Cellectis Announces Delivery of a Meganuclease Targeting the RAG1 Gene (Responsible for a Severe Immunodeficiency) to Professor LuigiNotarangelo’s research group at the Children’s Hospital Boston (USA)
BIOCITECH, France – November 15 2007 – Cellectis SA, the rational genome engineering company specializing in the production of meganuclease recombination systems and in meganuclease engineering, today announced the delivery of a meganuclease targeting the RAG1 gene to Professor Luigi Notarangelo’s research group at the Children’s Hospital Boston (USA).
The RAG1 gene is mutated in certain severe combined immunodeficiency (SCID) patients. To date, the only possible therapeutic response for these patients has been a bone marrow transplant, for reconstitution of their entire immune system. In 2000, new hope arose for patients with X-SCID (a type of SCID syndrome, see below), following promising gene therapy trials performed in France. However, several of the treated patients developed leukemia. These severe adverse events appear to be linked to insertion of the gene therapy vector in the vicinity of a particular gene. Meganucleases enable an alternative approach based on correction of the defective gene itself, rather than random insertion of a “gene drug”. The meganuclease technology should enable avoidance of the above-mentioned adverse event and should help make the gene therapy approach safer.
In August, Cellectis had already delivered a meganuclease to Professor Alain Fischer’s group (INSERM Unit U768 at the Necker Children’s Hospital in Paris) in order to test its potential ability to repair the defective gene in cell lines carrying a X-SCID mutation. X-SCID patients represent another class of SCID patients, in whom the disease is linked to a mutation in the IL2RG gene. Meganucleases targeting the RAG1 gene address another type of patient but this new collaboration demonstrated Cellectis’ emphasis on SCID syndromes in general. Indeed, Cellectis is strengthening its commitment to finding a treatment for this type of disease and, in the short- to mid-term, intends to evidence clinical proof of concept for its genome surgery approach in humans.
Cellectis SA is a world-leading company in genome engineering and genome surgery. The company is focused on developing and producing custom meganucleases for in vivo DNA surgery and also provides new tools for rational reverse genetics and targeted recombination. Cellectis’ products induce unique, site-directed, double-strand DNA breaks in a living cell and can be used in a wide range of biotechnological and therapeutic applications. To date, Cellectis has entered into more than 48 deals on its genome engineering technologies with major players in the pharma, biotech and agrobiotech industries. Cellectis is listed on the NYSE Euronext Alternext market (ticker code: ALCLS). For more information on Cellectis, visit our web site: www.cellectis.com
About Cellectis’ technology
A meganuclease is a (protein) molecule that cuts DNA at a highly precise site on a chromosome. Once DNA is broken, it has to be repaired by the cell’s natural endogenous maintenance systems. By providing a specifically engineered DNA molecule (called a repair matrix) which will be used as a template to repair the break, one can channel the repair pathway into an insertion, deletion or correction process. Thus, meganucleases can be used to trigger precise modification of specific genes in a variety of cells and organisms. By combining the meganuclease’s capacity to cut DNA and DNA’s ability to undergo repair, Cellectis is creating new generations of products for a wide spectrum of applications – including human health, since many genetic diseases result from a single mutation in a specific gene. Meganucleases can specifically target this same gene. In parallel, a DNA repair matrix (prepared by Cellectis and including a non-mutated copy of this gene) will be introduced into the cell.
Upon cleavage by the meganuclease, the repair matrix will be used as a template to restore a correct gene. By erasing the mutation, gene correction addresses the very cause of the disease, rather than its consequences.
About Cellectis’ collaboration policy
Cellectis’ policy is to foster research excellence in order to offer new solutions for genome engineering. To date, the major outcome of this effort has been the production of custom meganucleases that cleave genes of interest; this capability vastly expands the range of potential applications and is a prerequisite for therapeutic use. Whereas the core activity (i.e. the protein engineering itself) is usually conducted solely by Cellectis, upstream studies are often performed in collaboration with other major players in this field.
Cellectis’ Forward-Looking Statements
This communication expressly or implicitly contains certain forward-looking statements concerning Cellectis and its business. Such statements are based on assumptions and assessments made by Cellectis’ management in light of their experience and their perception of historical trends, current conditions, expected future developments and other factors they believe to be appropriate. They are not guarantees of Cellectis’ future performance and involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of Cellectis SA to be materially different from any future results, financial condition, performance or achievements expressed or implied by such forward-looking statements.
Cellectis is providing this communication as of this date and does not undertake to update any forward-looking statements contained herein as a result of new information, future events or otherwise. Potential risks and uncertainties which could cause actual results, financial condition, performance or achievements of Cellectis SA to differ from those contained in the forward-looking statements include, without limitation, the risks and uncertainties discussed in the Risk Factors (“facteurs de risques”) sections of the prospectus prepared by Cellectis approved by the French Autorité des Marchés Financiers (“AMF”) on January 22 2007 under visa number 07-023, available on the websites of the AMF (http://www.amf-france.org) [and Cellectis (http://www.cellectis.com)%5D.
For further information, please contact:
Cellectis S.A. Scientific Director
Frédéric Pâques, PhD.
Tel.: + 33 (0)1 41 83 99 00