This presentation contains forward-looking statements within the - - PowerPoint PPT Presentation

this presentation contains forward looking statements
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This presentation contains forward-looking statements within the - - PowerPoint PPT Presentation

This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements include, but are not limited


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This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements include, but are not limited to, the intended effect of SB-913, the design of the Phase 1/2 clinical trial and expected timing for release of data; the anticipated clinical development milestones; and the potential of SB-913 to successfully treat MPS II. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties. Factors that could cause actual results to differ include, but are not limited to, the potential for ZFNs to not work as intended, the dependence on the success of clinical trials of lead programs, the lengthy and uncertain regulatory approval process, uncertainties related to the timing of initiation and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of SB-913, uncertainties related to the initiation and completion of clinical trials, the reliance on partners and other third-parties to meet their

  • bligations. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist

in Sangamo’s operations and business environments. These risks and uncertainties are described more fully in Sangamo’s Annual Report on Form 10-K and Quarterly Reports on Form 10-Q as filed with the Securities and Exchange Commission. Forward-looking statements contained in this presentation are made as of the date hereof, and Sangamo undertakes no

  • bligation to update such information except as required under applicable law.

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IDS

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albumin gene

H TG H

transgene transgene

+

zinc finger nucleases

Strong albumin promoter

AAV vectors

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CTA granted Plan to initiate U.K. study by YE 2018 INDs open Study initiated 7 sites active 5 subjects treated (1 at highest dose)

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US

  • Orphan Drug
  • Fast Track
  • Rare Pediatric Disease

EMA

  • Orphan Medicinal Product
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– –

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