Precision BioSciences, Inc. (DTIL)
First Day Turnover
We are a genome editing company dedicated to improving life through our groundbreaking proprietary genome editing platform, “ARCUS.” We leverage ARCUS in the development of our product candidates, which are designed to treat human diseases and create healthy and sustainable food and agricultural solutions. We believe the versatility and breadth of ARCUS support our ability to develop products across the spectrum of biotechnology. We are actively developing product candidates in three innovative and high value areas where we believe our technology has the potential to overcome the limitations of other genome editing technologies: allogeneic CAR T immunotherapy, in vivo gene correction, and food. The U.S. Food and Drug Administration, or FDA, recently accepted our investigational new drug, or IND, application for our first gene-edited allogeneic CAR T cell candidate targeting CD19. We are currently screening patients for our planned Phase 1/2a clinical trial in patients with relapsed or refractory, or R/R, B-cell precursor acute lymphoblastic leukemia and R/R non-hodgkin lymphoma and expect to dose our first patient in this trial in April 2019. We believe this trial will be the first clinical investigation of an allogeneic CAR T therapy for non-hodgkin lymphoma. We believe our proprietary, one-step engineering process for producing allogeneic CAR T cells at large scale in a cost-effective manner will enable us to overcome the fundamental challenges of manufacturing that have limited the CAR T field to date.
We are dedicated to improving life. Our goal is to broadly translate the potential of genome editing into permanent genetic solutions for significant unmet needs. Our strategy to achieve this goal includes the following key elements:
• Create a fully integrated genome editing company capable of delivering solutions that address unmet needs impacting human health. We believe that, to be a leader in the field of genome editing and maximize the impact of our ARCUS genome editing platform, we must be able to control those elements of our business that may provide us with certain strategic advantages or operational efficiencies. We intend to continue to invest in comprehensive research, development and commercial capabilities that provide control and oversight of our product candidates from discovery through commercialization.
• Accelerate advancement of our first four allogeneic CAR T immunotherapy product candidates while investing in the research and development of additional allogeneic CAR T programs. We believe that we have developed the first allogeneic CAR T cell manufacturing platform capable of producing drug product at scale today. We have selected four validated CAR T cell targets that we believe offer the greatest chance of clinical success for our initial product candidates, which we intend to rapidly advance into clinical development. In November 2018, the FDA accepted the IND for our lead CAR T cell product candidate targeting CD19. We are currently screening patients for our planned Phase 1/2a clinical trial in patients with R/R B-cell precursor ALL and R/R NHL and expect to dose our first patient in this trial in April 2019.
• Advance in vivo genetic correction programs into human clinical trials. In our preclinical studies, we observed the high-efficiency and tolerability of in vivo genome editing in a non-human primate, as published in Nature Biotechnology in July 2018. To our knowledge, we are the first company to complete this milestone,
which we believe to be critical to successful in vivo genome editing therapeutic development. We intend to build on this early success by diligently advancing a diverse portfolio of preclinical in vivo gene correction programs through additional large animal studies, focusing initially on gene targets occurring in the liver and eye. Based on the results from these large animal studies, we intend to advance a subset of these programs to human clinical trials.
• Build a food business focused on developing products designed to improve human health and respond to the impacts of climate change. We believe that rapidly changing consumer preferences and food insecurity resulting from population growth and climate change will drive significant demand for genome-edited food products. We are building a fully integrated discovery and development platform that combines genome editing, gene discovery, plant transformation and high-throughput testing to enable accelerated innovation in the food industry. We employ a business model that is focused on collaborating with critical stakeholders within the supply chain from the outset of any given project. We believe that this approach will enable us to successfully respond to growing unmet needs within food supply to build a human health-focused business in a capital-efficient manner.
• Continue investing in the optimization of ARCUS and enabling technologies. We believe that a key to our future success is the quality of the genome editing tools that we produce. Since our founding, we have devoted ourselves to continuously refining the precision and efficiency of our core genome editing platform. We intend to continue this investment in ARCUS while surrounding it with enabling technologies and expertise to retain what we believe is a leadership position in the field.
• Create an environment that is a destination of choice for premier talent within the life sciences industry. We believe that we currently have among the deepest and strongest skill set within the genome editing industry and credit much of our past success to our commitment to our team and culture. Our future success will depend on our ability to continue to attract and retain world-class talent within our markets of interest. We intend to consciously invest in fostering an environment within our company that is both challenging and supportive and inspires our team to broadly translate genome editing into permanent genetic solutions.
• Expand the breadth of our operations through additional product platforms and strategic relationships. We believe that the ARCUS genome editing platform has broad utility beyond our current areas of focus. We intend to invest in the development of additional product platforms and seek collaborations with companies with expertise in areas outside of our current target markets to maximize the value of our company.