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The promise of stem cell research is being realized by biotechnology companies bringing the discoveries of great scientists out of the academic world, into therapeutic development, and to the market where they can be delivered to patients in need. Athersys Incorporated (Athersys), based in Cleveland, Ohio, is one of these biotechnology companies. Considered a clinical-stage biotechnology company, Athersys is developing therapies created using adult stem cells isolated from bone marrow and other tissue sources. They refer to these stem cells as multipotent adult progenitor cells (MAPC), which can be expanded on a large scale following isolation from a donor. Athersys’s current programs are based in part on the proprietary product under the registered trademark name MultiStem but also involve the use of their RAGE (Random Activation of Gene Expression) and other proprietary technologies used to identify various drug targets. These are described below.

History and Collaborations

Athersys was founded in October 1995 by Gil Van Bokkelen, PhD, and John Harrington, PhD, both who, as of 2014, continue to lead the company as chief executive officer and as chief scientific officer, respectively. Both also serve as directors. Dr. Harrington was a leader in the development of the proprietary RAGE technology. Described further below, RAGE is a novel gene activation approach that has applications in gene discovery, drug discovery, and commercial protein production, and has been in the demand of several other companies for use since its development. In 1997, Harrington helped Athersys create the first synthetic human chromosome. He now oversees process development, manufacturing, and clinical development.

In 2000, Athersys filed for initial public offering (IPO) with the goal of raising $115 million, as a functional genomics company. However, the proposed IPO was withdrawn six months later and instead went public in 2007 via a reverse-merger (or reverse IPO), with $65 million of funding coming from private companies Radius Venture, OrbiMed Advisors, RA Capital Management, Accipiter Capital Management, Hambrecht & Quist Capital Management, MPM BioEquities, and Pappas Ventures. The company now trades on the NASDAQ exchange under the ticker symbol ATHX.

In 2002, the two founders were the recipients of Ernst & Young LLP’s Entrepreneur of the Year Award in Biotech. The self-described “supersonic” projection of Athersys in 2002 continued through 2007, when the company advanced commercial collaborations with Bristol-Myers Squibb Company and Angiotech Pharmaceuticals Incorporated, representing success in applications of its technologies to pharmaceutical/drug applications. Athersys continues today as a biotechnology and regenerative medicine company whose primary activities are the discovery and development of therapeutic product candidates. As such, the company forms collaborations with other larger companies to take promising product candidates further to commercialization. Bristol-Myers Squibb primarily benefited from its multiyear collaboration with Athersys with RAGE technology, providing multiple successful drug targets in a variety of therapeutic areas, which have become active drug development programs at Bristol-Myers Squibb. On the other hand, the collaboration between Athersys and Angiotech is more narrowly focused per the interests of Angiotech, with Athersys providing multiple animal studies that demonstrate the safe delivery of MultiStem via cardiac catheter and the provision with such delivery, of functional benefit in models of acute myocardial infarction. Further details of RAGE and MultiStem follow.

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