BioWorld International Correspondent

MUNICH, Germany - Atugen AG is adding drug development to its research program in target discovery and its business in target validation.

The change signals both growing maturity in the technology of small interfering RNA (siRNA) and ongoing pressure in the German biotech sector, in which drug development and a product pipeline are essential for access to capital in private or public markets.

The new strategy "is all based on new developments in siRNA, where Atugen has made a breakthrough with novel structures, with molecules that are nuclease resistant," Klaus Giese, Atugen's chief scientific officer, told BioWorld International. "We're finding that they are stable in serum, that they have a long half-life, and we know that we haven't lost any activity."

SiRNA inhibits the workings of messenger RNA, effectively silencing a targeted gene or set of genes. It can thus block the expression of proteins that are key to disease processes in cells. Atugen said the potency of siRNA in inhibiting target mRNA and, indirectly, protein expression has been demonstrated to be 10- to 100-fold better than antisense oligonucleotides. Atugen said that the increased potency of siRNA molecules, with the company's progress in stabilizing siRNA molecules against degradation by nucleases, offers good reasons to pursue drug discovery programs for both systemic and topical indications.

"This is important," Giese said, "because if you go for a systemic indication, you have to use blood flow for delivery, as was shown 15 years ago with antisense. What we have done is to use our knowledge to modify siRNA molecules to keep their catalytic activity but still remain stable in serum. We have made several modifications that allow this, and we believe we have an advantage over competitor molecules. Ours are stable for at least 24 to 48 hours without losing activity."

In addition to the progress in molecular design, Giese said, "We also have very interesting and potent targets that we have identified in the last couple of years." He added that the company had patented novel oncology targets.

Acknowledging that there is a race to realize the potential in siRNA technology, Thomas Christely, Atugen's chief operating officer, said: "We hope that our advantage is the functional stabilization of these molecules. We are looking for a big partner to move this program as quickly as possible in as many indications as possible."

"In principle," said Andre Lochter, the company's director of business development, "we have all the capabilities and models. We can do all that is necessary. Working with a partner is more a question of scale."

"Our main goals within the next 12 months," Christely said, "are to generate more good in vivo data and to convince another large pharma partner to enter a research alliance."

The company is doing research in oncology and metabolic diseases and is preparing to sign topical partnerships with academic institutions. Giese declined to specify which institutions.

Atugen was founded in 1998 and has raised €26 million in three rounds of private investment. The company said that its current services business is profitable and that its positive cash flow covers most of the firm's internal research programs.