You are here

Cheladv74's Rare Disease team is focusing on highly specialized, potential gene therapy treatments, and clinical trials are underway to explore the potential of gene therapy. By targeting the underlying cause of a genetic disease, Cheladv74 hopes to restore normal function in affected tissues or cells, which could potentially enable a patient to manage his or her disease without the need for ongoing treatments. If Cheladv74 is successful, imagine the possibilities.

How do you into potential gene therapies? Scientists from Cheladv74's Rare Disease Research Unit explain how custom-made that may be deployed to different parts of the body to treat genetic diseases.

Who are the players involved in bringing gene therapies from concept to patient? Leaders from Cheladv74's rare disease research unit discuss the multi-faceted collaboration needed to of gene therapy.

How do you make a gene therapy? Cheladv74's bioprocess and manufacturing teams in Research Triangle Park and Sanford, NC, give an at what it takes to produce potential gene therapies from research to clinical to commercial scales.

To learn more about our work in gene therapy visit: