with a $20 million-plus round of venture capital like some of its predecessors, such as Allozyne, VLST, Theraclone Sciences? The big reason, Weissman says, is that Mirina doesn’t really need that much capital to invest in business infrastructure. It can stay lean—with just five employees at the moment—and still hit value-driving scientific goals as long as it relies on collaborations and business support from Accelerator, Weissman says.
Getting a small expansion round of financing isn’t going to keep Mirina going forever. Weissman noted, however, that this is a similar interim step that Allozyne and Theraclone Sciences followed before they eventually won bigger venture rounds.
Mirina is spearheaded by David McElligott, the vice president of R&D and a scientific veteran of Bothell, WA-based Icos. His team will be charged in the coming months with further developing the company’s microRNA technology platform, demonstrating its drug candidates have desirable properties in mice and other live animal tests, and further studying the unexpected new properties that the company is keeping secret for the time being. Mirina hasn’t published anything in the peer-reviewed literature, or talked at a scientific conference, and it won’t until it at least has its patent applications filed in the U.S., McElligott says.
As with any new mode of treatment, there is huge risk when venturing into the unknown. It’s entirely possible that shutting down entire networks of proteins could throw a wrench into essential biological functions, and cause dangerous side effects. While the heat is certainly on for Mirina and its peers to gather evidence from animals—and people—to prove the microRNA concept, the safety of these drugs will have to monitored with great care. That’s balanced out, McElligott says, by the potential of a new paradigm for addressing some of the most common diseases that have traditionally been very tough to treat, things like neurodegenerative diseases, and cancer.
Traditional research has often focused on making small-molecule chemicals that are increasingly specific to a given target, to avoid hitting similar protein structures, which can lead to “off-target” toxicity. MicroRNA research, in a sense, seeks to turn that notion on its head, arguing that many drugs are such rifle-shots at a specific target that they aren’t powerful enough to silence the chaos created by diseases that arise from an entire symphony of things going wrong inside cells.
“Hitting a target with one drug isn’t always beneficial, and making single molecules increasingly selective isn’t always for the best,” McElligott says. “Sometimes you need to hit multiple components to get the best outcome.”
