OVERVIEW

From discovery to clinical applications

NellOne Therapeutics, Inc. (NellOne) is a regenerative medicine company that has been spun out of the U.S. Department of Energy's Oak Ridge National Laboratory in Oak Ridge, Tenn.

Senior Staff Scientist Dr. Cymbeline Culiat in the Lab's Biosciences Division / Systems Genetics Group had identified the activity of the Nell1 gene's pathway in the formation of key mammalian tissues and organs, and Battelle Ventures founded NellOne to translate her basic science discovery into clinical applications.

The company is dedicated to developing and commercializing Nell1 protein therapeutics for restoring tissue mass and function in patients recovering from heart attacks.

The NellOne difference

Therapeutics being researched by NellOne have the potential to restore both tissue mass and function. This is the NellOne difference, as current approaches, such as those based on stem cells, have shown potential to restore tissue mass, but with only limited recovery of function.

It is not just cell regrowth, but also the way cells are organized to support organ function that is important. And Dr. Culiat's research strongly indicates that the Nell1 protein triggers maturation of certain cell types to enable new tissue to perform its normal functions.

The promise of restoring both normal tissue mass and function comes from leveraging a critical natural cell growth and maturation pathway. By directly influencing cell-signaling mechanisms in injured tissues of specific organ systems, natural cell regrowth can be initiated and maturation of tissue function can be stimulated.

Benefit highlights

According to the U.S. Department of Health and Human Services Centers for Disease Control and Prevention, in 2008 about every 26 seconds an American will have a coronary event; some 770,000 Americans will have a new heart attack; and about 430,000 Americans will have a recurrent attack.

Currently, there are no highly effective, permanent treatments for the restoration of heart muscle cells and function due to damage from heart attack.

If NellOne is successful in producing therapeutics that can restore muscle mass and significantly improve heart function – perhaps without the need for daily drug treatment – it would be a breakthrough.

Cost-effective, focused approach to development

NellOne operates 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.

While NellOne is being “spun out” of ORNL, it operates virtually. Its legal address is investor Innovation Valley Partners' office in Knoxville, Tenn., but the company has no employees and Battelle Ventures General Partner Tracy Warren is acting CEO.

The development path is focused on research and development of intellectual property.

'Proof of efficacy' research programs

Research is focusing on applications for rebuilding cardiac muscle and is being conducted at ORNL under the leadership of Dr. Culiat, who authored the various related patent applications filed.