A CRISPR model, and other approaches to translational medicine

November 11, 2014 ctheodoropulos

Successfully translating novel science from small university labs to large pharmaceutical development groups is one of the more delicate dances in biotechnology partnering.

To explore this interface between academia and industry, SciBX Executive Editor Simone Fishburn gathered a panel of representatives from internationally renowned translational centers.

“Our problem is translating the research to what pharma is requiring,” explained Rainer Wessel, the Cluster Management Director for Ci3, part of the Translational Oncology (TRON) organization at Mainz University.

Rainer Wessel, the Cluster Management Director for Ci3

Rainer Wessel, Cluster Management Director for Ci3

If you talk to people from universities, very often once they have a very good paper, they think that is 90% to getting a deal done,” he said. “The basic goal for TRON is to translate early research into a pharma application, which often requires more money. It can be through straight up licensing if there are early assets. The second possibility is to create a company backed by venture capital to get to the proof of concept phase. And the third way is to mature projects further through institutions like Ci3.”

Dieter Link, the Senior Licensing and Patent Manager for Max-Planck-Innovation echoed this perspective, saying, “In pharma it all must begin with a patient need. What our scientists do is not a perfect fit. It is difficult to come up with patient driven programs when they are focused on basic research. But our medicinal chemists at the Lead-Discovery-Center in Dortmund, established in 2008, are there to fill in that gap by teaming up with the scientists at MPG in an early development project to generate licensable high quality lead structures—as it was already proven for example by the licensing of a lead structure to Bayer in 2011 that has only recently entered clinical stage in 2013.”

According to Issi Rozen, the Director for Strategic Alliances at the Broad Institute in Cambridge, “Our mandate is very different. We like value, but what we try to maximize is the science. When I look for partnerships, unless we believe the party on the other side of the table has the best science to take the program forward we will not move the program to them, even if the offer is more generous. We will trade value for the ability to move the program forward.

“Our partnerships are based on sharing a scientific vision,” he told BIO-Europe® participants. “What we worry about is getting into a three- or five-year collaboration where we cannot agree on what is the right next step for the science, the right research plan.”

A dramatic demonstration of this principle was seen with the Broad’s decision on advancing CRISPR (clustered, regularly interspaced short palindromic repeats), a new technology that speeds gene editing.

“Human genetic targets are more difficult,” said Rozen. “When we license a drug, a pharma partner is terrific on executing with molecules. Where we have a platform technology, something like CRISPR, though many pharmas are interested, we decided not to give the therapeutics aspect of that platform to pharma.”

Instead Rozen explained that a new entity, Editas Medicine, was formed and funded by venture capital partners.

“Here we have a team that spends 100% of their time thinking about the technology, versus a pharma that may think about it 5% of the time while spending the rest of their time trying to maximize the therapeutic potential,” he said.

Moderator Fishburn noted that one of the hurdles historically for translating science from academia is the time it takes to create an agreement, to get the IP sorted. Are we making progress, are we getting faster? she asked the panel.

“One of the ways to get agreement reached more quickly is to get the lawyers out,” suggested Michael Dalrymple, Director for Business Development at the MRC Technology, which manages the technology transfer needs of the United Kingdom’s Medical Research Council.

“Yet the fact of life is that this is a legal process and we are always starting from two polarized positions,” he continued. “Things that concern lawyers for warrants and liabilities are intrinsic to the system. Though it is always surprising how quickly things can go when there is a determination. Where there is a will there is a way.”

Translational Medicine Panel, BIO-Europe 2014

Translational Medicine Panel, BIO-Europe 2014

Rainer at Ci3 agreed, saying, “Whenever there is a good match, when there is someone who understands the project, someone who wants it, that is the way it will move forward.”

Rozen from the Broad Institute followed on Dalrymple suggestion about lawyers, adding that, “The key is to control the lawyers. The lawyer’s job is to protect a company from any possible risk. If there is a 0.1% chance of risk, the lawyers will go back and forth as much and as long as they want. Somebody needs to make a call at this point. Unless business development can assume some risk, the deal will take forever. Where there is risk we can accept, we should accept that risk.”

Noting that there are increasingly programs in the United States to encourage academic scientists to think of the commercial prospects for their work at an early stage, Fishburn asked if it isn’t time to start training and teaching European academics about the translational process, some of the practicalities of what it takes to make a drug that is commercially viable.

“We are a blue sky research institute, and we are still building this up and don’t yet have a clear strategy for focusing this work,” said Egenhard Link who is driving a wide ranging program to establish a basic research facility as the head of Technology Transfer for IST Austria.

“We can offer perhaps an idea of the context, but we won’t force anyone to do what they do not want to do. Actually, we don’t want them to think the world is bigger than the ivory tower where they are working.”


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