Page Title
Drug Development Pipeline
Nanite
Status
Pre-clinicalTherapeutic Approach
Genetic Therapy
This program is exploring polymer nanoparticles, a type of delivery vehicle that could be used to carry genetic therapies into the lung. Compared to other delivery vehicles, these polymer nanoparticles could more accurately target lung cells and potentially better resist the thick, sticky mucus that clogs the lungs of people with CF.
At first, Nanite will work on developing polymer nanoparticles that can deliver messenger RNA (mRNA) therapy into the lungs. Delivery of mRNA would allow lung cells to make full-length, functional CFTR protein, regardless of an individual’s CFTR mutations. Eventually, Nanite hopes these nanoparticles can be used to deliver other types of genetic therapies, such as gene therapy or gene editing, and target other organs affected by CF, such as the pancreas.
Status
Laboratory studies to develop and test this technology are underway.
Sponsor
This program is sponsored by Nanite Inc. and partially funded by the Cystic Fibrosis Foundation.

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