REGENXBIO (NASDAQ:RGNX) announces that it has completed dosing of three subjects in cohort 2 in a Phase 1/2 clinical trial evaluating gene therapy RGX-121 in patients with mucopolysaccharidosis type II (MPS II), a rare inherited disorder in which the body lacks a key enzyme to break down long chains of sugar molecules called glycosaminoglycans. The buildup of the sugars in cells leads to a range of sequelae including short stature, joint deformities, cognitive decline and heart problems.
The gene therapy was well-tolerated with no treatment-related serious adverse events. Additional data should be available in H2.
On a similar note, a single patient with MPS I who received gene therapy RGX-111 is also doing well, experiencing no serious treatment-related adverse events. Additional site activations are underway.
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