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Re: SGMO TranscriptIn the immediate future, we are focused on moving four proprietary programs into the clinic: hemophilia A and B, MPS I, and MPS II. Hemophilia B, MPS I and MPS II are all in-vivo genome editing programs. Our hemophilia A program uses an AAV including a [Factor VIII] cDNA which we believe, based on published non-human primate data, has a potential to be best-in-class. While this is something of a departure from a previous ZFP focus, going forward we will use our considerable experience in gene therapy, genome editing and gene regulation, to develop the best medicines that we can to serve patient need. We will complete the follow-up of subjects in our HIV T cell program, and will look for an opportunity to present and publish the data from these studies. Our stem cell study is ongoing as an investigator-sponsored study at City of Hope, and funded in part by CIRM, and we will continue to prosecute this trial through to its completion. However, we will not commit to additional studies or investment in HIV without a partner. In terms of the next wave of programs in our pipeline, as you know, we are also working with Biogen to develop ex-vivo genome editing applications for beta thalassemia and sickle cell disease, and expect to have more information to share on the progress of these programs in future calls With positive data from the in vivo genome editing programs that are entering the clinic in the next months, we have additional proprietary programs that'll be thoughtfully moved into our clinical pipeline. We have very interesting data in our other lysosomal storage disorder, particularly our Fabry disease program. We also have considerable experience in genome editing of T cells, and continue to see T cell oncology as a promising area for future collaborative ventures. ..... We have initiated the first trial of our in vivo genome editing platform, our hemophilia B study, and expect our MPS I and MPS II studies to be opened in early 2017. All of these trials are dose escalation studies, and will enroll adult subjects. However, our goal with sufficient dosing and safety data is to move rapidly into pediatric patients. We believe that our ability to make a permanent change to the genome is where the specific advantage of our genome editing approach lies, and makes this an obvious choice for a potentially life-long therapeutic solution. We expect to have preliminary data from these trials at the end of 2017 and when we have clinically-relevant data, we will present it at an appropriate scientific or medical meeting, as has been our previous practice. So, in summary, the third quarter was a very busy and productive period for us, with steady progress in terms of initiating clinical activities for proprietary programs, and in the reorganization of the Company and its procedures and programs. Some of this has been more visible internally than externally, however, it will all positively impact the development and advancement of our therapeutic programs. While we are investing heavily in our organization, we expect to end 2016 with at least $140 million in cash and cash equivalents. We are in a position to file an IND application for the hemophilia A program this year, and in the next year to achieve proof of concept in clinical studies which will provide not only safety and early efficacy data, but drive significant value. My goal and my responsibility is to build an organization as both mature and reliable, but nimble and imaginative. We will take small steps, but step-by-step we will achieve what we promise, and in doing so make significant progress in bringing products through clinical development to the patients whose lives they can change, creating shareholder value in the process. We look forward to updating you at several upcoming investment banking conferences. We will be presenting at the Jefferies Global Healthcare Conference, the Piper Jaffray Healthcare Conference in November, and at the 35th Annual JPMorgan Healthcare Conference in January of 2017, all of which will be webcast and available on the website. We will also present pre-clinical data from our hemophilia program at the annual meeting of the American Society of Hematology, or ASH, in early December. |
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