U.S. Markets close in 2 hrs 55 mins

Wired News – Sangamo Therapeutics’ Preclinical Study Data from MPS II in Vivo Genome Editing Program Published in Molecular Therapy

Stock Monitor: KalVista Pharma Post Earnings Reporting

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:


Active-Investors.com is currently working on the research report for KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), which also belongs to the Healthcare sector as the Company Sangamo Therapeutics. Do not miss out and become a member today for free to access this upcoming report at:


Active-Investors.com is focused on giving you timely information and the inside line on companies that matter to you. This morning, Sangamo Therapeutics most recent news is on our radar and our team decided to put out a fantastic report on the company that is now available for free below:


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.


Active-Investors (A-I) produces regular sponsored and non-sponsored reports, articles, stock market blogs, and popular investment newsletters covering equities listed on NYSE and NASDAQ and Canadian stocks. A-I has two distinct and independent departments. One department produces non-sponsored analyst certified content generally in the form of press releases, articles and reports covering equities listed on NYSE and NASDAQ and the other produces sponsored content (in most cases not reviewed by a registered analyst), which typically consists of compensated investment newsletters, articles, and reports covering listed stocks and micro-caps. Such sponsored content is outside the scope of procedures detailed below.

A-I has not been compensated; directly or indirectly; for producing or publishing this document.


The non-sponsored content contained herein has been prepared by a writer (the "Author") and is fact checked and reviewed by a third-party research service company (the "Reviewer") represented by a credentialed financial analyst [for further information on analyst credentials, please email info@active-investors.com. Rohit Tuli, a CFA® charterholder (the "Sponsor"), provides necessary guidance in preparing the document templates. The Reviewer has reviewed and revised the content, as necessary, based on publicly available information which is believed to be reliable. Content is researched, written and reviewed on a reasonable effort basis. The Reviewer has not performed any independent investigations or forensic audits to validate the information herein. The Reviewer has only independently reviewed the information provided by the Author according to the procedures outlined by A-I. A-I is not entitled to veto or interfere in the application of such procedures by the third-party research service company to the articles, documents or reports, as the case may be. Unless otherwise noted, any content outside of this document has no association with the Author or the Reviewer in any way.


A-I, the Author, and the Reviewer are not responsible for any error which may be occasioned at the time of printing of this document or any error, mistake or shortcoming. No liability is accepted whatsoever for any direct, indirect or consequential loss arising from the use of this document. A-I, the Author, and the Reviewer expressly disclaim any fiduciary responsibility or liability for any consequences, financial or otherwise arising from any reliance placed on the information in this document. Additionally, A-I, the Author, and the Reviewer do not (1) guarantee the accuracy, timeliness, completeness or correct sequencing of the information, or (2) warrant any results from use of the information. The included information is subject to change without notice.


This document is not intended as an offering, recommendation, or a solicitation of an offer to buy or sell the securities mentioned or discussed, and is to be used for informational purposes only. Please read all associated disclosures and disclaimers in full before investing. Neither A-I nor any party affiliated with us is a registered investment adviser or broker-dealer with any agency or in any jurisdiction whatsoever. To download our report(s), read our disclosures, or for more information, visit http://active-investors.com/legal-disclaimer/.


For any questions, inquiries, or comments reach out to us directly. If you're a company we are covering and wish to no longer feature on our coverage list contact us via email and/or phone between 09:30 EDT to 16:00 EDT from Monday to Friday at:

Email: info@active-investors.com
Phone number: 73 29 92 6381
Office Address: 6, Jalan Kia Peng, Kuala Lumpur, 50450 Kuala Lumpur, Wilayah Persekutuan Kuala Lumpur, Malaysia

CFA® and Chartered Financial Analyst® are registered trademarks owned by CFA Institute.

SOURCE: Active-Investors