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Re: DanWebzster post# 103

Monday, 04/16/2018 11:40:21 AM

Monday, April 16, 2018 11:40:21 AM

Post# of 438
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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