Drug Development Pipeline
This program is developing a novel approach to deliver a gene therapy to the lung cells of people with CF. Researchers are exploring how to engineer bacteriophage — specialized viruses that typically target bacteria — to deliver the CFTR gene to people with CF. Researchers believe phage-derived particles might be effective because they are unlikely to trigger an immune response, allowing the gene therapy to be re-dosed when needed. Other advantages of this approach include the potential to target specific organs with full-length human genes.
If successful, this gene delivery vehicle would deliver a healthy CFTR gene to lung cells, which would allow the cells to produce functional CFTR protein. This approach has the potential to provide a treatment for all people living with CF, regardless of mutation.
Laboratory studies to develop and test this potential therapy are underway.
This program is sponsored by Gensaic Inc. and partially funded by the Cystic Fibrosis Foundation.
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