Two biotechnology companies are joining forces in commercial pursuit of an emerging scientific field that could lead to treatments for numerous diseases.
The companies, Alnylam Pharmaceuticals and Isis Pharmaceuticals, expect to announce today an equally owned joint venture to develop drugs that work by blocking so-called micro-RNAs.
Micro-RNAs, virtually unheard of a few years ago, are tiny snippets of RNA — the chemical cousin of the genetic material DNA — that have been found to play a major role in controlling biological processes. Scientists have identified about 500 different micro-RNAs that are made by human cells, and these snippets in turn appear to influence the activity of thousands of genes.
Studies have already linked micro-RNAs to cancer, viral infections, immune disorders and other diseases. So blocking specific micro-RNAs — or perhaps stimulating them — could theoretically provide a powerful way to treat diseases.
“It’s as if we’ve discovered a new kingdom of regulation in biological metabolism that we didn’t know was there,” said David Baltimore, a Nobel laureate and the president emeritus of the California Institute of Technology, who will be a director of the new company as well as chairman of its scientific advisory board.
The new company, called Regulus Therapeutics, will be equally owned by Isis and Alnylam but will have its own management and board. The management team has not been identified yet.
Both Alnylam and Isis are pioneers in developing drugs that are made of RNA and seek to suppress particular genes. The companies hope that combining their skills — and their patents — will allow them to dominate the micro-RNA therapeutics business.
“This is the first time I’ve seen a biological advance as broad as this where the intellectual property is as concentrated in a single company as with Regulus,” said Stanley Crooke, chief executive of Isis.
Other companies are also starting to focus on micro-RNA. One is Rosetta Genomics, an Israeli company that is already collaborating with Isis.
So far, however, RNA-related drugs have been difficult to develop. Isis, based in Carlsbad, Calif., has had many setbacks developing drugs using a technology called antisense. But its stock has nearly doubled in the last year on hopes for a drug that has sharply lowered cholesterol in clinical trials.
Alnylam, based in Cambridge, Mass., is trying to develop drugs that turn off specific genes by harnessing a natural phenomenon called RNA interference. Two of the discoverers of the phenomenon were awarded the Nobel Prize last year.
Micro-RNAs are related to RNA interference. But John Maraganore, chief executive of Alnylam, said that while his company aims to shut off specific genes, Regulus might be able to inactivate entire networks of genes. The broader effect, he conceded, could raise the risk of side effects.
To block a micro-RNA, Regulus will probably use the antisense approach — synthesizing short strands of an RNA-like molecule that would bind to the micro-RNA and inactivate it.
Both companies are contributing intellectual property rights to Regulus. Alnylam is also contributing $10 million, since it has fewer patents to offer.
After this agreement, the companies will fund the venture equally. Mr. Crooke said that Regulus, which is likely to be based in the San Diego area, is expected to have about 20 people employed next year and to spend up to $20 million over its first two years. Regulus has no drugs in clinical trials yet.
Victor Ambros, a professor at Dartmouth who in the early 1990s discovered the first micro-RNA, in the roundworm, said it was exciting to see companies being formed. “There’s a lot of excitement, and I think justifiably so, for the prospect of targeting the micro-RNAs,” said Professor Ambros, who has no ties to the companies.
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