uniQure incorporates novel synthetic promoter developed by Synpromics in new preclinical target

EDINBURGH, UK - Dec 5, 2018 - Synpromics is pleased to hear that its partner, uniQure (NASDAQ:QURE), 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.

uniQures 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.

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For further information, contact:

Corporate Contact:
Dr Michael L Roberts, CSO and Founder
Synpromics Ltd
T: +44 (0)131 651 9662
E: Michael.roberts@synpromics.com

PR Contact: 
Deborah Cockerill / Juliette Craggs
Sciad Communications Ltd
T: +44 (0)20 7470 8801
E: synpromics@sciad.com

Notes to Editors

About Synpromics
Synpromics is the leader in gene control, improving human health by enabling safer, more effective cell and gene medicines through proprietary genomics, bioinformatics and intelligent data-driven design. The Company has developed PromPT®, its multi-dimensional bioinformatics database, that enables product-specific promoter design and selection empowering the next generation of cell and gene based medicines and bioprocessing applications. The Company operates in a diverse range of fields, including broad applications in cell and gene based medicine, biologics manufacturing and viral vector bioprocessing. Current partners include Audentes, Biomarin, uniQure, AGTC, Solid Biosciences, Homology Medicines Inc, Adverum Biotechnologies, GE Healthcare and Sartorius-Stedim Cellca, as well as numerous undisclosed partners in the pharmaceutical sector.

About synthetic promoters
Naturally occurring promoters have evolved for biological functions but have limitations when utilised in industrial or therapeutic applications. 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, tissue or environmental condition. Each synthetic promoter represents a novel invention and thus can be patented.

For more information visit www.synpromics.com