Scientific Approach

Acceleron’s Novel Approach Unlocks the Therapeutic Potential of the GDF Family

Acceleron applies its unique insight on the Growth and Differentiation Factor (GDF) protein family to develop novel biotherapeutics to modulate the growth of bone, muscle, fat and the vasculature. The initial attempts by other companies to capitalize on the therapeutic promise of the GDF family were predominantly based on the concept of administering GDF family ligands to achieve a therapeutic effect. Despite the proven biological activity of these compounds, progress in drug development was thwarted by technical challenges.

Acceleron has taken a fundamentally different approach to harness the power of the GDF family. Rather than administer a GDF ligand, our approach is to administer a GDF antagonist to inhibit the activity of certain members of the GDF family. Because both positive and negative regulators exist in the GDF family, we are able to achieve the desired therapeutic outcome, while bypassing many of the technical challenges faced by other companies.

Translating GDF Biology into Therapy

This novel approach has been the key to unlocking the therapeutic potential of the GDF protein family. We have focused on discovering and engineering novel GDF antagonists, leveraging our deep understanding of its biology. This has yielded a rich pipeline of powerful, systemically-active biotherapeutics that can modulate a broad spectrum of tissue types.

For example, in situations where we seek to increase tissue mass and strength - such as bone in multiple myeloma or muscle in muscular dystrophy - our approach is to inhibit negative regulators of tissue growth. By inhibiting the negative regulators of tissue growth, we free the body’s normal, positive biochemical machinery to increase bone and muscle mass and strength.

Conversely, in situations in which there is inappropriate tissue proliferation, such as the vasculature in cancer or fat in obesity or diabetes, our approach is to inhibit these positive regulators of tissue growth, thereby freeing natural negative control of these processes to decrease tissue mass.

A Highly Productive and Efficient Platform for Drug Development

Acceleron’s strategy combines its approach to drug discovery with efficient, reliable and proven technologies for drug discovery and development. Utilizing familiar technology will reduce technical risk, improve cycle times, allow us to capitalize on regulatory experience and enhance physician comfort with using our drugs. Furthermore, Acceleron has brought these technologies “in-house” to streamline and avoid potential bottlenecks in the development of our agents. Together, all of these factors work to increase the speed and improve the probability of creating drugs that will dramatically improve the lives of the patients.

Therapeutic Areas That Acceleron is Targeting