Medical researchers' altruism collides with a for-profit feud

Just over a year ago, Cambridge's prestigious Broad Institute started an idealistic medical-research project, fueled by millions of dollars from drug companies, to create powerful new molecules and make them cheaply available to lab researchers around the world.

Called the RNAi Consortium , the program runs on donations from Novartis AG , Bristol-Myers Squibb Co. , and Eli Lilly & Co. , among others. It has designed a huge collection of molecules to block the workings of each human gene -- a new and increasingly important technique for scientists and drug makers. The project embodies the ambitious goals of the three-year-old Broad Institute, which united the czars of top science labs at Harvard University and the Massachusetts Institute of Technology to turn genetic research into real treatments for diseases.

But now the altruistic RNAi project has run into the shoals of commerce. The Broad relies on two for-profit companies to produce and distribute the new molecules to researchers, and one of those companies is suing the other to stop it from sending them out.

Sigma-Aldrich Corp. , a global lab supply company based in St. Louis, filed suit against Open Biosystems Inc. of Alabama, a private firm specializing in supplying genetic material, charging that it infringes two key scientific patents.

Although the Broad Institute invents the RNAi molecules, it can't produce them in the volume needed for research experiments. So it has licensed the two suppliers to keep a ready stock of Broad-invented material in their warehouse freezers to sell to customers. The companies make a profit, but because the Broad Institute absorbs the high cost of the original research, they can keep prices down for their customers.

If the lawsuit succeeds in shutting down Open Biosystems, it would give Sigma an effective monopoly, leading scientists to worry that a resource built with philanthropic money and intended for public access would become unaffordable.

``Our goal is easy access to the world research community," said David Root , the Broad Institute scientist who manages the RNAi Consortium. ``We went to two distributors with the idea of trying to make sure it's widely available."

Open Biosystems denies it violates any patents, and has continued to ship the Broad Institute molecules.

The suit illustrates an emerging challenge for supporters of publicly accessible medical research: Patents and intellectual property in the life sciences have grown so valuable that they are jealously guarded as potential keys to millions of dollars in revenue.

``You have this situation where you're doing research and you don't really know who owns what you're doing anymore," said John Quackenbush , a genetic-data researcher at Dana-Farber Cancer Institute.

It also pits two very different companies against each other. With offices around the world, Sigma-Aldrich is one of the largest suppliers of laboratory research chemicals, with 7,000 employees and $1.7 billion in annual sales. It has a growing business in so-called biotechnology reagents, highly specialized chemicals that let laboratories use the new tools of genomics to test potential drugs. It was also one of the original $3.6 million donors to the Broad Institute's RNAi project.

Open Biosystems, with 55 employees, is a four-year-old private company that sells only human genes and related chemical tools. Its 36-year-old cofounder describes the company as the ``iTunes of the genomics world," with a website that allows users to pick and choose individual genes and other chemicals, which are shipped overnight in dry ice via FedEx. The website, www.openbiosystems.com, trumpets the company's ``open source business philosophy" of ``democratizing access to materials." The company does not release annual sales figures.

The legal fight is being waged over short hairpin RNAi molecules, tiny strands of genetic material that can essentially switch a single gene to the ``off" position and shut down its function in a cell. First identified in 1998, RNAi gave medical researchers a new and powerful tool to study and potentially devise treatments for a wide range of genetically linked health problems -- a discovery so important that RNAi was named the ``breakthrough of the year" by Science Magazine.

As RNAi has developed into a critical tool of medical research, it has also become a legal battleground. Companies hoping to make RNAi-based drugs, including Alnylam Pharmaceuticals Inc. of Cambridge, have been racing to lock up the legal rights to use the molecules as therapy. Sigma-Aldrich has moved to become the most important supplier of RNAi as a research tool, spending more than $10 million to buy and license patents connected to its use.

Two of those patents, licensed last year from the British company Oxford BioMedica PLC , are the basis of the suit. The patents cover a ``lentivirus," a disabled version of the HIV virus that works like a microscopic needle to inject RNAi into cells. Although Open Biosystems does not sell RNAi packaged inside the lentivirus, as Sigma-Aldrich does, the suit claims that Open Biosystems' use and testing of the RNAi includes techniques and gene sequences that violate its patents.

Troy Moore , chief technology officer and cofounder of Open Biosystems, said the company has its own patents and does not violate those named in the lawsuit.

Moore suggested that the suit contradicts the basic idea behind the RNAi Consortium. If RNAi ``only ends up in one group's hands, that's kind of a monopoly situation, and that's not healthy for anyone," he said in an interview.

Keith Jolliff , strategic marketing director for Sigma-Aldrich's biotechnology business, said the company supports the public mission of the Broad Institute but filed the lawsuit to protect its heavy investment in becoming a leading RNAi supplier.

``There's no point in having patents if you're not going to enforce them," he said.

The legal case is still in its early stages. Sigma-Aldrich's complaint, filed in US District Court in the Eastern District of Missouri, asks the judge to block Open Biosystems from using or selling any RNAi that violates its patents. It also requests damage payments for money lost to the violation. Open Biosystems has until July 28 to file a formal response to the suit.

The Broad Institute, meanwhile, is left in the delicate position of having one of its key donors trying to shut down a member of the group whose goals are, on the surface, closely aligned with its own.

The institute has not taken an official position on the case, and plans to meet with Sigma-Aldrich to discuss how it might be resolved, said spokesman Fintan Steele .

``We hope they sort it out quickly," he said, ``and get back to the business of getting this stuff out."

Stephen Heuser can be reached at sheuser@globe.com.