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While we wait...Snip from 3rd quarter CC As part of our recognition of the clinical group, I am putting a flatter management structure in place. I will not hire a new head of R&D, but will have Michael Holmes, our Head of Research, report directly to me, and hire a new Head of Clinical Development, a position for which we are actively recruiting and expect to hire soon. We are developing a regulatory strategy for our program, which includes expansion beyond the US for hemophilia and NPS clinical studies, and a pathway to treatment of the pediatric population in these indications. We see pediatric patients as potentially benefiting the most from our genome-editing approach, and we are committed to moving these therapeutics into younger patients where the medical need is greatest. This must be carried out in a prudent fashion, however, particularly for the MPS disorders. There's a striking need for better options for these children. As I mentioned earlier, our hemophilia B program was awarded orphan drug designation in July of this year. This designation is important, as it means that we are eligible for a seven-year period of US marketing exclusivity upon approval, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of the Prescription Drug User Fee Act, PDUFA, filing fees. We are also developing a research organization that can not only support efforts to generate IND enabling studies, but still has the capacity to great and innovative science for which it is rightly famous. The team is already beginning to re-find its mojo, and I look forward to sharing in more detail some of the game-changing improvements that they have made to the technology, that will not only benefit immediate programs like the Biogen collaboration, but will have far-reaching consequences for the power of the entire populations and our programs going forward. We have a mandate to continue to build our technical operations group, which is being bed by a recent addition to our team, Dr. Mohammad El-Kalay, who brings over 25 years of operational management experience in biologics manufacturing to our organization, and will be responsible for overseeing all processes including external manufacturing. We have also been focused on corporate hygiene, a term which we use to include a variety of activities including strengthening processes and procedures for development and prosecution of our pipeline; optimization of our organizational structure; general employee care and maintenance; and while making Sangamo a great place to work. And last but not least, we're also working hard to improve our image and the communication of our strategy, expected milestones and progress. We will behave differently, setting clear goals and achieving them on time. My anticipation is that we will not just look different for the sake of looking different, but our new face will truly reflect the energy, enthusiasm and creativity of our people, the power of the technology that we have built, and the potential that we can realize to make a significant difference to the lives of patients and families. As I mentioned at the beginning of the call, we laid out plans in each of these areas for the Board who are fully-supportive, and I look forward to further discussing the fruits of this plan with you in upcoming calls. In 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. |
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Msg # | Subject | Author | Recs | Date Posted |
84934 | Re: While we wait... | ErnD | 6 | 2/2/2017 8:51:54 AM |