As interest in RNA interference increases with the start of the first ever clinical trial of an siRNA-based drug, and as efforts in related areas forge ahead, the time seems right to try to bring together the key oligonucleotide therapeutics players in a bid to nurture and grow the field.
This is exactly what a group of top researchers are doing as the recently formed Oligonucleotide Therapeutics Society begins to take shape.
“Various scientists working in the oligonucleotide field … have been talking about having a society devoted to this field, [and] it first came up probably close to 10 years ago in the heyday of antisense,” Art Krieg, CSO of Coley Pharmaceuticals and a vice president of the Oligonucleotide Therapeutics Society, told RNAi News this week. “Then antisense didn’t really blossom and flourish the way everyone had hoped, and the idea died.”
According to Cy Stein, professor at Albert Einstein College of Medicine, scientific advisor to Genta, and treasurer of the society, a resurrection of sorts took place in 2002 during a scientific meeting in Italy.
“This was an oligonucleotide therapeutics meeting, and it was held in a little town in Tuscany, not far from Siena,” he told RNAi News. At the meeting, Burkhard Jansen, former vice president of clinical development at Oncogenex, broached the idea of an oligo therapeutics society, Stein said, and while the concept was well-received, “it kind of sat for a while.” (Though the society was officially incorporated in December 2002, it has only been really active since around the beginning of this year, Stein said.)
But as Genta’s cancer drug Genasense moved closer toward regulatory approval and becoming a validation of antisense technology, and as RNAi made its way to the top of Science’s breakthroughs of the year list, interest in an oligo therapeutics society was re-ignited, Stein said.
“In the beginning, I was a little skeptical about starting a therapeutics society with no therapeutic products, but there was so much hype that I said, ‘we’ve got nothing to lose,’” Stein said.
Although Genasense was ultimately rejected by a US Food and Drug Administration panel in May, the oligonucleotide therapeutics field remains strong: Genta has said it remains committed to developing the drug, and work by antisense companies such as Isis Pharmaceuticals and AVI BioPharma proceeds apace.
Meanwhile, Acuity Pharmaceuticals said it has begun a phase I trial of its RNAi-based age-related macular degeneration treatment (see RNAi News, 10/8/2004), while Sirna Therapeutics has filed an investigational new drug application with the hopes of getting its own AMD drug into humans before the end of the year (see RNAi News, 9/10/2004).
Additionally, researchers continue to work on developing drugs based on such diverse technologies as aptamers, CpG oligos, and locked nucleic acids.
As such, “the time is really now right for the birth of an oligonucleotide therapeutics society to bring all [the different] groups [in the field] together,” Krieg said.
The driving forces behind the creation of the Oligonucleotide Therapeutics Society include Krieg, Stein, and John Rossi, a Beckman Research Institute scientist, Benitec collaborator, and president of the society. Also contributing to the society’s development is University Hospital Zurich’s Uwe Zangemeister-Wittke, who is also the society’s secretary; Alan Gewirtz, a professor at the University of Pennsylvania School of Medicine and the inventor listed on a patent application that is the core of Acuity’s RNAi intellectual property estate; and Rockefeller University researcher and Alnylam Pharmaceuticals co-founder Tom Tuschl.
At this point, these people, along with about 40 others, essentially represent the whole society.
“We haven’t even started really recruiting for members yet,” Stein said. That is something that will begin in earnest at the society’s first annual meeting, which is scheduled for Sept. 15-18, 2005 at Rockefeller University in New York, Rossi told RNAi News.
“We’re going for 500 to 1,000 [members in total],” Stein said. “That might not be realistic for the first meeting, but there are a lot of people working in the oligonucleotide therapeutics area now, and ultimately I think it’s a very reasonable goal.”
“In terms of the number of people we hope to get at the first meeting, if we can get 300 to 350, we’d be pretty happy,” he added.
