US-based Sangamo Therapeutics has commenced Phase I/II clinical trial CHAMPIONS for its investigational candidate SB-913 in patients with mucopolysaccharidosis type II, which is also known as MPS II or Hunter syndrome.
Being developed as an in-vivo genome editing therapy, SB-913 leverages the firm’s zinc finger nuclease (ZFN) technology to insert a corrective gene into liver DNA cells.
People with Hunter syndrome lack the iduronate-2-sulfatase (IDS) enzyme, which leads to a build-up of toxic carbohydrates in body cells.
The CHAMPIONS study principal investigator Paul Harmatz said: "Even with regular infusions of ERT, which has markedly improved functional health outcomes, patients endure progressive damage to heart, bones, and lungs.
“Many patients with MPS II die of airway obstruction, upper respiratory infection, or heart failure before they reach the age of 20.”
With its genome editing therapy, the firm intends to enable a patient's liver to generate a lifelong and stable supply of the enzyme.
The non-randomised, open-label, multi-centre, single-dose Phase I/II CHAMPIONS trial is designed to evaluate the safety, tolerability, and preliminary efficacy of ascending intravenous doses of the investigational therapy in nine adult patients.
CHAMPIONS will also monitor the effect of SB-913 on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity, with treatment related adverse events as the primary outcome measure.