News Release

Latest Scripps Florida Spin Off Builds on Advanced Non-Coding RNAs Technology

JUPITER, FL, August 27, 2008—Claes Wahlestedt, the Scripps Florida professor who heads the biomedical research institute's efforts to develop drug candidates for diseases of the central nervous system, has a development of his own these days, a brand new company based on an advanced technology licensed from his work at The Scripps Research Institute's Jupiter facilities.

The new company, called cuRNA, was recently founded with Joe Collard, a South Florida business consultant and long time friend of Wahlestedt's.  The two men met several years ago through a connection with an early stage investment firm in Boca Raton.

The heart of the new company is a novel technology licensed from Scripps Research that is based on the therapeutic potential of what are known as non-coding RNAs, small molecules that do not produce proteins and that were once thought of as little more than evolutionary leftovers. However, as Wahlestedt and other scientists have discovered, these non-coding RNAs have been shown to play a vital role in gene expression, a process critical to a number of different disease states. 

There are several different types of non-coding RNA, including microRNA and small interfering RNA (siRNA).  MicroRNAs regulate gene expression and have been shown to have links to both cancer and heart disease; siRNA is part of a pathway that inhibits gene expression and is critical to the immune system and how it responds to viral infection. 

The technology licensed by Wahlestedt and Collards is broad based and, at this stage of the game, will take a significant amount of investment to develop commercially. That seems to have only whetted Wahlestedt's enthusiasm for the project.

"We have licensed a fairly broad patent with many different targets in major therapeutic areas that fall under the non-coding RNA umbrella including metabolic disease and cancer," Wahlestedt said, "Depending on the specific nature of the RNA involved, they can either elevate or suppress gene expression. These things can be used in a number of important ways – to treat disease or as diagnostic markers or tools. All in all, they have some very significant therapeutic potential. "

A recent study highlighted some of that potential. In the July 2008 issue of Nature Medicine, Wahlestedt showed for the first time not only how complicated the human genome actually is, but just how important those seemingly extraneous parts can be.

The Nature Medicine study clearly demonstrated that a specialized form of noncoding RNA was directly linked to increased levels of amyloid plaque in the brains of Alzheimer's patients.

"Our in vivo studies show that this may turn out to be one of the keys to the pathological progression of the disease," Wahlestedt said.

Building in South Florida

Wahlestedt, 49, has been with Scripps Florida since 2005.  Prior to that, he was the founding director of the Center for Genomics and Bioinformatics at the Karolinska Institute in Stockholm, Sweden. His interest in non-coding RNA goes back several years, and he has been a pioneer in the development of better targeting strategies through the use of antisense RNA, siRNA or small molecules that target RNA.

He also spent a decade directing drug discovery and genomics efforts in the pharmaceutical industry, so the idea of his own biotechnology start-up isn't entirely alien to him.

"In my former life, I was also employed by Pharmacia – so I really had two jobs while at the Karolinska Institute, "Wahlestedt said. "Also, I've helped many companies over the years through my work on various scientific advisory boards but this is the first one I've done on my own.  It's really been a lot of fun, and it's what South Florida is looking for in terms of scientific development. We're very glad to be starting it here." 

Collard echoed Walhlestedt's comments.

"Ever since Claes and I met several years ago, we've been interested in doing something in South Florida," Collard said. "We looked at a lot of opportunities, but since Scripps Florida has been here, it has definitely raised the bar on what can be done in the area."

Collard and Wahlestedt worked closely with the Scripps Research business development offices both in Jupiter and in La Jolla, California to secure the patent. 

 "Everyone at Scripps Research was extremely helpful in getting the patent and in making sure that everything was done by the book," Wahlestedt said.

Scripps Research actively encourages scientists to think entrepreneurially, both in terms of seeking funds for their research, and to develop the business potential of their research. Wahlestedt's company is the second spin-off for Scripps Florida; the first, Xcovery, was launched in 2006 by Scripps Research scientist Chris Liang.

Officially started in June of this year, cuRNA quickly hired a high level bioinformatics consultant to identify potential targets; a second Florida employee was hired full time in July. More recently, the company has begun buying some highly specialized equipment, and setting up its own laboratory in the offices of another biosciences firm in Palm Beach Gardens. 

Wahlestedt said that eventually he expects to identify specific compounds for some of these broad therapeutic targets, to conduct further research and to register additional patents. But biotechnology is a long term process and those developments are far in the future. Right now, both men have their hands full getting cuRNA firmly established.

The Scripps Florida affiliation has been a strong selling point.

"One of the best surprises has been to discover that there are so many talented people who want to live in South Florida and work with Scripps Florida scientists," Collard said. "They're interested because of proven leaders like Claes who are at the cutting edge of biomedical science.  We're looking at the best people in the world and we hope some day to be hiring more than a few of them for cuRNA."

About The Scripps Research Institute

The Scripps Research Institute is one of the world's largest independent, non-profit biomedical research organizations, at the forefront of basic biomedical science that seeks to comprehend the most fundamental processes of life. Scripps Research is internationally recognized for its discoveries in immunology, molecular and cellular biology, chemistry, neurosciences, autoimmune, cardiovascular, and infectious diseases, and synthetic vaccine development. Established in its current configuration in 1961, it employs approximately 3,000 scientists, postdoctoral fellows, scientific and other technicians, doctoral degree graduate students, and administrative and technical support personnel. Scripps Research is headquartered in La Jolla, California. It also includes Scripps Florida, whose researchers focus on basic biomedical science, drug discovery, and technology development. Currently operating from temporary facilities in Jupiter, Scripps Florida will move to its permanent campus in 2009.

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