The platform is based on Sangamo's zinc finger DNA-binding protein (:ZFP) genome-editing technology.
We note that Sangamo is focused on the research, development and commercialization of engineered DNA-binding proteins for the development of novel therapeutic strategies for a range of diseases. This production in turn can provide potential curative treatments for a range of monogenic diseases such as hemophilia and lysosomal storage disorders (:LSD) including Gaucher and Fabry disease.
The data demonstrated therapeutic levels of gene modification in non-human primates (NHPs).
The pre-clincial data presented validates the application of genome editing strategy in large animals and suggest that Sangamo can achieve circulating levels of protein above the threshold for therapeutic effect thereby providing a potential cure of a range of monogenic diseases.
Sangamo further optimized its ZFP technology to demonstrate a single systemic treatment which enables stable liver-specific production of replacement protein.
We remind investors that Sangamo has a collaboration agreement with Shire (SHPG) in 2012 to develop therapeutics for hemophilia, Huntington's disease and other monogenic diseases.
The company has a pipeline of ZFP Therapeutics. The most advanced ZFP Therapeutic candidate, SB-728-T, is being developed for the treatment of HIV/AIDS with data expected later in 2013.
Sangamo currently carries a Zacks Rank #4 (Sell). Some of the better-ranked stocks which look attractive at current levels include Actelion (ALIOF) and Aeterna Zentaris Inc. (AEZS). Both carry a Zacks Rank #1 (Strong Buy).