Jiji Press English News Service 07-08-2005
By Katsuyuki Nakajima

Tokyo, July 8 (Jiji Press) -- In the world of biotechnology, short fragments of ribonucleic acid, or RNA, have quickly become an important tool for penetrating the code of life. Acknowledging the big therapeutic and business potential of small RNAs with only 20-30 base pairs, scientists and biotech firms across the world are now racing to develop practical ways to apply them to genetic research, disease treatment and drug development.

"Following the completion of human genome sequencing, small RNAs have emerged as the key to unleashing the secrets of genes," said Sizuyo Suto, president of iGene Therapeutics Inc. The company, a biotech startup based in Tsukuba, Ibaraki Pref., northeast of Tokyo, was set up in March 2003 by two leading Japanese and U.S. professors in RNA research - Kazunari Taira of the University of Tokyo and John Rossi of the Beckman Research Institute of the City of Hope in California.

It was also the first venture firm derived from Japan's National Institute of Advanced Industrial Science and Technology, better known as AIST, at which Taira also serves as head of the Gene Function Research Center. Like its rivals, iGene is focusing on a biological mechanism called RNA interference, or RNAi, in which a double-stranded small RNA, a chain of nucleotides like DNA, with a particular base sequence prevents a specific gene from functioning or alters the gene's function level.

For decades, RNAs were dismissed as messenger molecules that merely help convert DNA's gene information into proteins. But the discovery of the gene-silencing mechanism in human and other mammalian cells, which received the "Breakthrough of the Year" award of the renowned Science magazine of the United States in 2002, changed the longstanding notion and reinforced the view that RNAs have their own world that is deeply involved in life's evolution. "Through collaboration between the governmental institute and universities, iGene was created to develop a new type of medicine by utilizing RNAi, which should be seen as the 'wonder boy' of the postgenome era," said Suto. As a venture firm authorized by the University of Tokyo and Shujitsu University, where Suto serves as pharmaceutical science professor, in addition to AIST, iGene chiefly capitalizes on the Taira team's discoveries and technologies, for which the company has priority use. It currently designs, makes and sells under tie-ups with Takara Bio Inc. various expression vectors--vehicles that carry genetic information like base sequences of desired small RNAs--to induce RNAi in cells. The firm also synthesizes and sells "perfect double-stranded" small interfering RNAs, or siRNAs, a type of gene-silencing RNAs, through Hokkaido System Science Co. In addition, it is working to establish a "library" of ready-to-use siRNA expression vectors in cooperation with AIST and the government-affiliated New Energy and Industrial Technology Development Organization. Suto said iGene products are currently used by researchers at some 200 laboratories and companies in Japan, who find siRNAs indispensable for carrying out genetic research projects that require certain genes, such as those suspected of causing cancer, be "knocked down" to see what will happen, or not happen. "Since its business launch, iGene has stayed profitable," he added.

But the company has no intention of remaining a mere supplier of biomaterials. "Our goal is to develop RNA drugs," Suto stressed. "The vector and library businesses are income sources, and using the money iGene aims to develop new types of drugs for intractable illness," he said. "AIDS, cancer, hepatitis C and autoimmune disorders are prime targets, while preparatory work is under way for the development of a hepatitis-related drug."

As for potential partners, Suto thinks Japanese companies are desirable because his company uses AIST-owned properties. "But as a business, iGene cannot entirely rule out foreign firms," he said, indicating the possibility of conducting clinical trials abroad for smooth approval to make its drugs widely available. Currently, the venture firm has no specific plan to go public. It will consider stock listing once the company obtains substances that have strong drug potential, he noted. Though constantly generating profits, iGene is still a small company that rents a room in an AIST building in Tsukuba for its head office. Suto, who spent nearly 30 years undertaking genetic research projects at the Nomura Research Institute and Itoham Foods Inc. , accepted in 2003 Taira's request to head iGene shortly after starting the same year his current position at Shujitsu University, where he had been invited to establish a new faculty of pharmaceutical sciences.

Taira was attracted by Suto's experience in the business world, as well as his expertise in genetic research. Suto said he agreed to become the president not because he wanted to make money but because he judged that through iGene he could have access to up-to-date information on RNAi and the development of related drugs. "For a pharmacy department professor, it was not a bad deal," Suto noted, showing few worries about receiving no income from iGene. "With my living financed by Shujitsu University, I am a volunteer here to make a contribution to society," the soft-spoken professor said with a smile. "RNAi technologies will certainly enable us to tackle incurable diseases."