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MTSL (BioInvest) Issue 860: SGMO - company update

 SGMO – Presents Preclinical Oncology Platform; Partnership Soon
 
SGMO presented preclinical data demonstrating the Company’s engineering capabilities in T cell genome editing using zinc finger nucleases (ZFNs) at the Immuno-Oncology Summit in Boston (8/28-9/1 http://www.immuno-oncologysummit.com). The talk, entitled “Engineering T Cells for Cancer Therapeutics,” showed off Sangamo’s cell therapy expertise and included data from the Company’s recent research in the development of allogeneic T cell immunotherapies utilizing ZFN editing techniques. Highlights of the platform include:
1. Elimination of endogenous T cell receptor (TCR) expression by knock-out of the TCR alpha constant locus (TRAC) with greater than 90% efficiency
2. Elimination of human leukocyte antigen (HLA) Class I proteins by knock-out of b2-microglobulin (B2M) with greater than 90% efficiency
3. Co-delivery double knock-out of TRAC and B2M with greater than 90% efficiency
4. Targeted integration of a green fluorescent protein (GFP) expression cassette into either the TRAC or B2M locus with ~90% efficiency with double knock-out of TCR and HLA Class I
5. CD19 chimeric antigen receptor (CAR) integrated into either the TRAC or B2M locus demonstrated strong antigen specific killing with a clear dose response
6. No evidence of off-target cleavage in human primary T cells
Through the company’s original T cell genome editing programs, Sangamo has accumulated clinical expertise in therapeutic T cell development and manufacturing of clinical grade materials. In approximately 100 HIV patients treated with ZFN-edited T cells, SGMO has demonstrated persistent engraftment and established a strong safety profile for the duration of observation (3 years post infusion). SGMO’s technology has the potential to create off the shelf CAR-Ts and significantly improve the manufacturing process which would create a much larger market opportunity.

Gene editing remains one of the more exciting drug development technologies today. The Company is ideally suited for allogeneic or autologous approaches with its ex-vivo clinical and manufacturing experience in cell engineering and development. Dr. Lee’s slides are here (http://c.eqcdn.com/sangamo/files/documents/GLee_IO+Summit+Aug2017+vFINAL.pdf).

We believe SGMO is well-positioned yet unrecognized by most investors in immuno-oncology. A corporate collaboration with an experienced cancer company is possible by the end of 2017. The Company is already engaged with potential partners and a deal would not only bring favorable economic terms but more importantly, validate SGMO as a participant in the I/O world. In our view, SGMO’s experience in T-cells and its proprietary ZFN technology uniquely position the company to be a leader in developing the next generation CAR-Ts.