Precision BioSciences will use their novel genome editing technology called ARCUS® to develop a potentially curative treatment for sickle cell disease (SCD) and other hemoglobinopathies in collaboration with Novartis Pharma. The technology will use a custom nuclease to insert, in vivo, a therapeutic transgene at a “safe harbor” location in the genome.

“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,” the chief scientific officer and cofounder of the company Derek Jantz, PhD, said in a press release.

ARCUS uses sequence-specific nucleases to insert, remove, or repair DNA in vivo. It is based on I-CreI, a naturally occurring genome editing enzyme, which makes precise cuts in the DNA. ARCUS can be used to insert a healthy copy of a mutated gene at its usual site or at a so-called “safe harbor” to allow the production of a normal protein without affecting the expression or regulation of adjacent genes.

SCD is caused by a mutation in the gene that encodes the beta-subunit of the hemoglobin protein. This causes hemoglobin to polymerase causing red blood cells to acquire a sickle-like shape reducing their ability to carry oxygen. It can also lead to microvascular blockage and ischemic tissue damage.

Read more about the etiology of SCD

It is thought that sickle cell anemia affects around 20 million people worldwide, most of whom live in sub-Saharan Africa.

“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,” Dr. Jantz said. “Perhaps most importantly, it could open the door to treating patients in geographies where stem cell transplant is not a realistic option.”


Precision BioSciences announces in vivo gene editing collaboration with Novartis to develop potentially curative treatment for disorders including sickle cell disease. News release. Precision BioSciences; June 21, 2022.