Early results from a gene-editing trial suggest that the treatment may be working, the Associated Press reports.
In its Phase 1/2 CHAMPIONS Study, Sangamo Therapeutics is examining an in vivo genome editing treatment for Hunter syndrome, a condition marked by the accumulation of glycosaminoglycans, which leads to the enlargement of organs, tissue damage, and more. In particular, patients in the trial were given low, medium, or high doses of SB-913, a zinc-finger-based genome editing therapy that aims to insert a functional copy of the IDS gene that is affected in patients.
As the AP reports, the two patients given the medium dose saw drops in their urine glycosaminoglycans levels — after four months, they experienced an average 51 percent decline in glycosaminoglycans. The low-dose patients, however, saw a rise in glycosaminoglycans, and the high-dose patients are still being evaluated. Still, the AP says these results may be cause for cautious optimism.
"This is not proof that this is a successful therapy yet, that these patients had enough gene editing to now supply them with the enzyme they need for the rest of their life," the University of North Carolina, Chapel Hill's Joseph Muenzer, who leads the study, tells the AP. He adds, though, that the treatment appears safe.
The trial includes Brian Madeux, who was the first to undergo the treatment, the AP notes.