Launched in 2006 after its co-founder pitched the concept at a Federal Express business plan competition, Ablitech Inc., started the New Year in a new location. The small biotech relocated from Hattiesburg, Miss., to the BioPark at the University of Maryland, where it is working to develop treatments for heterotropic ossification (HO) – a painful bone growth that occurs after severe trauma such as a concussion blast or amputation.

Ablitech's technology, Versadel, provides a nontoxic, biocompatible method to deliver antisense DNA and gene silencing (siRNA), both systemically and locally, to selectively turn off unwanted cell growth. Preclinical studies suggested the technology effectively halts the growth of affected cells and demonstrates the ability to turn off selected protein production in cancer cells.

The move to Maryland comes on the heels of a $2 million Department of Defense (DOD) grant to study HO. With more than $700,000 from private investors also in hand, Ablitech plans this year to investigate its delivery system in an initial animal model, according to Ken Malone, the company's CEO and chief financial officer.

In 2005, company co-founder and chief technical officer Nick Hammond – then a graduate student in polymer science at the University of Mississippi – and co-founder Lisa Kemp were among two dozen participants in a National Science Foundation-sponsored entrepreneurship program designed to marshal the scientific prowess in the U-Miss medicinal chemistry department and the University of Southern Mississippi's School of Polymers and High Performance Materials. Serendipitously, Malone managed the program. The co-founders' pitch in the business plan competition earned seed funding for Ablitech, which Hammond and Kemp then used to secure an NSF Small Business Innovation Research grant.

Their original idea involved a coronary stent, but the researchers quickly discovered they were behind the curve in the race to compete in that market, Malone explained. A year after exploring the initial concept, the company turned toward siRNA and began the development of Versadel.

The technology uses selective chemistry to attach a hydrophilic polymer, such as polyethyleneoxide, to a nucleic acid – antisense DNA or siRNA. The polymer creates a thin shell, protecting the nucleic acid until it is safely delivered inside a cell. Once inside, the chemistry is reversed, releasing the nucleic acid from the polymer, which is safely cleared from the body as the nucleic acid begins to interfere with the production of disease-causing proteins.

The therapeutic platform has the potential to treat a number of diseases and enable the multibillion-dollar RNAi therapeutics market, Malone said.

Versadel differs from other delivery mechanisms in several ways, according to Hammond. It's not a cationic polymer system, which are known to bind proteins but also can be toxic. It's not an encapsulation technology, so there's no risk of leakage, and it doesn't modify the native RNA or DNA sequences.

Rather, Versadel "is a programmed approach that's a nonviral delivery system for gene silencing," Hammond explained. Basically, the company can take any sequence of interest to "knock down" a disease state.

So far, most of the company's research has focused on cancer, "but we can modify our technology in a two-step process that basically gives the native DNA a prodrug effect," Hammond added, by changing the charge composition of the DNA strands to be delivered, resulting in improved stability.

The company also has the ability to place targeting groups at the ends of the prodrug polymers, allowing for increased uptake in cells of interest. In cancer cell culture, Ablitech has demonstrated the ability to release the prodrug moieties placed on DNA and RNA strands through a basic mechanism that doesn't require special enzymes or processes. When the polymer chains are released, they cause a bursting effect inside the cell, in turn releasing the DNA sequence.

Working in cooperation with the Army's Telemedicine and Advanced Technology Research Center, Ablitech will use the DoD grant to study HO – a significant area of interest for the U.S. military, given the number and scope of injuries suffered by soldiers in Iraq and Afghanistan. Up to 70 percent of bone damage following a blast injury results in HO, Hammond said, yet no therapies exist to prevent the condition, which also can occur following hip replacement surgery and amputation.

HO begins as the development of a matrix of soft tissue, typically three to four days following injury to the bone, that can't be detected by X-ray until it begins to calcify weeks later, Hammond explained. At that point, surgery is the only treatment option – in the case of amputation, often resulting in the loss of an additional section of the remaining limb.

"Our initial focus is a preventive measure that could be used locally," he said.

To move Versadel forward, Ablitech – a name that harkens to the company's original coronary stent technology – is seeking to raise $5 million to $10 million in equity financing this year while continuing to pursue grants, said Malone, who has been traveling the country to meet with potential investors. Once the technology has shown efficacy in animals, the company hopes to woo a partner – another biotech, large pharma or university with complementary intellectual property – to target a specific disease using a specific siRNA approach. For the time being, the company plans to remain small and nimble, supported by a backbone of eight employees while outsourcing much of its discovery and development work.

"We're optimistic," Malone said. "We've got a lot of people calling us. But as far as financing goes, it's always difficult to attract investors until you have real animal data."