According to the conference’s preliminary agenda, which was obtained by RNAi News, the society is putting together a full roster of high-profile researchers as speakers at the event. Among those confirmed to present are Peter Linsley of Rosetta Inpharmatics; Phillip Zamore from the University of Massachusetts Medical School; Mark Davis, a researcher from the California Institute of Technology and founder of Insert Therapeutics; Muthiah Manahoran from Alnylam; Judy Lieberman from Harvard’s CBR Institute for Biomedical Research; and Isis Pharmaceuticals CEO Stanley Crooke. Also scheduled to present are Tuschl, Stein, Krieg, and Rossi.
“It will [have] members [of the society] and industrial participants,” Stein said of the event. “This is not designed to be a purely academic, pie-in-the-sky-type deal — we really do want to have industrial participants and collaborators. It’s going to be a very interesting meeting with people coming in from all over speaking on all kinds of things.”
The conference will also include a follow-up meeting of the society’s board of directors, Rossi said.
The first board meeting, held in New York in May, included about 40 people — half from academia and half from industry — who were given an opportunity to speak about their work and their ideas for the society, Stein said.
“We all agreed that we wanted the society very firmly based in science, and that it would not simply be an industry lobbying organization or something like that,” Krieg noted.
“The people are committed to having an academic society in the sense that it’s not going to be controlled by any industrial agendas,” Stein added. “People were fairly definitive about that. It’s not [easy to do], but one advantage we have is that some of the companies are … a little bit jealous of other companies,” he said. “That allows us to steer a middle ground, the science, which to my way of thinking is the high ground.”
That is not to say that the society has no use for industry input. It is, after all, companies that conduct late-stage development of drugs and provide much of the funding for organizations like the Oligonucleotide Therapeutics Society.
“The funding for the society will come from industry, of course, and that is natural and good,” Krieg said. “One of the good things is that we do have so many companies developing technology in this area [and] that gives us the possibility to have a strong funding flow. Then the society can serve both the needs of industry and the needs of the scientists in the field.”
Stein noted that the society is currently in the black, having raised more money during the May meeting than it needed to run the annual meeting next year. “We’ve got a nice chunk of change in our treasury. It’s all [from] … companies, from the very, very big to the very, very small, that want to have access and want to meet the people and want the opportunity to pitch their wares.”
He noted, however, that the costs of putting together the annual meeting are significantly greater than those associated with the meeting in May, and that the task of raising additional funds has fallen to him as the society’s treasurer.
“Basically, I have to go around to contacts and ask them to pony up,” Stein said. “As they say in New York: You have to be a schnorrer.”
Despite the successes of the first meeting, the Oligonucleotide Therapeutics Society is still without a definitive plan for how it will conduct its mission of being “an open, non-profit forum to foster academia and industry-based research and development of oligonucleotide therapeutics.”
“This is a thing we’ve been talking about for awhile,” Stein said. “Basically, it’ll be what people want. Right now, we envision it as a way … to get everybody interested in this field together at one place at one time,” he said, adding that the society is also considering arranging for its members to receive discounts on the journal Oligonucleotides, which became the official journal of the society around the beginning of this year after changing its name from Antisense and Nucleic Acid Drug Development.
Rossi and Krieg see a potentially even larger role for the Oligonucleotide Therapeutics Society, one that will be fleshed out during the annual meeting.
“We’ll be [advancing the field] primarily through national meetings, [but] we’ll [also] have resources available on the web page” to supplement Oligonucleotides, Rossi said. “Eventually, we’ll probably have some sort of formal lobbying capacity where we can actually make statements to the press on behalf of [the] oligonucleotide therapeutics [industry].”
According to Krieg, “One thing we’d like to do is to try to set standards in this field for what are appropriate experimental methods and controls and so forth. This was one of the issues in the antisense field early on,” he said. “You had people publishing papers and saying they were studying antisense, when really what they were doing was nonsense.”
Krieg also said that the society is trying to work out whether it will have a role in facilitating interaction between its members, for example through the promotion of intellectual property cross-licensing deals.
“We haven’t really taken a position on that — it’s still pretty early [since] no one really knows what the key IP is going to be in this field,” he said. “We’re far enough away from having a product that it’s going to take more time for that to settle out, [and] … I don’t know realistically how easy it’s going to be for us to influence that, short of providing moral support and agreeing it’s important.”