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By Malorye A. Branca

June 17, 2004 | RNAi development continues its rapid pace as companies vie to be first with a drug based on the new gene-silencing technique. Three leading contenders in this race -- Alnylam, Atugen, and Benitec -- each teach took major steps this quarter.

Biotech IPOs have been a mixed bag so far, causing some companies to rethink their plans. However, Alnylam headed boldly into the erratic public market, launching its IPO during the third week of May.

The highest-profile of all the RNAi therapeutic developers, two-year old Alnylam still needs more than the $50 million it already raised from venture capitalists. The company aims to sell 5 million shares at between $10-$12. Alnylam is developing both RNAi-based drugs for localized diseases, such as eye conditions, as well as systemic treatments. 

Getting RNAi to work systemically in humans is a major hurdle. Atugen reported last month it had successfully treated rodents with a putative drug for diabetes. “We have determined [cellular] uptake and we established the phenotype,” says Klaus Giese, Atugen’s chief scientific officer. In other words, it works.

The study animals were genetically engineered to react inappropriately to glucose, leaving too much sugar in their blood. According to the company, animals treated with the Atugen compound showed lower peak glucose levels after a dose of sugar, and these levels returned to normal more quickly than those of animals treated with “control,” inactive, siRNA molecules.

Just a week after Atugen’s announcement, Benitec acquired competitor Avocel, gaining an anti-hepatitis C program, intellectual property, and a site for the Australian company’s U.S. subsidiary, Benitec, Inc. 

Becoming part of Benitec “is a synergistic move to really get in vivo use of RNAi into clinical application,” says Stanford University professor Mark Kay, founder of Avocel. “Now, we don’t need any additional technology, it’s just a matter of fine tuning and advancing.” Kay joins Benitec’s scientific advisory board and will consult with the company on its hepatitis C and other anti-viral programs. 

According to John McKinley, Benitec’s CEO, the company plans to launch a hepatitis C clinical trial in 2005.


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