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LONDON, UK / ACCESSWIRE / April 6, 2018 / Active-Investors.com has just released a free research report on Sangamo Therapeutics, Inc. (NASDAQ: SGMO). If you want access to this report all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=SGMO as the Company's latest news hit the wire. On April 04, 2018, the Richmond-based biopharmaceutical Company announced that the preclinical murine study data from the Company's in vivo genome editing program for Mucopolysaccharidosis Type II (MPS II) was published in the April 2018 issue of Molecular Therapy. Register today and get access to over 1,000 Free Research Reports by joining our site below:
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Study Provided Proof of Concept for Development of SB-913 Currently Being Evaluated in the CHAMPIONS Study
The preclinical study provided proof of concept for development of SB-913, an in vivo genome editing product candidate that the Company is currently evaluating in the CHAMPIONS Study, a Phase-1/2 clinical trial assessing the potential safety and efficacy of SB-913 in up to nine adult males with attenuated MPS II. SB-913 uses Sangamo's ZFN genome editing technology delivered intravenously via AAV6 vectors and is a single-treatment strategy intended to provide stable, continuous production of the IDS enzyme.
Preclinical Study Results
Male MPS II model mice between six and nine weeks of age were injected with one of three increasing dose levels of a genome editing treatment consisting of AAV2/8 vectors encoding a pair of ZFNs and a corrective human IDS (hIDS) gene. The treatment was designed to integrate the hIDS gene in a precise location within the albumin gene in liver cells.
Treated mice, at both one and four months after injection, exhibited dose-dependent expression of IDS enzyme in blood and peripheral tissues, including the spleen, kidney, lung, heart, and skeletal muscle. Enzyme expression in liver cells reached levels greater than 200-fold higher than in wild-type mice. In all tested peripheral organs, treatment at the highest dose resulted in greater than 95% reduction in glycosaminoglycan (GAG) substrate levels. Importantly, treatment with Sangamo's genome editing therapy at the highest dose level also prevented the development of neurocognitive deficit in the young MPS II model mice, as measured by the Barnes Maze test.
EMA Recommended Orphan Medicinal Product Designation for SB-913 for the Treatment of MPS II
In December 2017, Sangamo Therapeutics announced that the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA) has issued a positive opinion on the application for orphan medicinal product designation (OMPD) for SB-318 and SB-913 for the treatment of rare lysosomal storage disorders, MPS I and MPS II, respectively. SB-318 and SB-913 have already received Orphan Drug, Fast Track, and Rare Pediatric Disease designations from the US Food and Drug Administration (FDA).
About MPS II
Mucopolysaccharidosis type 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. It is a progressively debilitating disorder; however, the rate of progression varies among affected individuals. The symptoms of MPS II are generally not apparent at birth but usually start to become noticeable after the first year of life. Often, the first symptoms may include abdominal hernias, ear infections, runny noses, and colds. As the buildup of GAGs continues throughout the cells of the body, signs of MPS II become more visible. Physical appearances of many children with MPS II include a distinctive coarseness in their facial features, including a prominent forehead, a nose with a flattened bridge, and an enlarged tongue.
Sangamo's In Vivo Genome Editing Approach
Sangamo's ZFN-mediated in vivo genome editing approach makes use of the endogenous albumin gene locus, a highly expressing and liver-specific site that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein. The ability to permanently integrate the therapeutic gene in a highly specific, targeted fashion significantly differentiates the Companies in vivo genome editing approach from conventional AAV cDNA gene therapy. The design of these programs is ultimate to target a population that includes pediatric patients, and it will be important in this population to be able to produce stable levels of therapeutic protein for the lifetime of the patient.
About Sangamo Therapeutics, Inc.
Established in 1995, Sangamo Therapeutics 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.
Stock Performance Snapshot
April 05, 2018 - At Thursday's closing bell, Sangamo Therapeutics' stock dropped 7.41%, ending the trading session at $17.50.
Volume traded for the day: 2.52 million shares, which was above the 3-month average volume of 2.18 million shares.
Stock performance in the last three-month – up 0.57%; previous six-month period – up 4.48%; past twelve-month period – up 251.76%; and year-to-date - up 6.71%
After yesterday's close, Sangamo Therapeutics' market cap was at $1.57 billion.
The stock is part of the Healthcare sector, categorized under the Biotechnology industry.
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