RNAi drugs startup Kylin Therapeutics is taking aim at cancer with a drug-delivery technology it recently licensed from Purdue University, according to company officials.
However, additional details about the specific indications Kylin expects to pursue are being kept under wraps until the company announces an industry partnership in the next few weeks.
Earlier this month, Kylin announced that it had acquired an exclusive license to so-called packaging RNA, or pRNA, technology developed at Purdue University by the company’s scientific founder, Peixuan Guo.
Although Guo and his colleagues at Purdue have already conducted some animal studies using the pRNA technology to delivery therapeutic agents for hepatitis and cancer, the company sees the most promise in the latter, Kylin CEO and co-founder Eric Davis told RNAi News this week.
“We’re most interested, at the present time, in cancer [since] we have the most data of [the technology’s] efficacy in that area,” he said.
“From our standpoint, it’s just a decision we’ve made to go after cancer,” Joseph Trebley, Kylin’s director of technology development, added. “In our minds, we don’t see the platform technology as limited” to particular disease areas.
Davis declined to comment more specifically on Kylin’s pipeline — although he noted that “in vivo studies are ongoing now in several areas” — until the company formally announces its cancer collaboration with an undisclosed industry partner.
“We have to work with them to work out [the details of our therapeutic programs and] to decide how we’re going to tell people about it,” he said. “The point is, though, that we’re further along than just having a license” to delivery technology.
Gear in the Machine
Kylin was incorporated about a year ago by two venture capital firms: In-Vivo Ventures, of which Davis is a co-founder, and Golden Pine Ventures. But it wasn’t until December, when the company formalized its licensing agreement with Purdue for the pRNA technology, that its operations really began.
According to Davis, West Lafayette, Ind.-based In-Vivo became familiar with the pRNA technology as a result of its proximity to Purdue, and had been eyeing the technology for almost two years for its potential in delivering RNAi drugs.
But it wasn’t RNAi’s high profile that caught the VC firm’s attention so much as the data supporting the pRNA technology, he said.
“We don’t really work on the idea of finding a hot market and looking for a technology in that market,” he said. “We look for strong technologies that have opportunity and then pursue them. It’s our good fortune that the [RNAi] market is pretty hot right now.”
Packaging RNA is derived from bacteriophage phi29 and is “a gear in the DNA packaging machinery,” Trebley explained. “One of the things a phage has to do to form is tightly package all of its genetic material within the capsid. This is a very energy-dependent process and requires a lot of very complicated molecular machinery,” including pRNA.
“In nature, [pRNA] exists as a hexamer,” he said. “Each monomer is a single strand of RNA that folds back on itself to create three domains, [two of which are] loops that interlock with the other monomers to form the multimer.”
The third domain, however, “is a double-stranded region, and what Guo discovered at Purdue was that you can replace this double-stranded region with therapeutically active RNA molecules like siRNA, aptamers, and ribozymes,” Trebley added. “Not only does the pRNA maintain its ability to form the multimeric structure, but the [therapeutic RNA molecules] all maintain their function, as well.”
In May 2006, Gou and colleagues published data describing the construction of folate-conjugated pRNA for the delivery of siRNAs to cancer cells.
More recently, he reported that pRNA could be used to escort hammerhead ribozymes into cancer cells.
We don’t really work on the idea of finding a hot market and looking for a technology in that market. We look for strong technologies that have opportunity and then pursue them. It’s our good fortune that the [RNAi] market is pretty hot right now.”
“You can imagine with this system there is a lot of capability and a lot of flexibility,” Trebley said. “What Kylin Therapeutics is doing is applying [this] to RNA-based therapeutics.”
Davis added that although Kylin is focused on therapeutic RNAi, the company is open to licensing the technology to firms interested in applying it to other therapeutic RNA agents such as aptamers or ribozymes.
But before any such licensing deal is struck, Kylin is faced with the task of establishing itself as a player in the RNAi drugs game.
“Our model is to surround the technology with a management team and … complement the scientific founders with a scientific team to exploit the technology and get it transferred out of the academic lab into a corporate environment,” Davis said. “We’re well underway at this point.”
In addition to Davis and Trebley, Kylin has already appointed three board members: its chairman is Christopher Meldrum, managing director of Golden Pine Ventures; and it two directors include Mark Braughler, who most recently served as vice president of translational medicine at Intrexon, and Homer Pearce, who recently retired from Eli Lilly, where he held a number of positions including vice president of cancer research and clinical investigation.
Kylin also has two members on its scientific advisory board including David Johnston, vice president and CSO for the clinical trials division of LabCorp, and Olke Uhlenbeck, chairman of the department of biochemistry, molecular biology, and cell biology at Northwestern University.
But “like any startup that is at this stage, we still have team members to add,” Davis said. “We have the technology experts for pRNA, and now we need to complement them with some industry experts and industry technicians.”
He declined to specify how many employees Kylin has or expects to add in the near term, but said that an announcement about some recent additions to the company is expected to be made next week.
“Right now, we’re still fairly below the radar, and I don’t necessarily want to pop out and be tracked by everybody,” he said.