Precision BioSciences and Novartis announced this week that they will be working together on the research and development of in vivo gene editing therapies for sickle cell disease and other blood diseases.
Under the outlined agreement, North Carolina-based Precision will develop and conduct in vitro characterization of an ARCUS nuclease and Novartis will then handle the remaining research, development, manufacturing and commercialization. In exchange for exclusive licensing to the nuclease developed by Precision, Novartis will pay $75 million upfront, and up to $1.4 billion in future payments.
The ARCUS nuclease is designed and intended to insert an in vivo, transgene that addresses the genetic faults that lead to disease, offering a potential one-time transformative treatment option for diseases such as sickle cell and betha thalassemia.
“The in vivo gene editing approach that we are pursuing for sickle cell disease could have a number of significant advantages over other ex vivo gene therapies currently in development,” said Derek Jantz, Precision’s chief scientific officer and co-founder. “Perhaps most importantly, it could open the door to treating patients in geographies where stem cell transplant is not a realistic option. We believe that the unique characteristics of the ARCUS platform, particularly its ability to target gene insertion with high efficiency, make it the ideal choice for this project, and we look forward to working with our partners at Novartis to bring this novel therapy to patients.”