LSIPR Newsletter 04:14


IP STRATEGY: HORIZON DISCOVERY 15


Horizon Discovery’s cell line-creating technology has the potential to advance personalised medicine. But how does the gene editing company stay competitive in a crowded field? LSIPR found out.


Editing the genome


As a better understanding of how genes function in disease is developing, personalised medicine is becoming a reality for more patients.


Since the 1990s, advances in technology have allowed the manipulation, or ‘editing’, of the human genome so that the function of certain genes and how genetic mutations cause the onset of disease can be studied.


Tere are several varieties of this so-called ‘genome editing’. One of the most recent methods, the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system, uses a bacterially-derived protein and synthetic guide ribonucleic acid (RNA) to make breaks in targeted parts of the genome.


UK-based Horizon Discovery uses a combination of genome editing methods to create cell lines—what it calls ‘patients in a test tube’—which model the genetic anomalies found in patients with cancer and other diseases, for use by companies in the business of drug discovery and development, as well as in clinical diagnostic development.


“Gene editing allows Horizon Discovery to manipulate the genome,” explains Eric Rhodes, vice president of research and development, and chief technology officer at the company. “We can essentially recreate patient populations.


“As long as you have a unique patient population for a disease, you can recreate that disease in a cell and use that to test drugs to see if they have an effect.”


www.lifesciencesipreview.com Personalised medicine


Te technology’s potential application as a research tool in the development of personalised medicine is clear. It is an approach that can stop patients receiving treatments that will not work for them, while reducing the chance of an adverse effect.


Horizon Discovery brings together CRISPR, another genome editing method called Zinc Finger Nucleases, and its own proprietary recombinant adeno-associated virus (rAAV) technology to make gene point mutations, knockouts, deletions and insertions on the genome, and create a variety of different cell lines. Drug development companies can use them to see how a population will respond to a medicine before introducing it to a patient.


As gene function is oſten a factor in the development of cancer, Horizon Discovery has been focusing on creating research tools to assist in the development of treatments for the disease, although it is not limiting itself to oncology.


“Outside oncology there are other things you can do, such as modify cells to make them resemble monogenic diseases, such as Huntington’s or cystic fibrosis.


“Different types of gene editing are used to make specific cells, such as a cell that is typical of a certain type of cancer,” Rhodes explains.


“For example we may take a kidney cell and recreate kidney cancer by mutating a gene. We sell the non-mutated version of the cell next to the mutated version, so researchers can do studies to see the difference between the two when they’re treated with a drug.”


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