By Tom Ballard, Director of Innovation and Entrepreneurial Initiatives, Pershing Yoakley & Associates, P.C.
If you were looking for a regional model for a researcher turned entrepreneur, one name sure to be at the top of the list would be Cymbeline Culiat, more widely known as Bem.
The always smiling, effervescent Philippines native was a Senior Scientist in Molecular Genetics at Oak Ridge National Laboratory (ORNL) for about a decade. During those years, she discovered the role of the NELL1 signaling protein in pathways controlling the growth and maturation of the musculoskeletal and cardiovascular systems.
Culiat’s work attracted the attention of Battelle Ventures (BV), a fund established to accelerate commercialization of technologies, including those emanating from national laboratories like ORNL that are managed or co-managed by Battelle. BV and its local affiliate – Innovation Valley Partners – launched NellOne Therapeutics Inc. from ORNL in 2008, based on Culiat’s research.
Fast forward to today, and the two individuals most heavily involved from the outset – Culiat and BV’s Tracy Warren – are the co-founders and key executives in the Oak Ridge start-up. Culiat serves as Chief Scientific Officer and Chairman of the Scientific Advisory Board. Warren is the company’s Chair and Chief Executive Officer.
During my service at ORNL, I became very familiar with the company and its co-founders. Since launching teknovation.biz 20 months ago, I have been persistent in urging Culiat to sit for an interview. She finally did recently as she relayed a story typical of many start-ups – pivoting along the way.
“This is really a homegrown Tennessee story,” she proudly proclaims, noting that the basic research started in Oak Ridge, commercialization and technology transfer was successfully achieved out of a Tennessee-based federal lab, the company continues to be located there, and the start-up recently raised capital from local angel investors.
At the time of its founding, the company was focused on how the NELL1 protein could be used to regenerate human cardiac tissues damaged by heart attacks and healing of other acute soft tissue injuries such as severe muscle wounds.
“We licensed the heart and skeletal muscle regeneration patents from UT-Battelle exclusive worldwide,” Culiat says of the company’s agreements with ORNL. With NellOne playing a major role in the patent prosecution, these patents have just issued in the U.S., Europe (Germany, France and United Kingdom), and Israel, and are pending in several other countries.
In 2010, Culiat joined the start-up full-time as Chief Scientific Officer. Like any life science company, NellOne was faced with the typical high costs associated with bringing a regulated human clinical product to market, a persistent challenging economic climate and considerable uncertain regulatory and reimbursement hurdles.
The company executed an interim pivot in 2013 from its original path “after a lot of thinking” by Culiat and Warren. Prevailing after its first critical test in the past year, Culiat laughingly told us, “I walked through my first start-up ‘Valley of Death’ and survived.”
The change – shifting from human to veterinary applications – will allow the company to generate revenues faster and build a stronger foundation to pursue its long-term goal – products for humans.
“It can cost about $250 million to get a heart therapeutic/biologic from discovery through first human clinical trials,” Culiat noted. “By focusing on a veterinary application, the cost and regulatory requirements to get the first NELL1 product to market are much less.”
NellOne’s goal is to have a non-invasive, U.S. Department of Agriculture-approved treatment for treatment of severe wounds in high-value horses in the marketplace within three years.
“Tissue regeneration in severe horse injuries is estimated to be at least a $350 million a year U.S. market,” Culiat said at a recent Launch Tennessee event, adding that horses used for equestrian events and racing can cost as much as $250,000 to several million dollars. Regenerative medicine approaches are envisioned to completely heal potentially career-ending injuries to these horses.
A key research partner in this work is the Center for Equine Veterinary Research at the University of Tennessee, Knoxville’s College of Veterinary Medicine, a relationship that deepens the company’s roots in the state even more.
Although veterinary grade protein has different criteria from human clinical grade product, Culiat maintains that NellOne will benefit from the knowledge gained in large-scale manufacturing and development of the veterinary product. After all, she noted that developing human applications requires that animal trials be completed with satisfactory results before human trials can be undertaken.
“We can get to market, have our technology validated in the large animal market, and not preclude entry into the human market in the future,” Culiat said.
She explained that the shift also makes sense, considering the company’s location in the South, which has a sizable horse industry in Tennessee as well as neighboring states such as Alabama, Kentucky, North and South Carolina, and even Florida.
Europe is also a target market. Culiat says it has a pretty significant horse industry valued at 100 billion Euros of economic impact and growing five percent annually. Although it has a smaller horse population (6 million) compared to the US (10 million horses; about $120 billion industry), their horses have very high market value and represent important breeds that are exported to different countries.
Describing her last five years as an “unpredictable rollercoaster,” she said you need to “set the expectations right” and “be steady and don’t ride it (the roller coaster) emotionally.”
We’re betting that her determination will prevail in the end.