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August 16, 2022 News by Teresa Carvalho, MS

Nula-cel (GPH101) Gene Therapy Phase 1/2 Trial Doses 1st Patient

The first patient has been dosed in a Phase 1/2 trial assessing the safety and preliminary efficacy of Graphite Bio’s experimental gene editing therapy GPH101 (now called nula-cel) in people with sickle cell disease (SCD). The trial, called CEDAR (NCT04819841), is currently recruiting participants at three sites,…

May 5, 2022 News by Steve Bryson, PhD

FDA Grants Fast Track Status to SCD Gene-editing Therapy GPH101

Graphite Bio’s GPH101, an investigational gene-editing therapy that aims to correct the genetic mutation that causes sickle cell disease (SCD) and potentially cure the condition, has been awarded fast track designation by the U.S. Food and Drug Administration (FDA). The fast track process is designed to accelerate the…

March 22, 2022 News by Steve Bryson, PhD

Gene-editing Trial of GPH101 to Start Dosing 1st Patient This Year

Dosing will soon start in a Phase 1/2 trial of GPH101, an experimental gene-editing therapy designed to correct the genetic mutation that causes sickle cell disease (SCD). The first participant had been enrolled in the study late last year, but due to the recent surge of the SARS-CoV-2…

November 18, 2021 News by Marisa Wexler, MS

First Patient Enrolled in GPH101 Trial Testing Potential SCD Cure

The first participant has been enrolled in the Phase 1/2 clinical trial CEDAR evaluating Graphite Bio’s investigational gene editing therapy GPH101, which is designed to directly correct the genetic mutation that causes sickle cell disease (SCD). “We are thrilled that our first patient is now enrolled in our CEDAR…

October 21, 2021 News by Marisa Wexler, MS

Trial of Gene Editing Therapy – Potential SCD Cure – Now Recruiting

GPH101, Graphite Bio’s investigational gene editing therapy, is able to correct the disease-causing mutation in blood stem cells of people with sickle cell disease (SCD), new preclinical data shows. Graphite also announced it remains on track to start enrolling participants in a Phase 1/2 trial testing the gene…

March 25, 2021 News by Aisha I Abdullah PhD

Graphite Bio Raises $150M to Advance GPH101, Other Gene-editing Therapies

Graphite Bio has raised $150 million in Series B funding to expand and advance the clinical development of its pipeline of investigational gene-editing therapies — including GPH101, a potentially curative treatment for sickle cell disease (SCD). In addition…

January 19, 2021 News by Joana Carvalho, PhD

Clinical Trial Cleared for GPH101, First Potentially Curative SCD Therapy

The investigational gene editing therapy GPH101 will be the first potentially curative treatment for sickle cell disease (SCD) to be tested in a Phase 1/2 clinical trial. The U.S. Food and Drug Administration (FDA) has cleared GPH101 for clinical testing. With this decision, the therapy’s developer, Graphite Bio,…

September 22, 2020 News by David Melamed, PhD

Graphite Bio Raises $45M to Move New Way of Gene Editing Into Sickle Cell Trial

Graphite Bio, a startup biotech company, has raised $45 million in an initial financing round to advance its gene editing technology to treat sickle cell disease and other genetic conditions. The technology being developed at Graphite is designed to target a specific gene and repair it in living cells…

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