Companies to partner on one-time treatments for sickle-cell disease and β-thalassemia, using a nuclease designed for DNA insertion into the genome.
Precision BioSciences and its President and CEO Michael Amoroso have begun to deliver on the company’s commitment to maximize opportunities for its ARCUS genome editing platform —and satisfy Novartis’ growing interest in gene editing therapies—by inking an up-to-$1.4 billion in vivo gene editing R&D collaboration and license agreement with the pharma giant. The companies are partnering to develop one-time treatments for blood disorders that include sickle cell disease (SCD) and beta thalassemia. [Precision BioSciences]
Precision has agreed to develop for Novartis a single custom ARCUS sequence-specific DNA-cutting enzyme, or nuclease, designed to insert in vivo, a therapeutic transgene at an undisclosed “safe harbor” location in the genome where a new gene can be added without otherwise affecting the patient’s DNA of gene expression patterns. Precision has agreed to issue an exclusive license for the custom ARCUS nuclease to Novartis for further development . . .
