Horizon Discovery Establishes Three New Centers of Excellence in rAAV-mediated Cellular Genome Editing Dana-Farber Cancer Institute, University of Liverpool, and Babraham Institute join CoE network

25 Jun 2012

Cambridge, UK, 25 June 2012: Horizon Discovery (Horizon), a leading provider of research tools to support the development of personalized medicines, today announced it has established three new Centers of Excellence (CoE) for rAAV gene editing, at Dana-Farber Cancer Institute, in Boston, MA, the University of Liverpool, UK and the Babraham Institute, in Cambridge, UK. Through the CoE program, Horizon will support Dana-Farber Cancer Institute and the University of Liverpool in their application of Horizon’s propriety gene engineering technology, GENESIS™, to generate human isogenic cell lines incorporating genes involved in the development of specific diseases, with a focus on cancer.

The Dana-Farber CoE will focus on epigenetic mechanisms involved in the differentiation of normal stem cells and development of normal tissues, and how abnormalities in this process lead to initiation and progression of tumors. They will also develop isogenic cellular models to understand the roles of specific epigenetic genes and their role in breast cancer.

The University of Liverpool is interested the Ras family of proteins (N-, H- and K-Ras) which are involved in many key cellular processes, and how they produce different biological outputs despite their high degree of identity,. The University will produce isogenic cellular models that express different forms of these Ras proteins from their endogenous loci.

The Babraham Institute will use Horizon’s rAAV gene editing technology to understand the different PI3K isoforms and other components of the PI3K signaling networks. PI3K enzymes are critical regulators involved in many cellular responses.

“We are delighted these three new world class institutes have joined our Centers of Excellence program,” said Dr Rob Howes, Principal Scientist, Horizon. “Horizon is hoping to continue expanding the CoE network through 2012, and we look forward to working with an increasing number of excellent scientific groups.”

Horizon has also announced that it has launched a conference travel bursary awards scheme to support PhD students and early-stage researchers working with X-MAN cell lines. Once per quarter, scientists will be invited to submit an abstract of work they intend to present at an upcoming conference. Horizon will select up to five of these entries per quarter, to which they will award bursaries of £250 for national conference attendance, and £500 for international conference attendance. Scientists successful in their application are asked to write a one page report following the conference, which along with their poster will be posted to Horizon’s rAAVers.org website.

Horizon’s CoE program encompasses academic and not-for-profit research groups or laboratories to which Horizon commits resources to provide training and open access to its proprietary rAAV-mediated human gene-editing platform, GENESIS™. Horizon also recently launched an online support site, www.rAAVers.org, for scientists working with rAAV-mediated genome editing.

The new human isogenic cell lines generated by the CoE’s 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 2500 new X-MANTM (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.

The centers are part of the GENESIS Gene Editing Consortium, which includes rAAV GENESIS pioneers the University of Washington, the University of Torino, Johns Hopkins University, the Translational Genomics Institute (TGen), the University of Minnesota, the University of Maryland, Case Western Reserve University and the University of Pittsburgh.