FDA Clears Graphite Bio to Begin Trial for Gene Therapy in Sickle Cell Disease

In response to Graphite Bio’s investigational new drug (IND) application for its gene editing therapy, GPH101, the FDA will allow the pharmaceutical company to initiate a phase I/II trial in patients with sickle cell disease (SCD).

Aimed at curing SCD by targeting the gene mutation that causes damaged and sickled red blood cells, GPH101 uses CRISPR technology to cut out this mutation in the beta-globin gene and paste in the wild-type DNA sequence. The CEDAR trial will evaluate the safety, preliminary efficacy, and pharmacodynamics of GPH101 in adult and adolescent patients with severe SCD.  “We are eager to initiate enrollment for the CEDAR clinical trial in early 2021, which will be a historic milestone as the first experimental treatment designed to correct the mutation that is the underlying cause of sickle cell disease,” said Graphite Bio CEO Josh Lehrer, MPhil, MD. “With targeted gene integration, GPH101 seeks to restore normal hemoglobin expression, which has been the ultimate goal of sickle cell disease treatment for more than 70 years.”

Recently, with support from the California Institute for Regenerative Medicine, Graphite Bio entered into a definitive license agreement with Stanford University for the preclinical development of GPH101.

Source: Graphite Bio press release, December 14, 2020.