Sangamo Therapeutics has treated the first patient in the EMPOWERS Study, a Phase l/ll clinical trial examining SB-318 for the treatment of patients with mucopolysaccharidosis type I (MPS I, Hurler syndrome).
The open-label, ascending-dose trial aims to evaluate the safety, tolerability and preliminary efficacy of SB-318 in up to nine adult patients with attenuated MPS I.
Primary objectives of the trial are incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) in up to 36 months after the infusion of SB-318.
Secondary objectives include the effect of SB-318 on α-L-iduronidase (IDUA) activity, effect of SB-318 on urine glycosaminoglycans (GAG) levels, as well as adeno-associated virus type 2/6 (AAV2/6) clearance in plasma, saliva, urine, stool, and semen.
As part of the EMPOWERS Study, patients are currently being screened at various US-based hospitals specialising in the care of patients with MPS I, including hospitals in Oakland, Gainesville, Atlanta, Minneapolis, New York and Cincinnati.
Sangamo Therapeutics chief medical officer Edward Conner said: “With this first patient in the Empowers Study of SB-318 for MPS I, and with five patients now treated in the CHAMPIONS Study of SB-913 for MPS II, we are making strong progress in the evaluation of our in vivo genome editing approach for these two rare inherited metabolic diseases.
“We remain on track to announce preliminary data from the MPS II CHAMPIONS Study in late summer.”
Sangamo has used its zinc finger nuclease (ZFN) genome editing technology to develop both the SB-318 and SB-913.
The technology is capable of inserting a corrective copy of the IDUA (SB-318) or IDS (SB-913) gene into a precise location in the DNA of liver cells in order to enable a patient's liver to produce a lifelong and stable supply of enzyme.