Acceleron Pharma (www.acceleronpharma.com) was established in 2003 to develop biotherapeutics for musculoskeletal, metabolic, and cancer-related diseases. The company’s founders not only had novel insights about how to control protein families involved in tissue growth and repair, but they also set up their own in-house GMP-manufacturing facility. “From the start of the company, we have invested in basic biology research and a manufacturing infrastructure,” says Steven Ertel, vp of business development at Acceleron in Cambridge, MA.
Acceleron’s technology platform targets the growth and differentiation factor (GDF) and bone morphogenetic protein (BMP) families, both members of the TGF-beta superfamily. Within these well-studied protein families are potent factors that modulate the growth of various tissues, including bone, muscle, fat, and blood vessels. Acceleron was founded by Wylie Vale at the Salk Institute for Biological Studies in LaJolla, CA, Tom Maniatis at Harvard University, Mark Ptashne at Memorial Sloan-Kettering Cancer Center, Jasbir Seehra, Ph.D., now CSO at Acceleron, and John Knopf, Ph.D., executive vp of corporate development and interim president of Acceleron.
Other companies have tried to create therapies to control the GDF and BMP families by creating ligands that turn up the biological activity of bone and tissue generating factors. This strategy, however, has produced few successful drugs, except for a small number of products that are used locally in spinal fusions or to repair nonunion fractures, where gaps need to be filled between badly damaged bones. These approved drugs have limited application because they do not act systemically and cannot be used to treat diseases such as osteoporosis.