SGMO Shows Early Progress in Gene Therapy and Ex-Vivo Programs


Sangamo (SGMO) provided an update today on its clinical programs, with its first clinical data for its Phase I Hemophilia A (gene therapy) (See FIG. 1A below) and B thalassemia (ex vivo editing) programs. In Hem A, impressive early data was presented with 2 highest dose patients reaching normal range within 8 weeks and no bleeding events after treatment out to week 12 (1st high dose patient) or week 6 (2nd high dose patient). SMGO's Hem A program is licensed to Pfizer and its B-thal program is licensed to Sanofi. In B-thal first patient transfused showed expression of fetal hemoglobin for proof of concept.

Overall good update for SGMO but behind competitors such as Biomarin (BMRN) in Hem A gene therapy (See FIG. 1B below) and Bluebird (BLUE) in Beta-Thal gene therapy.

FIG. 1A SGMO Hem A gene therapy data presented in today's investor call

FIG. 1B Competitor BMRN Hem A gene therapy data presented at JP Morgan conference in January 2019

However, speed of action in Hem A (See FIG. 1A vs. 1B) and precision in B Thal could help differentiate with much more data needed to better understand competitive positioning.

No new data was provided by SGMO for its most noteworthy clinical program, its Zinc Finger in vivo gene editing program. Thus far, the data has shown some proof of concept, but limited potency questions whether this technology can be clinically meaningful. Next generation improved construct trials starting later this year should help answer that question of whether SGMO's in vivo gene editing technology has commercial promise.

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