UniQure and Gen-X Enter Partner to Develop Novel Gene Therapy Promoters

Rare Daily Staff

UniQure and Gen-X have entered into an exclusive research collaboration and license agreement to identify novel synthetic promoters, which are used to control the expression of therapeutic genes, for use in UniQure’s gene therapy products.

The agreement also gives UniQure an option to acquire Gen-X.

UniQure’s pipeline of experimental gene therapies several rare diseases, including hemophilia A, hemophilia B, Huntington’s disease, Fabry disease, spinocerebellar ataxia type 3, among others.

Gen-X’s proprietary SuRE technology performs functional annotation of the non-coding genome by measuring the potential of hundreds of millions of DNA elements for their ability to serve as promoters or enhancers, driving the gene expression with a range of unique properties for gene therapy applications. Gen-X, a spinoff from the Netherlands Cancer Institute, says its technology is unique among other promoter-identification technologies in finding non-coding promoter elements throughout the genome at remote sites where activity is regulated.

“Novel promoters are an important area of innovation in gene therapy with the potential to enable valuable functionality and improve treatment options,” said Jonathan Garen, chief business officer of UniQure.  “With the unique SuRE methodology developed by Gen-X, we will now leverage our modular technology platform to potentially identify new promoters and enhancers that drive gene expression in the right cell-type and under the right conditions.”

During the collaboration, Gen-X will work exclusively with UniQure to develop promoters for the UniQure research pipeline. Through the SuRE technology, the Gen-X promoter library is designed to identify novel promoters to regulate gene activity and, if needed, conditionally control protein production. The parties will also explore further development of AAV vector libraries containing fragments of the non-coding human genome. Active promoter elements can be directly identified following administration of these libraries in vivo.

“We believe that combining our platform with UniQure’s leading expertise in AAV gene therapy will accelerate the development of advanced gene therapy candidates,” said Joris van Arensbergen, founder and CEO of Gen-X.

UniQure has an option to Gen-X for a pre-agreed price during the research portion of the collaboration. Financial terms on the research collaboration and license agreement were not disclosed.    

Photo: Jonathan Garen, chief business officer of UniQure