Horizon Discovery Establishes Two New Centers of Excellence for rAAV Genome Editing in Cambridge, UK

Horizon Discovery (Horizon), a leading provider of research tools to support the development of personalized medicines, today announced it has established two new Centers of Excellence (CoEs) for gene editing in Cambridge, UK. The new Centers are at the Gurdon Institute, Cambridge University with Prof. Steve Jackson FRS, and the Cambridge Institute for Medical Research with Prof. David Rubinsztein.

Prof. Jackson aims to establish Horizon’s rAAV genome editing technology (GENESIS™) as a core technology within his lab at the Gurdon Institute for generating isogenic cell models of cancer. They will focus on generating cell lines with specific mutations in genes involved in DNA repair and DNA damage signaling, to study their relationship to cancer progression.

Prof. Rubinsztein’s lab will apply GENESIS to generate isogenic cell line models for the study of autophagy (degradation of unnecessary or dysfunctional cellular components), which has been a focus since their discovery that autophagy regulates the levels a set of specific proteins that cause many neurodegenerative diseases.

“The increasing number of research groups of this caliber working with our genome editing technology is very exciting for Horizon,” said Dr Rob Howes, Principal Scientist, Horizon. “We are delighted to welcome both the Gurdon Institute and Cambridge Institute for Medical Research to our Centers of Excellence network and look forward to working with them on developing the cell lines.”

The new human isogenic cell lines generated by the CoEs will be exclusively licensed to Horizon in return for future product royalties. Horizon will also have an exclusive option to license new intellectual property developed. This forms part of Horizon’s strategy to generate at least 2,500 new X-MAN™ (gene X- Mutant And Normal) models of cancer, neurodegenerative, and cardiovascular disease. These models support drug discovery researchers in their efforts to understand how complex genetic diseases manifest themselves in real patients, and help rationalize many aspects of drug development, reducing the cost of bringing to market new personalized therapies.

These centers become part of the larger Horizon Centers of Excellence network, which includes high profile Institutions such as UCL, Yale University, the National Cancer Institute, Washington University in St Louis and the National Cancer Centre, Japan.

Horizon sponsors an online community portal, www.rAAVers.org, for scientists working with rAAV-mediated and other forms of genome editing.