now painfully visible in the declining research productivity of the pharmaceutical industry. The early promise of biotechnology was to take drug discovery away from such shots in the dark to a model premised upon expanded knowledge of the molecular pathways of disease. The success of Genentech and its kin is testimony to the validity of this approach, but it is limited to diseases with wellknown causes. Too many diseases are the result of still-unknown pathologies. “I know the problems of pharma and they start at the beginning: choosing the wrong biology, the wrong targets and then pushing forward whatever you choose to the tune of billions of dollars,” said Berta Strulovici, iPierian’s vice president for research and chief technology officer, who previously held executive research positions at Merck & Co. “iPierian’s objective is to streamline drug discovery by getting the biology right,” she added.
Courtesy of iPierian
The Foundation of a New Platform Technology The unsung heroes of the biotech revolution were the makers of biological tools and enabling technologies. Although Kary Mullis ultimately received the Nobel Prize for the development of a technique for generating thousands to millions of copies of a particular DNA sequence, the further industrialization of these processes required many advances in hardware, software and biology. The same will be true for adult stem cells. Biotech pioneers such as the Cetus Corporation, where Mullis was employed, often had to invent and produce their own biological tools, but today there is a community of sophisticated vendors that has grown up along with the industry. These toolmakers present the first broad partnering opportunity for iPierian, which forms an element of its business model. Walker said that iPierian plans to strike a partnership with a single outstanding company for an exclusive long-term relationship, where the partner can turn iPierian’s innovations into product opportunities, so the company can access upfront capital as well as an ongoing stream of royalty payments. The partner will enable iPierian to monetize the offshoots of industrializing adult stem cells. Though “industrialize” is a tidy sounding verb, the actual task of scaling up stem cell processes from laboratory quantities to commercial production will be far from trivial. Yields must improve by at least an order of magnitude, if not two, coupled with a concomitant improvement in purity. “Working closely with a com-
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Differentiated motor neuron cell culture from healthy adult stem cells.
pany that is in the business of turning innovation into ready-to-use products will benefit iPierian directly by cutting the time we have to spend reducing science to practice,” Walker said. “And the collaboration will also benefit the broader life sciences community by making the fruits of our labor widely available.”
Two Models for Pharmaceutical Partnerships The same philosophy informs Walker’s, and now Venuti’s, partnering strategy with pharmaceutical and biotechnology companies. Walker foresees two distinct models. One model is for specific diseases that iPierian is focusing on internally, like spinal muscular dystrophy, a neurodegenerative disease that is the leading genetic cause of death in infants and toddlers. A second model will serve companies working in broad therapeutic areas, such as metabolic disease, cancer and cardiology. In both cases, however, iPierian will seek to partner much earlier than is common practice today, where collaboration is usually deferred until proof-of-concept, which may not be achieved until a molecule is well advanced in the three stages of human clinical trials required by the U.S. Food and Drug Administration. That risk-averse approach makes sense to small companies seeking to maximize the monetization of a core asset, and to large companies looking for product candidates with near-term potential. But with a breakthrough
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