Drug Development Pipeline
This program is working to develop a non-viral method to deliver a healthy copy of the CFTR gene into the lung cells of people with CF. This would allow cells to create normally functioning CFTR protein, regardless of an individual's specific CFTR mutation.
This program plans to use extracellular vesicles — bits of cell membrane that naturally bud off from cells to form tiny particles. Once loaded with the gene therapy, these particles can be either inhaled or infused through an IV. This method of delivering healthy CFTR genes shows promise because it harnesses a natural process in the body that is unlikely to trigger an immune system response.
Laboratory studies to develop and test this approach are underway.
This program is sponsored by Carmine Therapeutics and partially funded by the Cystic Fibrosis Foundation.
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