PJ Analyst Schimmer " even modest penatration of sickle cell market will be worth over $1 Billion"

To flesh out the report that JBWIN posted on InvestorVillage this morning, I want to add a comment found on the analystratings.com website. This Website contains a quote from PJ analyst Joshua Schimmer that reiterates even a minor penetration of the sickle cell market is worth $1 Billion dollars. http://www.analystratings.com/2015/04/30/biogen-or-bluebird-which-will-lead-in-scd/402054/

"Schimmer also analyzes the pipeline drug developed in partnership between Biogen and Sangamo BioSciences (NASDAQ: SGMO), though he focuses his analysis on Biogen. The two companies are working on a ZFN approach to target BCL11a, meaning the use of artificial enzymes to target a specific protein. Schimmer notes that this approach offers “a viable alternative” to LentiGlobin. If the product is successful, Schimmer believes that “even modest penetration” in the market will deliver over $1 billion. "


I also wanted to reemphasize the point that the PJ analysts made yesterday, that Sangamo has a very good chance of its therapy succeeding.


Piper hosted a call on sickle cell. The focus was largely on what is likely to be technically required for a gene therapy approach to work. Regarding SGMO, the analysis seems to indicate that knock-out of Bcl11a is likely to succeed, with the ever-present caveats of impact of off-target DNA breaks as well as off-target effects of the desired genome modification. The above mentioned erythroid-specific-enhancer knockout for Bcl11a we think typifies the sophistication of Sangamo’s approach to gene therapy. In any case, the Piper analysts estimate that roughly 10-30% bone marrow chimerism could rescue sickle cell, and our discussions with SGMO suggest that level of rescue should be within reach.