Graphite Bio has enrolled the first subject in the Phase I/II CEDAR clinical trial of its experimental therapy, GPH101, to treat sickle cell disease (SCD).
The company anticipates treating the first subject in the first half of next year.
A gene-edited autologous hematopoietic stem cell therapy, GPH101 is claimed to directly rectify the genetic mutation that causes SCD.
The open-label, multicentre trial will evaluate the safety, pharmacodynamics, success of engraftment, rate of gene correction, total haemoglobin in individuals with severe SCD treated with GPH101.
The trial will also assess various clinical and exploratory goals.
Nearly 15 adult and adolescent subjects will be enrolled at up to five trial centres in the US.
Graphite Bio noted that GPH101 is the first experimental treatment to leverage a well-differentiated technique to correct the mutation in the beta-globin gene.
This approach will aid in reducing the production of sickle haemoglobin and reinstate the expression of normal adult haemoglobin, potentially treating SCD.
The preliminary proof-of-concept results from the trial are expected by the end of next year.
Graphite Bio CEO Josh Lehrer said: “GPH101 is the first investigational therapy to enter clinical development that uses our next-generation gene-editing platform technology to directly correct the mutation in the beta-globin gene that causes sickle cell disease.
“Using our gene correction approach, we have demonstrated in preclinical studies an ability to decrease the production of harmful sickle haemoglobin and restore the expression of normal adult haemoglobin.
“This approach has the potential to restore normal physiology and is viewed as the gold standard for curing sickle cell disease.”
A genetic blood disorder, SCD is caused by a mutation in the beta-globin gene. The mutation causes red blood cells to become misshapen leading to anaemia, blood flow obstructions, and a reduced lifespan, among others.