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Sangamo and Pfizer Report Positive Results for Hemophilia A Study

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Sangamo Therapeutics and partner Pfizer reported positive results for phase 1/2 Alta study for hemophilia A at the International Society on Thrombosis and Hemostasis Congress in Melbourne, Australia.

Sangamo Therapeutics Inc. (SGMO:NASDAQ) shares are up more than 8% today on high volume after the company and its partner Pfizer Inc. (PFE:NYSE) jointly announced updated results after the close of trading Friday evening for their Phase 1/2 Alta study evaluating investigational SB-525 gene therapy for severe hemophilia A.

The company reported that data showed that SB-525 was generally well tolerated and demonstrated a dose-dependent increase in Factor VIII (FVIII) activity levels.

"The initial results of the Alta study presented at International Society on Thrombosis and Hemostasis Congress demonstrate that SB-525 has the potential to be a predictable and reliable treatment that may bring clinical benefit to patients with hemophilia A," stated Adrian Woolfson, M.D., Ph.D., Sangomo's EVP of Research and Development.

The joint news release stated that "based on the accumulating results from the Alta study, the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation for SB-525 gene therapy to treat severe hemophilia A."

In addition to partnering with Pfizer on hemophilia applications, Sangamo also collaborates with Kite, a Gilead Sciences, Inc. (GILD:NASDAQ) company, on developing next-generation ex vivo cell therapies to treat cancer; Sanofi (SNY:NASDAQ) on developing gene-edited cell therapies for the treatment of beta thalassemia and sickle cell disease; and Shire plc now Takeda Pharmaceutical Co. Ltd. (4502:JP) on developing therapeutics for Huntington's disease.

Sangamo shares have traded more than 14 million shares in the first half of trading, greatly exceeding the 200-day average daily volume of 1.944 million shares. So far today, the firm's shares have traded between $12.20 to $13.33 and are currently trading at $12.34/share, up $0.94/share (+8.25%) over Friday's close of $11.40. While today's trading action appears very positive, it is important to note that the stock is trading pretty much at the mid-point of the 52-week high and low prices of $19.25 (09/04/18) and $6.26 (02/07/19) respectively.


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