"We have about eight patients that are in the insurance approval process," she said, for gene therapy treatments for beta thalassemia and sickle cell disease. "It's something that we do expect to ...
The strategic shift will allow the firm to focus on advancing a newly announced in vivo gene-editing therapy candidate that's in preclinical development.
Their lead program, Reni-cel, is a gene-edited cell therapy targeting hemoglobinopathies such as sickle cell disease and beta-thalassemia. Reni-cel is progressing in sickle cell disease ...