Inventors: Jordan J. Green, Stephany Yi Tzeng, Garry R. Cutting, Erin W. Kavanagh
Unmet Need
Cystic fibrosis (CF) is a progressive, genetic disease that affects the lungs, pancreas, and other organs. There are close to 40,000 children and adults living with cystic fibrosis in the United States (see CFF). In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional. A number of therapeutic compounds called ‘CFTR modulators’ have been identified that restore the expression or function of CFTR, but 10% of CF patients carry premature stop codon mutations in which CFTR modulators would be ineffective (see Sharma et al). Therefore, there is a strong need for the development of a gene editing therapy that can be used to correct genetic mutations.
Technology Overview
Researchers at Johns Hopkins have developed a nanoparticle that is capable of organ specific delivery of gene editing therapies. The nanoparticle is composed of a well-tolerated polymer that can be modulated to target specific organs in the body. The nanoparticle also contains gene editing components that can edit malfunctioning genes to instead express the correct version of the protein. Researchers were able to demonstrate recovery of CFTR function after delivery of the nanoparticles in a CF model cell line.
Stage of Development
Preclinical data is available.
Publication
N/A
