Alnylam, Tekmira and New Northwest Firm, AlCana, Push Boundaries of RNAi Delivery

Xconomy Boston — 

Alnylam Pharmaceuticals is looking to the frontiers of science for ideas on how to best deliver RNA interference drugs throughout the body, and the Cambridge, MA-based company sounds most jazzed about what it sees emerging from laboratories in Vancouver, BC.

Back in May, I wrote about how Alnylam—one of the richest companies in biotech—was leaning on partners at Vancouver, BC-based Tekmira to deliver an RNAi drug that can circulate through the body. Last week, Alnylam said it is doubling down with its Canadian friends. The company has picked a second drug candidate that uses the same technology, and is sponsoring research at a new company made up of four former Tekmira employees who are pushing further on the leading edge of drug delivery, at Vancouver-based AlCana Technologies.

These are important developments to watch, because while RNAi has been one of biotech’s hot concepts for years, scientists still have plenty of work left to prove these drugs can be efficiently delivered. RNAi treatments are thought to be superior to conventional pills because they specifically hit targets on cells that other drugs can’t, and can silence malfunctioning proteins that cause disease. It sounds great, but when small interfering RNA drugs are injected into the body on their own, they get chewed up by enzymes, or flushed through the kidneys long before they ever get to the desired target on diseased cells. Alnylam has worked around this with locally-delivered drugs that don’t have to circulate through the body, but only a limited number of diseases can be treated that way (and that approach poses challenges too, as I described earlier this week.)

Alnylam, and Swiss pharma giant Roche, see big potential to deliver RNAi drugs through the body using Tekmira’s approach, with lipid nanoparticle capsules (sort of like little grease balls, as Alnylam CEO John Maraganore told me last December) designed to protect the drug in the body until it gets to the diseased cells. This is the approach that Alnylam has implemented with ALN-VSP, an experimental drug for liver cancer that is in clinical trials, and it’s the same technology it plans to use for a second candidate called ALN-TTR, for a rare genetic disease called amyloidosis.

“It’s great technology. It works well, you can manufacture it,” says Maraganore. “It’s ready for prime time.”

But this being the leading edge of biology, what’s prime time today can turn obsolete really quickly. I wanted to know from Maraganore what advantages he sees … Next Page »

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