Graphite Bio has dosed the first subject in the Phase I/II CEDAR clinical trial of investigational gene editing therapy, nulabeglogene autogedtemcel (nula-cel/ GPH101) in sickle cell disease (SCD) patients. 

The multicentre, single-dose, open-label trial will analyse the safety, initial efficacy and pharmacodynamics of nula-cel in nearly 15 severe SCD patients. 

Furthermore, engraftment success, gene correction rates, total haemoglobin and various other clinical and exploratory endpoints will be analysed in the trial.

At present, the trial is enrolling subjects at various US sites.

The preliminary proof-of-concept findings from the trial are expected in the middle of next year.

A gene editing autologous hematopoietic stem cell (HSC) therapy, nula-cel is created to directly rectify the genetic mutation that leads to SCD and cure the ailment.

It leverages a well-differentiated gene correction method to accurately correct the mutation in the beta-globin gene. 

This aids in lowering the production of sickle haemoglobin (HbS) and restoring the expression of adult haemoglobin (HbA) to potentially cure SCD. 

Previously, nula-cel obtained Fast Track and Orphan Drug designations from the US Food and Drug Administration (FDA) for treating SCD.

Graphite Bio CEO Josh Lehrer said: “We continue to make tremendous progress with the development of nula-cel, which in preclinical studies successfully corrected the sickle cell disease mutation, directly reducing sickle haemoglobin and restoring healthy adult haemoglobin to potentially curative levels.

“We believe nula-cel could be a definitive cure for sickle cell disease, with the potential to address all complications associated with this life-threatening disease.”

SCD is a fatal inherited blood disorder and impacts millions of people, making it the most common monogenic disease globally. 

In November last year, the company enrolled the first participant in the CEDAR trial of GPH101 to treat SCD.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

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