Sangamo Therapeutics: راه حل های مهندسی ژنوم برای بیماری های نادر


Sangamo just became the first company to do in vivo genome editing in the clinic.

One of the four trials Sangamo has in the clinical is SB-525. Pfizer agreed to potential milestone payments of $475 million plus royalties to collaborate with them on this product.

Sangamo has hired 5 to 10 new business development employees in the past few months to manage in-licensing and new collaboration opportunities in oncology, tauopathies, and HIV.


Sangamo Therapeutics (NASDAQ:SGMO) has been a leader in genome editing for over 20 years. Its rich history of scientific discovery includes being the first to do gene editing in vitro (in cell within the glass), ex vivo (outside the body) and just last month in vivo (inside the body). Sangamo’s approach to genome editing focuses on zinc finger nucleases “ZFNs” which are a class of DNA-binding proteins engineered to facilitate targeted genome editing. Though this approach is more complicated, it believes it provides a more flexible and efficient editing approach that reduces the risk of off-target edits. The company itself could be considered version 2.0 after a year of leadership turnover led by CEO Sandy Macrae who brought a therapeutic focus to a company steeped in scientific research. The key for 2018: Data from pivotal clinical trials designed to measure treatment-related adverse events. Sangamo has smartly balanced its risk profile with a partnering strategy explained below. There are a number of potential catalysts upcoming which have the potential for outsized performance. The risk-reward profile is weighted to the upside, but downside risk from dosing effects can’t be ignored.

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Sangamo Therapeutics: راه حل های مهندسی ژنوم برای بیماری های نادر – اخبار زیست فناوری

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