Sangamo Therapeutics Inc (NASDAQ: SGMO) rose Wednesday after the company disclosed that the first patient has been treated in landmark Phase 1/2 clinical trial evaluating in vivo genome editing for mucopolysaccharidosis type II (MPS II).
The company, based out of Richmond, California, aims to treat MPS II by using genome editing to insert a corrective gene into a precise location in the DNA of liver cells with the goal of enabling a patient's liver to produce a lifelong and stable supply of an enzyme he or she currently lacks.
Without that enzyme, called iduronate-2-sulfatase (IDS), people with MPS II suffer debilitating buildup of toxic carbohydrates in cells throughout their body. Approximately one in 100,000 to one in 170,000 people are born with MPS II.
Many people with MPS II receive weekly infusions of enzyme replacement therapy (ERT), the current standard-of-care treatment. Within a day of receiving ERT, however, IDS quickly returns to near undetectable levels in the blood.
CEO Sandy Macrae said "For the first time, a patient has received a therapy intended to precisely edit the DNA of cells directly inside the body. We are at the start of a new frontier of genomic medicine.”
Two additional clinical trials are underway in the United States to evaluate Sangamo's in vivo genome editing therapeutics for hemophilia B and MPS I, which is also known as Hurler or Hurler-Scheie syndrome. All three trials use ZFNs designed to edit liver cells at the same location in the albumin gene, but differ in delivering the corrective gene relevant to the respective disease.
Shares in Sangamo galloped $1.85, or 14.3%, to $14.75, in the last hour of trading on Wednesday.
