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Nexell: Playing to Win or Playing Board Games?

This article was originally published in Start Up

Executive Summary

Nexell failed to take advantage of the financing window for biotech last year, instead opting to establish a $25 million equity line of financing in January 2001. Some observers say its former parent, Baxter, is still calling the shots, including blocking any financing activities that would significantly dilute its investment. How aggressively Nexell draws down the $25 million may serve as an indicator of management's current commitment and belief in itself.

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The Bricks and Mortar of Personalized Medicine

With an increasing number of therapies derived from a patient's own cells, companies now have experience to draw on in designing commercial infrastructures for delivering these un-drug-like products. Indeed, armed with some small experience around how well they fit--or don't fit--into standard medical practice, cell-based therapies may finally be going commercial. And not only in terms of a transition from acute to chronic wound healing indications, where commercial success was first anticipated, but also as stem cell therapies, which would open up a multi-billon dollar opportunity in regenerative medicine, including replacements for organ and transplant surgery and cures for diabetes and neurological diseases such as Parkinson's Disease and Alzheimer's Disease, as well as autologous cancer immunotherapies. These companies are finding value in the experiences of earlier, technology-constrained companies whose attempts at developing cell therapies failed--Applied Immune Sciences and Systemix Inc., for example, both of which have virtually disappeared. Companies today are trying to overcome the challenges of product preparation, process standardization, and delivery. Other first generation cell therapy companies, like Genzyme Tissue Repair and Cell Genesys Inc., are still around. That a number of senior executives at today's leading-edge cell-therapy companies cut their eye teeth at those first-generation start-ups attests to their continuing belief in the broad commercial opportunities these technologies offer. But significant questions remain: Can companies solve the production economics of delivering products that require cell culture and expansion using a centralized, company-controlled laboratory model? The alternative, a decentralized technology transfer approach, would bring the product closer to the patient, but raises the question of whether the technology exists to standardize processes and satisfy regulators' quality concerns. Then, if these issues are solved, one must ask whether a hospital lab is the right setting for a commercial operation. Are there better commercial infrastructures, such as blood processing centers, with which companies could form alliances? And does the prospect of developing off-the-shelf, allogeneic-cell therapies-mass-produced stem cells that can pass the immune system undetected or vaccines derived from shared tumor antigens from cell lines, for example-threaten to obsolete autologous products before they become profitable?

Rewriting Fate: The Stem Cell Transplant Business

Physicians, patients, and companies can now see the stem cell transplant business taking shape. Stem cell trials are finally in progress, allowing investors and corporate partners to finally see just how effective this multi-billion-dollar opportunity to replace many highly problematic organ transplants, and dramatically expand the procedures into new areas, can be. Beyond the lack of clinical conformation, stem cell transplantation therapy has been held back by the inability to culture large quantities of cells--as with whole organ transplants, there simply isn't enough tissue to go around. For the most part, companies have had to propose autologous transplantation strategies or, at best, transplants from one donor for just a few patients--an economically difficult-to-justify business. Geron, the only player in this group to have benefited from the last nine months of biotech stock mania, holds a key competitive advantage: its ability to use embryonic stem cells (ESCs) to create, theoretically, nearly infinite quantities of any cell in the body. But recent developments have stirred the competitive pot: a number of companies have created methods for growing enough adult stem cells to provide them to significant numbers of patients. Meanwhile, researchers have transformed fully committed adult stem cells into cells of different lineages, raising the possibility that alternatives to ESCs for tissue replacement or repair exist. The transplant replacement business--regenerative medicine--has caught the public's attention. Nonetheless, the stem cell industry has remained a start-up's trade, and it's easy to see why: large-company experiences in the area have not, for the most part, been happy ones, as companies face the issues of installing unpharma-like infrastructures to deliver personalized therapy, as well as ethical concerns, which must be given appropriate consideration. But these and other hurdles shouldn't obscure the growing opportunity. Stem cell therapy, so long a therapeutic chimera, now seems ripe for commercial exploitation.

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