By Allan Maurer
KNOXVILLE, TN—Battelle Ventures and its Tennessee affiliate, Innovation Valley Partners, is investing an initial $1.5 million to spin-out promising regenerative medicine startup NellOne Therapeutics Inc. from the U.S. Dept. of Energy’s Oak Ridge National Laboratory (ORNL). The “breakthrough” treatments the firm is developing may one day help restore functioning tissue to damaged hearts.
Battelle says the investment will be tranched based on key technical milestones. Battelle’s General Partner Tracy Warren, acting CEO, says ORNL Senior Staff Scientist Dr. Cymbeline Culiat in the Systems Genetics Group identified a novel gene signaling pathway—activity of the Nell-1 gene—that is critical to tissue growth and maturation in key mammalian organs.
“We created this company to translate Dr. Culiat’s discovery into clinical applications,” said Warren. NellOne is the seventh life sciences company in New Jersey-based Battelle’s portfolio and the first in therapeutics.
“The promise of restoring both normal tissue mass and function comes from leveraging a critical natural cell growth and maturation pathway,” said Dr. Culiat.
“We will be testing approaches to stimulate this cell-signaling mechanism in injured tissues of specific organ systems, to initiate cell regrowth, and subsequent maturation of tissue structures so that normal organ functions are restored.
“It’s not just cell regrowth that is important, but also the way cells are organized to support organ function,” Dr. Culiat said.
One of the problems with stem cell therapies aimed at regeneration is the need to push the cells to maturity, so they have the right shape and coordination and communicate. “Their collective shape is key to their abilitiy to function together as tissue in an organ,” she told TechJournal South.
“Current approaches, such as those based on stem cells, have shown the potential to restore tissue mass, but with only limited recovery of function,” noted Warren.
“Data from small-animal studies show that the Nell-1 protein triggers maturation of certain cell types to enable new tissue to perform its normal functions,” she said.
Dr. Culiat’s lab discovered the genetic pathway while working with a mutant mouse line to study why the line possessed certain traits. Although the potential therapy may apply to several key organs, NellOne is focusing initially on creating and commercializing protein therapeutics that can be delivered to damaged tissue to restore both mass and function in patients recovering from heart attacks.
Warren said that NellOne is currently operating in the “virtual company” model that Battelle Ventures has developed to provide the most productive and capital-efficient environment during the highest-risk phases of a company’s development – in this case, the proof-of-efficacy stage.
“Most of the funding is dedicated to development of intellectual property,” she said, adding that there are no employees and scientific activity remains for now at ORNL. The company’s official address is the Innovation Valley Partners’ office in Knoxville, TN.
Warren says it will take about a year to complete proof of efficacy studies and early pre-clinical work. “Then we will seek a typical Series A round to fund early clinicals,” she says. The company will likely need to make the long trek through U.S. Food and Drug Administration trials that take ten years or longer to move a therapy to commercial viability, Dr. Culiat said.
Dr. Culiat, who authored the various related patent applications filed, will lead ORNL research focusing on applications for rebuilding damaged cardiac muscle.
“While cardiovascular therapeutics is a fairly crowded space, there are medical needs that are not being adequately addressed,” Warren added.
“We created NellOne to explore its unique, breakthrough potential. Treatment of cardiovascular diseases presents a huge and growing market and current treatment options, such as daily drug doses, are poor, at best.
“We believe that the novel features of NellOne’s platform, if proven commercially viable, could propel the company to a leading role in treating vascular disease.”
On the Web: www.battelleventures.com/health_life_sciences.html
www.ornl.gov/ornlhome/about.shtml
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