which proteins to make. Sarepta (NASDAQ: [[ticker:SRPT]]) and Prosensa Holding (NASDAQ: [[ticker:RNA]]) are developing drugs that “skip” over a faulty section of a gene and help produce important proteins that a patient would otherwise lack.
Most of these approaches, while tantalizing to the scientific community, haven’t led to approved drugs as of yet. Only Isis has a marketed drug, and others still have a long way to go. Delivering RNAi therapeutics, for instance, has been a major challenge that remains mostly unsolved.
It’s hard enough to develop a drug; it’s even harder to develop one that uses a completely new mechanism. That’s the type of challenge Renaud is facing at RaNA.
“I think here, it’s not really so much a challenge as it is a big opportunity,” Renaud says. “We’re getting very intelligent about how to work with these RNA targets, and this [company] in particular for me presented a very interesting and intriguing opportunity in that its approach was very different from what others were working on in the space. If we can actually get those genes switched on, we may have a very beneficial impact across a number of therapeutic areas.”
Here is the situation Renaud is stepping into. The company has all the marks of an established biotech: well-known investors, full-time management, a growing patent portfolio, and financial breathing room. (Renaud wouldn’t say exactly how much, just that the company has runway “into next year.”) Now it has to prove its idea works beyond cells grown in petri dishes.
Renaud says the focus in 2015 is to take early drug candidates into preclinical studies, but he wouldn’t commit to a timeline to get into clinical trials, saying only “we’re still a bit away.” Those compounds are single-stranded therapeutic oligonucleotides—compounds that can bind to cell molecules such as RNA that are involved in gene expression—that would be delivered via subcutaneous injections.
RaNA has done the most work so far in spinal muscular atrophy (SMA), a rare genetic disease that causes severe muscle atrophy and weakness in newborns and children; and Friedreich’s Ataxia, an often fatal genetic neuromuscular disorder. Other companies like Isis (via a partnership with Biogen Idec) are well ahead of RaNA, but the company believes that SMA and Friedreich’s are good showcases for its approach, because they occur when normal genes are silenced. Switching them on should have a benefit if RaNA’s treatments work as they expect.
Still, it’s unclear just what RaNA will look like a few years down the road, and which therapeutic areas it’ll evolve to focus on. RaNA is set up as a limited liability company, making it easier to sell off individual drug candidates or groups of them to partners who don’t want to buy the entire company. Shaping RaNA’s business strategy is just one of the decisions Renaud is facing.
“I think at this point, as we develop the data, we’ll see what makes the most sense for the company,” he says. “But right now, my vision would be that we hold onto these [programs] for as long as we possibly can, given that it’s our technology, it’s our platform, and we think we can take these for a pretty good distance with the resources we have.”
