Sangamo Announces U.K. Authorization Of Clinical Trials Evaluating Zinc Finger Nuclease In Vivo Genome Editing Treatments SB-318 For MPS I And SB-913 For MPS II
RICHMOND, Calif., June 4, 2018 /PRNewswire/ --
"Patients with MPS I and MPS II have very few treatment options, and we are excited to expand access to our clinical trials to the
The CTA for SB-913 allows for treatment of children as young as five years of age following a review of cumulative safety data from adult and adolescent cohorts. The SB-318 CTA application was based on the protocol of the ongoing Phase 1/2 clinical trial which includes only adult patients. Sangamo plans this year to request a protocol amendment for the SB-318 study to include younger patients.
Sangamo expects to initiate clinical trial sites in the
Sangamo's In Vivo Genome Editing Approach
Sangamo aims to treat patients with MPS I and MPS II using its proprietary ZFN genome editing technology 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 the alpha-L-iduronidase (IDUA) and iduronate 2-sulfatase (IDS) enzymes for MPS I and MPS II, respectively.
To restrict editing to liver cells, the ZFNs and the corrective gene are delivered in a single intravenous infusion using an AAV vector that targets the liver. The ZFNs enter the cells as inactive DNA instructions in a format designed only for liver cells to unlock. Once "unlocked," the ZFNs then identify, bind to and cut the DNA in a specific location within the albumin gene located in liver cells. Using the cells' natural DNA repair processes, liver cells can then insert the corrective gene for IDUA (MPS I) or IDS (MPS II) at that precise location.
The potential to permanently and precisely integrate the therapeutic IDUA (MPS I) or IDS (MPS II) gene into the DNA differentiates Sangamo's in vivo genome editing approach from conventional AAV cDNA gene therapy and from lenti- or retroviral-based gene therapies that insert genes randomly into the genome.
This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, references to Sangamo's ability to open clinical sites in the
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