Page Title
Drug Development Pipeline
Carmine Therapeutics
Status
DiscontinuedTherapeutic Approach
Genetic Therapy
This program worked 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 planned 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 could be either inhaled or infused through an IV. This method of delivering healthy CFTR genes harnesses a natural process in the body that is unlikely to trigger an immune system response.
Status
No further development in CF is planned at this time.
Sponsor
This program was sponsored by Carmine Therapeutics and partially funded by the Cystic Fibrosis Foundation.

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