BioWorld International Correspondent

PARIS - Cellectis SA completed a second financing round in which it raised €16 million (US$15.7 million), bringing to €20 million the total capital the company has raised since its inception in December 1999.

The latest financing was subscribed mostly by four new investors: BankInvest BioMedical Venture, of Copenhagen, Denmark, and three Paris-based venture capital funds: AGF Private Equity, LCF Rothschild Asset Management and Odyssée Venture. Representatives of both BankInvest BioMedical Venture and AGF Private Equity joined the Cellectis board of directors.

Paris-based Cellectis, which describes itself as a "rational genome engineering" company, was founded by nine researchers from the Institut Pasteur, a not-for-profit medical research establishment. The company is housed in the Institut Pasteur's biotechnology incubator, and CEO André Choulika told BioWorld International that it now has to leave and move into premises of its own. With a work force of 35 that is still growing, he said, the company needs a substantial amount of space. It is looking at a number of sites in Paris and hopes to be in a position to move by the end of the year, depending on the time taken to set up and equip the laboratories it will need.

The main priority for Cellectis is to install automated production facilities for manufacturing large quantities of custom-made Meganucleases. It says it is the first company to apply the Meganuclease Recombination Systems approach to in vivo genome engineering, and Choulika stressed the difference between its approach and that of "all other biotechnology companies, which do reverse genetics."

Cellectis' proprietary technology platform is covered by nine families of patents to which it has full rights under license from the Institut Pasteur. The technology makes it possible to engineer Meganucleases as homing endonucleases so they can recognize specific DNA sequences. Cellectis thus creates novel Meganucleases that can target a unique DNA break in vivo and induce a site-directed recombination.

The company is using the gene targeting technology to develop drugs for treating single-gene diseases, such as cystic fibrosis, Duchenne's disease and familial hypercholesterolemia, as well as viral infections.

It also licenses out its technology to both biopharmaceutical companies and academic laboratories, Choulika said. One technology licensing agreement was signed in September with the German company Zentaris AG, which acquired exclusive worldwide rights to Cellectis' sLTR (single long terminal repeats) gene transfer technology for application in therapies for cancer. Those sLTR vectors make it possible to eliminate tumor cells without affecting healthy tissues, since they enable a gene to be introduced into cellular DNA, after which both the vector and the transferred gene self-destruct.

Choulika said Cellectis' current funding would be sufficient to finance its activities until the end of 2004, when it plans to have achieved the break-even point. But he added that it might move into profit before then "if our revenues continue to grow as fast as they are at the moment." He declined to put a figure on those revenues, however.