Page Title
Drug Development Pipeline
Carmine Therapeutics
Status
Pre-clinicalTherapeutic Approach
Genetic Therapy
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.
Status
Laboratory studies to develop and test this approach are underway.
Sponsor
This program is sponsored by Carmine Therapeutics and partially funded by the Cystic Fibrosis Foundation.
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