PARIS, France I July 4, 2013 I Cellectis (ALCLS.PA) (ALCLS.PA), the genome engineering specialist, announced today the successful in vivo proof of concept testing of their flagship UCART19 product for curative therapy of leukemia.

 UCART19 is a revolutionary engineered T-cell product that leverages Cellectis’ core technologies in genome engineering. This standardized “off-the-shelf” therapy product is designed with a “chimeric antigen receptor” (CAR) to target and kill the most common types of acute and chronic leukemia and are engineered with Cellectis’ nucleases enabling the use of non-patient derived (allogeneic) T-cells. The results obtained show that the UCART19 product was able to eradicate human leukemia cells that had been transferred into mice, effectively curing the mice of tumors, and that the engineered T-cells performed as well as traditional CAR T-cells. 

 “These results are a crucial proof of concept for our engineered T-cell product strategy”, stated Andrew M. Scharenberg M.D., Cellectis therapeutics Chief Scientific Officer. “They validate our platform’s ability to mass produce T-cells that can be used to target any type of cancer”. 

“We believe that our recent advances in the development of our allogenic CAR T-cell platform will hopefully position Cellectis as a major player in this new emerging approach to treating cancer” said Mathieu Simon M.D., Chief Executive Officer of Cellectis therapeutics.

 The results announced today arise from collaboration with a University College London (UCL) multi-disciplinary research team led by Dr. Martin Pule, Dr. Karl Peggs and Prof. David Linch. Dr. Pule states “These results are an important step towards an ‘off-the-shelf’ immune therapy for acute and chronic leukemia.” 

About Cellectis
Founded in France in 1999, the Cellectis Group bases its work on highly specific DNA engineering technologies. Its application sectors are human health, agriculture and bio-energies. Cellectis was co-founded by André Choulika, its Chairman and CEO, and is now one of the world’s top companies in the field of genome engineering, with revenue of $27 million in 2012. Leading the field of pluripotent stem cells, Cellectis has developed expertise in drug discovery, toxicity testing, and regenerative medicine. Cellectis has a solid background in the large-scale handling of stem cells up until their maturation and differentiation into functional cell types. We employ a workforce of 230 people at 5 sites worldwide: New Brighton (Minnesota) & Cambridge (Massachusetts) in the United States, Gothenburg in Sweden, and Paris & Evry in France.
The Group has signed more than 100 industry agreements with pharmaceutical, agrochemical, and biotechnology companies. Our clients and partners include University College London (UCL), the National Institutes of Health (NIH), Novo Nordisk, the Center for iPS Cell Research and Application (CiRA) of Kyoto University, AFM, Novartis, BASF, Bayer, and Limagrain. Since 2007, Cellectis has been listed on the NYSE Euronext Alternext market (ALCLS.PA) in Paris.
For more information, visit our website: www.cellectis.com.

About the UCL department of hematology
The department of hematology forms part of the UCL Cancer Institute and is the major tertiary referral center in the UK for all types of hematological malignancies. They have assumed a global leadership position in stem cell transplantation and autologous adoptive cell therapy for leukemia patients.
Dr. Martin Pule as well as being a senior Lecturer in UCL, holds an honorary consultant post as a clinical hematologist in the UCL Hospital (UCLH) and runs a laboratory research program in chimeric antigen receptor design and adoptive immunotherapy.
For more information, visit the UCL Cancer Institute website: www.ucl.ac.uk/cancer  

SOURCE: Cellectis