Building on CRISPR and Striving for Definitive Cures: Josh Lehrer, MD
The chief executive officer of Graphite Bio discusses the phase 1/2 CEDAR trial of GPH101, an investigative Cas9-edited autologous CD34+ cell therapy for the treatment of sickle cell disease.
"Patients are trusting us to come in and go through a procedure where we're manufacturing their own cells to potentially cure them. It's a big commitment."
Graphite Bio expects to treat its first patient with sickle cell disease later this year with GPH101, its investigational next-generation gene-edited autologous hematopoietic stem cell product candidate.
Josh Lehrer, MD, MPhil, FACC, chief executive officer at Graphite Bio, joins CGTL to share updates on the phase 1/2 CEDAR trial (NCT04819841), his company's approach and platform, and other investigational therapies in the pipeline, including for β-thalassemia and Gaucher disease.
Graphite announced the first patient had been enrolled1 in the CEDAR trial at the
GPH101 was developed using Graphite’s next-generation targeted gene integration platform, which relies on Cas9 technology and a non-integrating DNA template to correct the mutation in the β-globin gene through the homology directed repair cellular pathway.
Reference
1. Graphite Bio enrolls first patient in phase 1/2 clinical trial of GPH101 for sickle cell disease. News release. Graphite Bio. November 17, 2021. https://www.businesswire.com/news/home/20211117005527/en/Graphite-Bio-Enrolls-First-Patient-in-Phase-12-Clinical-Trial-of-GPH101-for-Sickle-Cell-Disease
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