Interim results presented by UNC's Joseph Muenzer, MD, PhD, provide preliminary evidence that in vivo genome editing occurred in a clinical trial testing a treatment for Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome.
CHAPEL HILL, NC – Preliminary molecular and enzymatic evidence of editing of the human genome in vivo (inside the body) was presented today by UNC School of Medicine’s Joseph Muenzer, MD, PhD, at the WORLDSymposium 2019 being held in Orlando, Florida.
This finding was part of the interim results from the Phase 1/2 CHAMPIONS Study evaluating SB-913, a zinc finger nuclease (ZFN) in vivo genome editing product candidate for patients with Mucopolysaccharidosis Type II (MPS II).
MPS II, also known as Hunter syndrome, is a rare genetic disorder caused by a deficiency of iduronate-2-sulfatase (IDS), a lysosomal enzyme which is required to break down or recycle the toxic buildup of glycosaminoglycans (GAGs). Without IDS enzyme activity, GAGs accumulate in cells throughout the body, leading to widespread tissue and organ damage. The current standard-of-care treatment for MPS II is enzyme replacement therapy (ERT), given as weekly intravenous infusions. SB-913 is an investigational product candidate being evaluated to treat MPS II using ZFNs, which are designed to insert a normal copy of the IDS gene into a precise location in the DNA of liver cells. The goal of SB-913 treatment is to enable a patient's liver to produce a continuous supply of functional IDS enzyme for life.