SGMO has 6 clinical stage zinc-finger platform gene editing indications in its pipeline ( CRSP has one )
Zinc-finger gene editing may be preferable to CRSPR due to SGMOs head start and due to concerns about CRSPR off target editing.
In mid-2018 PH1/2 data in Hemophilia and MPS will either validate or invalidate the zinc-finger gene editing platform.
If these two read-outs are positive, SGMO could easily double its MC. It is trading at 18.64 as I type.
has partnerships with PFE and GILD, but standalone success could be driven by its self-directed programs.
Self directed programs come in 3 waves: 1. Fabry disease filing in mid-2018 2. Validation of AAV crossing the BBB: Tau reduction, ALS, FTLD and Huntington's 3 Oncology collaboration with Kite.
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