Engineering Synthetic Sputum-penetrating Gene Carriers

C11417: Synthetic Sputum-penetrating Gene Carriers

Novelty:

The technology provides novel compositions and methods to deliver gene therapy to cystic fibrosis (CF) patients.

Value Proposition:

CF is one of the most common genetic disorders. Currently, there are no aerosol gene delivery methods to efficiently penetrate the hyperviscoelestic sputum in the lungs that is a result of CF. This creates a barrier for the treatment of CF and other obstructive airway diseases. This invention consists of synthetic gene carriers to more efficiently deliver the gene therapy necessary to treat CF. By tuning the polymer properties of the gene carrier to a specific size and surface charge, this platform is able to administer gene therapy for CF through purulent sputum. These synthetic gene carriers present a novel and efficient method for the treatment of CF. Key advantages of this technology include:

Technical Details:

Johns Hopkins researchers have invented a novel technology to deliver gene therapy through the purulent sputum that is caused by CF. The invention consists of a mixture of PEG-conjugated and unconjugated cationic polymers. Through manipulation of carrier size and surface charge, these synthetic carriers are able to diffuse across sputum. Further, the use of PEG-conjugated cationic polymers reduces lung inflammation and also increases gene carrier distribution and retention in the lungs and airways. From animal model data, this technology shows promise as an efficient means of gene therapy to treat CF.

Looking for Partners:

To develop and commercialize the technology as a treatment for CF and other obstructive airway diseases.

Stage of Development:

Preclinical

Data Availability:

Mouse data

Publications/Associated Cases:

1. Biomaterials, 30(13), 2591-2597. 2. Advanced Drug Delivery Reviews, 61(2), 158-171. 3. Advanced Drug Delivery Reviews, 61(2), 73-74.