Wired News – Sangamo Gets CTA from UK Agency for Evaluating SB-FIX for Treatment of Hemophilia B
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LONDON, UK / ACCESSWIRE / March 02, 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 February 28, 2018, the Company, which is a pharmaceutical organization that focuses on translating groundbreaking science into genomic therapies for transforming patients' lives, declared that the United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA) has granted the Clinical Trial Authorization (CTA) for enrollment of subjects into the Company's ongoing Phase-1/2 clinical trial for evaluating SB-FIX. Register today and get access to over 1000 Free Research Reports by joining our site below:
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SB-FIX is a zinc finger nuclease (ZFN)-mediated in vivo genome editing treatment for hemophilia B. This CTA would permit initiation of Europe's first in vivo genome editing study.
Hemophilia B, A Bleeding Disorder
Hemophilia is a rare bleeding disorder characterized by the abnormal clotting of blood, caused by mutations in genes that encode factors that help the blood clot and stop bleeding when blood vessels are injured. A defect in the gene-encoding clotting Factor IX protein leads to Hemophilia B. Many a times, people suffering with Hemophilia B have spontaneous bleeding episodes, which often lead to joint diseases such as arthritis.
As per the National Hemophilia Foundation and the World Federation of Hemophilia, Hemophilia B occurs in approximately one in every 25,000 male births in the US. At present, Hemophilia B affects around 4,000 males in the US and 1,500 in the UK.
Proper Hemophilia B treatment needs ongoing, preventive infusions. The existing treatment of Hemophilia includes protein replacement of the defective clotting factor with regular infusion of recombinant clotting factors or plasma concentrates. However, these therapies are high-priced and may even incite the body to create antibodies against the factors that constrain the benefits of treatment. Thus, patients with Hemophilia B need improved treatment options.
What Makes Sangamo's In Vivo Genome Editing Approach Unique
Sangamo intends to treat Hemophilia B by leveraging its proprietary ZFN genome editing technology. The in vivo genome editing approach permanently and precisely integrates the therapeutic Factor IX gene into the DNA. This differentiates from the conventional AAV cDNA gene therapy and the lenti-or retroviral-based gene therapies that insert genes randomly into the genome.
This technology will help insert a corrective gene into an accurate location in the DNA of liver cells, which would enable the patient's liver to produce a lifelong and stable supply of the Factor IX protein.
Moreover, the ZFNs and the corrective gene are provided in a single intravenous infusion using AAV vectors that target the liver. This helps restrict the level of editing to liver cells.
ZFNs then enter the cells as inactive DNA instructions in a format designed specifically for liver cells to unlock. And after the format is unlocked, the ZFNs identify, bind to, and cut the DNA in the precise location within the albumin gene.
Thereafter, liver cells use the cells' natural DNA repair processes to insert the corrective gene for Factor IX at that specific location.
Implications of the CTA
The CTA allows evaluation of SB-FIX in both adults and adolescents. If SB-FIX demonstrates its preliminary safety and efficacy in the ongoing Phase-1/2 clinical trial in adult patients of 18 years or older, Sangamo will commence the enrollment of adolescent patients of 12 to 17 years of age into the study. In fact, the clinical sites in the US have been initiated and are already screening adult subjects.
Sangamo intends to initiate clinical sites in the UK for the SB-FIX Phase-1/2 clinical trial in the later part of 2018. Besides, the Company also plans to file additional CTAs for SB-318 and SB-913 in vivo genome editing treatments for Mucopolysaccharidosis Type I (MPS I) and MPS II, respectively.
Improved Treatment Options for Hemophilia B Patients
Edward Conner, M.D., Sangamo's Chief Medical Officer, shared his pleasure on signing the agreement with UK's MHRA to expand the SB-FIX clinical trial to sites in the UK. The Company's in vivo genome editing has the potential to provide a life-long therapeutic solution for various genetic diseases. He believes that the greatest benefit of this approach would be for the treatment of children. Thus, Sangamo aims to collect more safety and efficacy data for supporting progression of clinical trials into younger patient populations.
Stock Performance Snapshot
March 01, 2018 - At Thursday's closing bell, Sangamo Therapeutics' stock fell 1.88%, ending the trading session at $23.50.
Volume traded for the day: 2.68 million shares, which was above the 3-month average volume of 1.90 million shares.
Stock performance in the last month – up 12.17%; previous three-month period – up 62.63%; past twelve-month period – up 400.00%; and year-to-date - up 43.29%
After yesterday's close, Sangamo Therapeutics' market cap was at $2.02 billion.
The stock is part of the Healthcare sector, categorized under the Biotechnology industry.
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