SAN DIEGO - RNA, a silent guest at the 50th birthday party for its counterpart, DNA, could be having its own coming-out party soon - if expectations become fulfillment, if the current intellectual property morass is sorted out and if institutions pool their knowledge through alliances to focus on marketed human therapeutics.
That sentiment was prevalent at the 2nd annual siRNA in Drug Discovery and Development Conference, sponsored by the Strategic Research Institute and held at the Marriott Del Mar hotel here. The conference was primarily attended by researchers, scientists and venture capitalists, and was heavy on sharing the direction and progress of laboratory research and the use of reagent tools relative to RNAi-based drug development.
RNA interference, or RNAi, refers to the relatively new discovery that small RNA can initiate gene suppression, which could cause cells to "turn off" the negative disease-specific function of genes.
Long dismissed as an important, yet not crucial, component in gene therapy, RNA stands poised to potentially become the most important recent discovery in biotechnology and human therapeutics.
The fact that there are presently more RNAi litigation cases than there are RNAi products puts a perspective on the hype associated with the technology. Litigation in the current patent landscape is a major issue facing RNAi technology, as there is confusion over inventorship, conception and patent filing for such an emerging technology.
That issue was at the heart of a session titled "The Intellectual Property Landscape in the RNAi Industry," which began with a talk by Richard Warburg, partner at Foley & Lardner, then concluded with a panel discussion, in which he participated.
Warburg referenced more than a dozen incidents involving patent oppositions, interferences and litigation, and related them to the difficulty associated with biological applications, particularly with regard to getting the word out to an immense number of industry players that a claim has been staked.
Warburg discounted the theory that the U.S. Patent Office was a culprit in the current chaos and said, against public perception, a government agency was actually aggressively and effectively working to eliminate a quagmire.
"The U.S. Patent Office, over the last few years, has actually increased its number of patent attorneys, while decreasing the waiting time for decisions, in spite of its continuing tradition of training its employees, then losing them to private corporations," Warburg said.
Warburg suggested several ways to eschew confrontation: concession in order to settle litigation, increased willingness to license technology and participation in patent pooling.
"Patent pooling may seem like a compromise, but a portion of the most promising technology right now beats either none or valuable time lost in litigation at the most crucial time," he said.
Some speakers stressed the importance of collaborating in order to reach the ultimate goal of providing human therapeutics in a timely manner. A common concern was that the desire to be the "first" might protract research that could benefit from pooled knowledge, resulting in faster development.
David Haen, director of business development at Los Angeles-based CytRx Corp., which has retooled its strategy to rely on alliances that already have made inroads into the RNAi technology, pointed out the virtues of the ground-breaking technology, as well as the focus of direction his company has assumed.
He explained that CytRx licensed out its former lead product, Flocor, and all other co-polymer technologies to concentrate solely on RNAi, then President and CEO Steven Kriegsman flew to Boston and did not leave until securing exclusive worldwide royalty-bearing license agreements with the University of Massachusetts Medical School (UMMS) to use its RNAi gene-silencing technology in the development of treatments for ALS, obesity and Type II diabetes. (See BioWorld Today, April 22, 2003, and Oct. 24, 2003.)
As a result of that deal, UMMS became the second-largest CytRx shareholder overnight, trailing only Kriegsman.
If the expectations do become reality, and RNAi technology eventually produces blockbuster drugs, there is a possibility that CytRx could miss out on potentially epic deals with some major pharmaceutical companies, but Haen asserts they are content to forge ahead with the partners who shared their vision near the beginning of the journey.
"While there is the possibility that future drug development opportunities with pharma giants may be affected by our early alliances, we in no way second guess ourselves, inasmuch as we definitely would not even be in the position we are now without the current deals we chose to make," Haen said. "We feel comfortable with our alliance strategy and the University of Massachusetts' capacity to maintain its position as a pioneer and leader in the RNAi field."
Another notable move was the recruitment of Craig Mello, co-discoverer of the revolutionary RNA interference technology, to the scientific advisory board of CytRx.
Mello, who still works for UMMS, is considered a technological innovator in the field of molecular genetics. He, along with Andrew Fire at Stanford University, received the first approved patent for the use of double-stranded RNA for gene silencing, and subsequently put the technology on the map.
"Certainly, Dr. Mello's faith in CytRx's commitment and potential to developing RNAi therapeutics was a tremendous boost to our pursuit of drug development in this field," Haen said.
The conference ended Wednesday.