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Sangamo Therapeutics, Inc.(SGMO - Free Report) and partner Pfizer Inc. (PFE - Free Report) announced the dosing of first patient in a phase I/II study on its gene therapy candidate, SB-525, for treating hemophilia A. In fact, SB-525 is considered to be one of Sangamo's four leading product candidates.

In May 2017, the candidate also received a fast-track designation from the FDA and currently enjoys orphan drug status in the United States.

Consequently, shares of the company have skyrocketed 250.8% year to date, outperforming the industry’s gain of 7.2%.

At present, the open-label phase I/II Alta study is evaluating the safety and tolerability of SB-525 in up to 20 adult subjects with severe hemophilia A. Also, it is expected to find a suitable dose-range of the candidate.

In fact, SB-525 is being developed as part of a global collaboration between the two companies to develop and commercialize gene therapy programs for hemophilia A.

Data from this study are expected in late 2017 or early 2018.

Hemophilia A is a monogenic, rare bleeding disorder in which the blood does not clot normally. Per the press release, the number of hemophilia A patients in the United States is estimated to be 16,000 and over 150,000 globally.

Other companies are also developing gene therapy for the indication. In July 2017, Roche Holdings AG (RHHBY - Free Report) announced positive data from the phase III study, HAVEN 1, on its hemophilia A candidate, emicizumab. Again, Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) is developing fitusiran in a phase II study for the treatment of patients with hemophilia A and B.

Currently, the company carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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