Synthetic gene promoter targets hemophilia

Novel approach appears to circumvent inhibitors to factor VIII in early clinical studies

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AMSTERDAM & EDINBURGH, U.K.—Two companies utilizing gene therapies to target disease, uniQure and Synpromics, are sharing resources in a new collaboration to target hemophilia. As part of this, uniQure recently announced that it will be utilizing one of Synpromics’s synthetic promoters in its preclinical research area looking at a novel approach to treating hemophilia A.
People affected with hemophilia are born missing some or all of a type of protein called clotting factor, leading to bleeding disorders. People with hemophilia A are missing some or all of the clotting factor VIII, while people with hemophilia B are missing some or all of a different clotting factor called factor IX.
Some people with hemophilia A are affected because they make a dysfunctional version of the factor VIII protein, while the remainder are affected because they produce factor VIII in insufficient amounts. The number of people with hemophilia in the United States is estimated to be about 20,000, with hemophilia A occurring nearly four times more frequently than hemophilia B.
Netherlands-based uniQure aims to deliver curative gene therapies, with a current focus on the treatment of hemophilia and Huntington’s disease. Their latest gene therapy targets hemophilia A. About one-third of patients with severe hemophilia A develop an inhibitor that nullifies factor VIII (FVIII) activity. To date, gene therapy via naturally occurring promoters has not been successful for these patients. However, uniQure’s new treatment candidate incorporates a short, highly selective and powerful liver promoter that was developed by Synpromics, which appears to circumvent inhibitors to FVIII in early clinical studies, which may provide long-term prevention of bleeds.
Dr. Michael Roberts, chief scientific officer of Synpromics, commented, “The decision from uniQure to incorporate this promoter in a further candidate demonstrates their confidence in Synpromics’ technology, supported by a growing body of data. These recent steps mark further progress to seeing Synpromics’ technology reach the clinic and, ultimately, its inclusion in gene medicines.”
Synpromics, headquartered in Edinburgh, is an industry leader in synthetic promoter development, filling gaps where naturally occurring promoters have not been successful. Using proprietary genomics, bioinformatics and intelligent data-driven design, Synpromics has harnessed gene control, generating exciting possibilities in the creation of cell and gene therapy products. The PromPT database is a bioinformatics platform and library-focused DNA construction technology that allows the company to design, build and test synthetic promoters that regulate gene expression in a highly specific and machine-driven manner.
Synthetic promoters with DNA sequences not found in nature are designed to better regulate gene activity and precisely control protein production. Synpromics creates highly specific promoters designed to drive gene expression at the desired level and specificity in any cell type, in response to any biological or chemical stimuli or environmental condition. Each synthetic promoter represents a novel invention, and thus can be patented.
Synpromics and uniQure presented positive data about the new promoter at the European Society of Gene and Cell Therapy conference in October 2018. Those findings reportedly demonstrated that the highly selective liver promoter is at least eight times more potent than the current industry standard, providing uniQure with a powerful tool from which to drive the development of a variety of liver-directed gene therapies.
Commenting on the results, Roberts said, “This is fantastic data and shows the translatability of our company’s technology to advanced disease relevant models. This is the culmination of a productive partnership with uniQure in which we have worked closely to deliver a highly selective promoter, whose performance has exceeded initial expectations. This promoter enables uniQure to develop safer, targeted gene therapies for liver disease.”

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