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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 63rd Annual American Society of Hematology (ASH) Meeting back in December and now expects to treat its first patient by the end of the year.

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.

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