Sangamo Therapeutics (SGMO) has delivered excellent news across their proprietary ZFN drug development platform, asserts biotech expert John McCamant, editor of The Medical Technology Stock Letter.

The company report with promising human data from both the Hemophilia A program with Pfizer (PFE) and Beta Thal with Sanofi SA (SNY). 

In addition, the company has expanded their internal gene therapy manufacturing capacity and inked an agreement with longtime manufacturing partner Brammer signaling both confidence in clinical success and the need to prepare for multiple commercial launches. 

In the unexpected update, SGMO provided impressive proof-of-concept human data for two separate programs and significantly expanded the company’s manufacturing footprint. 

The take home message is that SGMO has a high level of confidence in their clinical success and needs to be prepared for multiple commercial launches as their platform has delivered successful POC data in two major programs that significantly de-risks the entire ZFN pipeline. 

With the initial data and partner PFE’s blessing/support, SGMO is clearly in the hem A races — competitors will have to take note. The Hem A gene therapy data is excellent and looks at least as good as BMRN’s valrox while also providing a positive read through for the Fabry program (also gene therapy),  that is expected to enter the clinic this year. 

The ST-400 data in Beta Thal data also looks quite strong and, while only one patient, it appears to measure up well against BLUE’s treatment in the most difficult population while also providing a positive read through for the sickle cell anemia and off the shelf CAR-T programs that are also both set to enter the clinic this year. 

In our view, the sum of this data is substantial as it provides broad validation for SGMO’s proprietary ZFN platform that should result in improving previously skeptical sentiment on the Street.

With the plethora of positive and de-risking news, SGMO is now a "buy" under $20 (up from $15) with a target price of $30 (up from $25). 

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