uniQure incorporates Novel Synthetic Promoter Developed by Synpromics in New Preclinical Target
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Synpromics is pleased to hear that its partner, uniQure, has announced the incorporation of its promoter, developed by Synpromics, in a new preclinical research target. This follows recent positive data, presented at the ESGCT world congress, demonstrating that this highly selective liver promoter is at least eight times more potent than the current industry standard.
uniQure’s new gene therapy aims to treat all hemophilia A patients, one of the two main types of hemophilia. Hemophilia A is caused by missing or defective Factor VIII, a protein essential for clotting. Approximately 30 percent of patients with severe hemophilia A will develop an inhibitor that neutralises the infused Factor VIII (FVIII) activity. This patient population has in the past been excluded from gene therapy approaches in clinical development, however, uniQure's new candidate has been demonstrated in preclinical studies to circumvent inhibitors to FVIII. This data shows that the candidate may lead to durable expression in hemophilia A patients and may provide long-term prevention of bleeds. The promoter, developed by Synpromics, is a key component of this new candidate.
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 collaborates with a wide range of pharmaceutical companies, working with these partners to develop highly selective synthetic promoters which enable greater control of gene expression and can be incorporated into gene and cell therapies for a range of diseases.
uniQure’s new gene therapy aims to treat all hemophilia A patients, one of the two main types of hemophilia. Hemophilia A is caused by missing or defective Factor VIII, a protein essential for clotting. Approximately 30 percent of patients with severe hemophilia A will develop an inhibitor that neutralises the infused Factor VIII (FVIII) activity. This patient population has in the past been excluded from gene therapy approaches in clinical development, however, uniQure's new candidate has been demonstrated in preclinical studies to circumvent inhibitors to FVIII. This data shows that the candidate may lead to durable expression in hemophilia A patients and may provide long-term prevention of bleeds. The promoter, developed by Synpromics, is a key component of this new candidate.
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 collaborates with a wide range of pharmaceutical companies, working with these partners to develop highly selective synthetic promoters which enable greater control of gene expression and can be incorporated into gene and cell therapies for a range of diseases.
