4s3, With $20M in Tow, Pursues Route for Delivering Drugs to Muscle

3/8/12Follow @arleneweintraub

If you ask 4s3 Bioscience CEO Timothy Harris what the name of his Medford, MA startup signifies, you won’t get much of an enthusiastic response. It’s not a play on words or reference to the company’s scientific platform, but rather “it’s just the name we ended up with,” Harris admits.

But ask him about the company’s underlying drug delivery technology for treating muscle disorders, and his enthusiasm level rises dramatically. “There are a lot of drug-targeting companies, but none that are taking our approach,” he says. “We believe this is a platform technology that can be applied to a number of different muscle diseases.”

4s3′s development plan got a big boost on March 5, when the company announced it had closed a $20 million Series A financing with KLP Enterprises. KLP is a trust formed by Karen Pritzker—a member of the famed Pritzker family, creators of the Hyatt hotel chain—and her husband, investor Michael Vlock. 4s3 will work with KLP subsidiary Alopexx Enterprises, which was formed in 2011 to offer startups assistance in drug development, manufacturing, and regulatory affairs.

Harris co-founded 4s3 in 2007 with Dustin Armstrong, who serves as vice president of research, and built it on an antibody-delivery technology they licensed from the University of California in Los Angeles. The technology consists of therapeutic molecules tagged to antibody fragments. Those fragments home in on muscle cells, burrow inside them, and deliver proteins and other therapies exactly where they’re needed, Harris says.

4s3′s lead programs target myotubular myopathy and myotonic dystrophy, both of which are rare, genetic diseases that result in abnormal coding for proteins that are essential for muscular function. “Because of the nature of those diseases, they’re a very good fit for what our technology can do in terms of protein replacement,” Harris says.

The company previously raised $2 million in a seed round from Genzyme Ventures and others, Harris says. It has generated strong pre-clinical data and is now developing a timeline for human clinical trials. Because 4s3 is targeting orphan diseases for which there are no therapies on the market, Harris says, he’s hopeful the FDA will accelerate the regulatory process for the company’s lead drugs.

Like most startups in biotech these days, 4s3 will have to think about whether it needs to take on a deep-pocketed pharma or biotech partner. “We’ll have to see how the technology develops,” Harris says. “Myotubular myopathy, for example, affects very few patients, so it’s one disease area where small companies can think about moving forward on their own.” The new funding, he says, will help to speed up and enhance that program. “This funding was essential for moving the company to the next stage.”

But if the company ultimately decides to use its platform to develop remedies for more common illnesses, it may need to take on a partner. “When you look at the breadth of what we think we can do with this platform technology,” Harris says, “it’s hard to imagine any single company being able to do everything.”

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