Horizon Discovery and University of Leicester Form Center of Excellence in rAAV-mediated Genome Editing

Posted by pt91 at Jul 17, 2012 10:49 AM |
Collaboration will focus on developing isogenic models of cardiovascular disease
Horizon Discovery and University of Leicester Form Center of Excellence in rAAV-mediated Genome Editing

Professor Nilesh Samani, Head of the Department of Cardiovascular Sciences.

Issued by Horizon Discovery on 16 July 2012

Cambridge and Leicester, UK, 16 July 2012: Horizon Discovery (Horizon), a leading provider of research tools to support the development of personalized medicines, today announced it has established a Center of Excellence (CoE) for gene editing at the University of Leicester, UK. The program will focus on the creation of isogenic cell lines that contain ‘risk’ or ‘protective’ SNPs associated with cardiovascular disease, identified from GWAS studies.

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™. The University of Leicester is the first of Horizon’s Centers of Excellence to focus on cardiovascular disease.

The new human isogenic cell lines generated by the University of Leicester 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-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.

“Horizon’s rAAV-mediated genome editing can fundamentally alter the genome of cells, leading to the creation of patient-relevant disease models. The use of these models in oncology is well established, and has predicted patient responses to targeted therapies both during drug development and in the clinic,” commented Dr Rob Howes, Principal Scientist, Horizon Discovery. “By developing the application of this technology to other disease areas such as cardiovascular disease, we aim to lead the development of personalized medicines in these areas.”

Prof Nilesh Samani, British Heart Foundation Professor of Cardiology and Head of Department of Cardiovascular Sciences at the University of Leicester, commented: “The opportunity to apply Horizon’s GENESIS technology for development of cardiovascular disease cell lines offers an exciting progression in the study of the disease. We look forward to working with Horizon on this program.”

Dr Tom Webb, British Heart Foundation Lecturer in Cardiovascular Genomics, at the University of Leicester, said: “We are delighted to have this partnership with Horizon. By using rAAV-mediated genome editing we can create cell models to investigate how specific genetic variants contribute to the development of cardiovascular disease.”

The Centers of Excellence are part of the GENESIS Gene Editing Consortium, which includes rAAV GENESIS pioneers the National Cancer Institute, Cambridge University, Yale University, and Dana-Farber Cancer Institute.

Horizon recently launched an online support site, www.rAAVers.org, for scientists working with rAAV-mediated genome editing.

ENDS

Notes to Editors

Contacts:

At Horizon:

Dr Rob Howes

Principal Scientist

Tel: +44 (0) 1223 655 584

Email: r.howes@horizondiscovery.com

Media enquiries:

Katie Odgaard

Zyme Communications

Tel: +44 (0)7787 502 947

Email katie.odgaard@zymecommunications.com

At the University of Leicester:

Nilesh Samani

British Heart Foundation Professor of Cardiology

Head of Department of Cardiovascular Sciences, University of Leicester

Director Leicester NIHR Biomedical Research Unit in Cardiovascular Disease

Email: njs@le.ac.uk

Tom Webb

British Heart Foundation Lecturer in Cardiovascular Genomics

Department of Cardiovascular Sciences, University of Leicester

Tel: +44 (0)116 258 3716

Email: tw126@le.ac.uk

About Horizon Discovery http://www.horizondiscovery.com/

Horizon Discovery Limited (Horizon) is a leading provider of research tools to support translational genomics research and the development of personalized medicines. The Company’s proprietary rAAV gene‐editing technology, GENESIS™, is industry leading. Using GENESIS, Horizon is able to alter any endogenous gene sequence of a human or mammalian cell‐line quickly, reliably and without introducing unwanted and confounding genotypes and/or phenotypes.

Horizon has applied GENESIS to create over 400 X‐MAN™ cell lines, the world’s first source of genetically‐defined and patient‐relevant human cell lines, accurately modeling the disease‐causing mutations found in cancer patients. These ‘patients‐in‐a‐test‐tube’ are being used by academic and industry leaders to identify the effect of individual or compound genetic mutations on drug activity, patient responsiveness, and resistance, leading to the successful prediction of which patient sub‐groups will respond to currently‐available and future drug treatments. This enables the design of shorter, more focused, and less expensive clinical trials, ultimately providing the tools to identify the ‘right drugs’ for the ‘right patients’ based upon the unique genetic mutations that define their disease.

In addition to the X‐MAN cell lines, Horizon provides GENESIS and X‐MAN™ derived products and services, with industrial application in: bio‐pharmaceutical process optimization; clinical diagnostic development; drug discovery & development; and the provision of reference standards for genomic‐based clinical research platforms.

GENESIS and XMAN are registered trademarks of Horizon Discovery Limited.

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