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New Study Raises Doubts About Some RNA-Based Drugs

One of the hot new fields in biotechnology, RNA interface, has previously been lauded as a way to turn off specific disease causing genes. Now, however, a new study raises doubts about what makes the drugs effective as many big pharmaceutical companies and up and coming biotech startups jockey to develop new drugs that harness this technology.

Dr. Jayakrishna Ambati, senior author of the study and professor of opthamology at the University of Kentucky, thinks that some of these drugs don't work in the way that they're supposed to. In fact, instead of turning off genes, she suggests that they may instead work by activating a patient's immune system.

"It seems to be working by a completely different mechanism that's unreleated to the underlying premise," she said.

Two particular drugs were examined in the study, one by Allergan and the other made by Opko Health. Both drugs are prescribed to treat macular degeneration, one of the leading causes of blindness in elderly people. Each company had said that their drugs work through the intended mechanism and are safe.

Investors, it seems, weren't very reassured by the statements released by the companies. Once the study was published online in the march 26th issue of Nature, shares of Opko Health fell almost ten percent. Allergan, a more diversified company that sells products like breast implants and Botox, wasn't hit as hard by the study.

Other companies are banking that the study's results won't apply to all of the other RNA drugs being researched and marketed. "It's sort of like saying all birds don't fly when you are studying a penguin," said the chief executive of Alnylam, John Maraganore. Alnylam is currently conducting clinical trials of an RNA drug to see its effects on a respiratory virus.

 

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