Monday, March 3, 2008

Former Sirna talent leads Dicerna to next RNAi stage

By Ryan McBride

A biotech startup in the sizzling field of RNA-interference says its technology has opened a "second doorway" that offers a different method of silencing disease-causing genes from competitors such as Alnylam Pharmaceuticals Inc.

Dicerna Pharmaceuticals Inc., based in Cambridge, has attracted a team of West Coast investors and executives experienced in the area of RNA-interference, or RNAi, due to its novel technology and plans to begin injecting animals with its experimental RNAi treatment this week, officials said.

After keeping much of its business strategy under wraps, Dicerna has revealed how it plans to open a gateway into the RNAi arena that doesn't rely on patents that other local companies use, said Dicerna CEO James Jenson.

Billions of dollars are being committed by big pharmaceutical companies to the field of RNAi, which some say could be the source for a new class of drugs able to block, or silence, genes that make proteins linked to certain diseases. RNAi biotech Sirna Therapeutics Inc. of San Francisco set the highwater mark in 2006 when it was acquired for $1.1 billion by New Jersey drug giant Merck & Co. Inc.

Locally, life sciences giants such as Swiss firms Roche Holding AG and Novartis AG have forged major licensing deals with Cambridge-based Alnylam, but there have been few avenues to break into this hot field without licensing technology controlled by either Alnylam or Merck's Sirna. But Dicerna thinks it has a lock on just such a new scientific portal.

To those without deep knowledge of RNAi, it's tricky to explain the nuances between the RNAi technology of Dicerna and that of its competitors. Simply put, Dicerna uses longer molecules of RNA than both Alnylam and Sirna to form the materials that ultimately silence genes. Jenson also argues that Dicerna's process of RNAi more closely resembles the natural process of gene silencing in human cells, increasing the potency of its drugs.

Industry watchers believe there is room for new approaches to RNAi. "As more companies enter the RNAi space, the patent landscape will become even more crowded, making it more difficult to enter the market," said David Dykeman, a life sciences patent attorney at the Boston office of law firm Greenberg Traurig LLP.

Attracting West Coast talent

Dicerna's technology was invented by John Rossi, a researcher at City of Hope biomedical center in Duarte, Calif., and Mark Behlke, a vice president at Integrated DNA Technologies Inc., a maker of synthetic genetic materials in Coralville, Iowa. Jenson, a biotech veteran, said he learned of the technology in 2006 and showed it to Doug Fambrough, a general partner at Boston venture firm Oxford Bioscience Partners.

Fambrough, who had led Oxford's successful investment in Sirna, got his firm to house Dicerna and Jenson (then the only employee of the startup), in its offices beginning in July 2007. Oxford led Dicerna's $13 million Series A round of venture capital, first reported by Mass High Tech in November 2007, which included funding from Skyline Ventures of Palo Alto, Calif. Dicerna board member Stephen Hoffman, of Skyline, had also managed an investment in Sirna.

Dicerna's Sirna connections grew stronger last summer when Roberto Guerciolini, a physician by training and former chief medical officer of Sirna, joined the RNAi startup as senior vice president of pharmaceutical development.

Dicerna executives declined to reveal their diseases of interest, but Jenson did say the company would focus on developing RNAi treatments for cancer, viruses and metabolic diseases. The biotech aims to develop two or three drugs internally while licensing its technology to drug companies for other uses.

Because Dicerna takes a different approach, Jenson said, the company and its partners can target nearly any gene without requiring licenses from Alnylam and other institutions. Dicerna has already begun to meet with potential licensees, and Jenson said he and other company officials plan to travel to Japan in April to meet with large pharmaceutical firms based there.

Beth Arnold, a partner in the life sciences practice of Boston law firm Foley Hoag LLP, said it's important that biotechs have intellectual property that enables them freedom to develop drugs without requiring licenses from one or more other companies. But it's sometimes difficult for biotechs to obtain broad IP protections, or blocking patents, from the U.S. Patent and Trademark Office, she added.

Jenson said that Dicerna has already invested heavily in ensuring its IP is well protected. "We are very comfortable with this and believe we have a strong position," he said.