Graphite launches sickle cell study

 Graphite Bio 

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 announced the initiation of patient dosing in the Phase 1/2 CEDAR trial with its asset GPH101 (nulabeglogene autogedtemcel) in people with sickle cell disease (SCD).

Nula-cel is an investigational next-gen gene-editing autologous hematopoietic stem cell (HSC) therapy designed to directly correct the genetic mutation that causes sickle cell disease (SCD).

The CEDAR Phase 1/2 single-dose clinical trial is evaluating the safety, preliminary efficacy and pharmacodynamics of nula-cel in approximately 15 patients at multiple sites in the United States with severe SCD.

Josh Lehrer, Chief executive officer, said, For decades, the goal of gene editing has been to precisely correct genetic mutations that cause disease. Today, we took an important step toward achieving that goal by dosing our first patient with nula-cel, the first investigational therapy designed to correct a mutated gene to normal. This first use of high-efficiency precision DNA repair to correct a genetic mutation is an important milestone not only for our company but also for the gene editing field and, hopefully, for the sickle cell community.”

The company is planning to announce initial proof-of-concept data readout from the CEDAR trial in mid-2023.

https://www.benzinga.com/general/biotech/22/08/28458313/graphite-bio-shares-soar-as-initial-stage-blood-cell-disorder-study-launched