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LONDON, UK / ACCESSWIRE / November 17, 2017 / Pro-Trader Daily takes a look at the latest corporate events and news making the headlines for Sangamo Therapeutics, Inc. (NASDAQ: SGMO), following which we have published a free report that can be viewed by signing up at http://protraderdaily.com/optin/?symbol=SGMO. The Company announced on November 15, 2017, that first patient has been treated in the Phase-1/2 clinical trial (the CHAMPIONS study) evaluating SB-913, an investigational invivo genome editing therapy, for the treatment of patients with mucopolysaccharidosis type II (MPS II). For immediate access to our complimentary reports, including today's coverage, register for free now at:
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Sangamo at the Start of a New Frontier of Genomic Medicine
Dr. Sandy Macrae, Chief Executive Officer of Sangamo, stated that for the first time, a patient has received a therapy intended to precisely edit the DNA of cells directly inside the body. Dr. Macrae added that the Company is at the start of a new frontier of genomic medicine.
SB-913 Utilizes Sangamo's ZFN Genome Editing Technology
SB-913 is a genome editing product candidate for the treatment of MPS II, and is designed as a single treatment strategy intended to provide stable, continuous production of the IDS enzyme for the lifetime of the patient. Using Sangamo's zinc finger nuclease (ZFN) genome-editing technology, SB-913 inserts a corrective gene into a precise location in the DNA of liver cells. To restrict editing to liver cells, the ZFNs and the corrective gene are delivered in a single intravenous infusion using AAV vectors that target the liver.
Sangamo Working Closely with FDA and NIH Recombinant DNA Advisory Committee
Ed Conner, M.D., Chief Medical Officer of Sangamo, expressed that the Company has been working closely with the FDA and the NIH Recombinant DNA Advisory Committee to make sure that it is thoroughly and prudently developing this new class of medicines.
FDA Granted Fast Track Designation and Orphan Drug Designation for SB-913
On March 01, 2017, the US Food and Drug Administration (FDA) granted orphan drug designation to SB-913 for the treatment of MPS II. On July 13, 2017, FDA granted Fast Track designation to SB-318 and SB-913, for the treatment of MPS I and MPS II, respectively. Additionally, in February 2017, SB-318 for MPS I and SB-913 for MPS II have also received Rare Pediatric Disease designations from the FDA.
About the CHAMPIONS Study
The CHAMPIONS study is an open-label clinical study designed to assess the safety, tolerability, and preliminary efficacy of the SB-913 investigational genome editing therapy in up to nine adult males with MPS II.
About MPS II
MPS II, also known as Hunter syndrome, is a condition that affects many different parts of the body and occurs almost exclusively in males. MPS II has a wide range of symptoms that vary in severity and can be managed and treated with enzyme replacement therapies, but there is no cure for MPS II. MPS II patients are missing the enzyme iduronate sulfatase, which is essential to breaking down the mucopolysaccharides dermatan and heparan sulphate. These materials remain stored in the body's cells, causing progressive damage.
About Sangamo Therapeutics, Inc.
Established in 1995, Sangamo Therapeutics, Inc. is a clinical-stage biopharmaceutical company, focused on translating ground-breaking science into genomic therapies that transform patients' lives using the Company's industry-leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is headquartered in Richmond, California.
Last Close Stock Review
Sangamo Therapeutics' share price finished yesterday's trading session at $14.60, marginally sliding 0.34%. A total volume of 1.30 million shares have exchanged hands. The Company's stock price soared 47.47% in the last three months, 87.18% in the past six months, and 251.81% in the previous twelve months. Additionally, the stock skyrocketed 378.69% since the start of the year. The stock currently has a market cap of $1.22 billion.
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