome composition for treating cystic fibrosis comprising an isolated porosome comprising a WT-CFTR protein and a pharmaceutically acceptable excipient. The porosomes can be porosomes isolated from human epithelial cells and may also contain additional CFTR associated proteins, such as SNAP-23 or IQGAP1. The composition can be a liquid or solid (fine granule) composition suitable for oral or nasal inhalation. Nebulizer compositions are particularly provided. The disclosure also provides methods of treating cystic fibrosis comprising administering a composition of the disclosure to a patient in need of such treatment.
Claims23
A porosome composition for treating cystic fibrosis comprising (i) an isolated porosome comprising a functional CFTR protein and (ii) a pharmaceutically acceptable excipient.
A method of treating cystic fibrosis in a patient comprising
- (i) Solubilizing cells comprising functional CFTR containing porosomes to provide solubilized cells,
- (ii) Isolating the functional CFTR containing porosomes from the solubilized cells to provide isolated functional CFTR containing porosomes;
- (iii) administering a therapeutically effective amount of the isolated functional CFTR containing porosomes to the patient.
A method of improving CFTR function in a patient comprising
- reconstituting a porosome complex comprising a functional CFTR protein into a respiratory epithelial cell of the patient.
Dependencies are inferred from the claim text.