Open Competition 3 - Biotechnology

Gene Delivery System for Bone Regeneration

Sponsor: Regeneration Technologies, Inc.

• Project Performance Period: 10/1/2001 - 9/30/2004
• Total project (est.): $2,197,038.00
• Requested ATP funds: $1,995,319.00

Every year, an estimated 33 million people sustain musculoskeletal injuries that theoretically could be treated with bone grafts. The same type of treatment could benefit many of the nearly 10 million Americans who have osteoporosis, because bones heal very slowly in older patients. Regenerative Technologies plans to design an orthopedic implant containing genetic material that will help modulate wound healing to promote bone growth in patients with significant bone defects. Biomimetic bone-derived materials in the implant will attract the body's mesenchymal (connective tissue) cells, into which new genetic material will be delivered at the appropriate time. The new material is a mouse gene transcription factor that regulates the expression of extracellular matrix genes and heavily influences cell differentiation. The idea is to change the fate of the mesenchymal cells to accelerate mineralization of skeletal defects, thereby restoring both form and function to affected bones. The company will determine, both in vitro and in animals, whether the new gene will induce cells migrating into the implant to differentiate into bone cells, and if so, take measures to increase the efficacy of the graft. The primary difficulty is that the gene must transfect cells and reach their nuclei; achieving transfection in living things is a significant technical challenge. The proposed technology is unique because the effects are precise and direct on all responding mesenchymal cells. The Bone Tissue Engineering Center at Carnegie Mellon University (Pittsburgh, Penn.) will perform the animal studies. ATP support is needed because of the project's high technical risk and the long period of research needed (6-9 years, including clinical trials) before commercial deployment. If successfully developed and commercialized, the orthopedic implants will improve clinical outcomes for patients with bone injuries and deterioration, reduce hospital stays, and return billions of dollars to the economy in medical cost savings and reductions in lost earnings. The graft technology also may be useful in artificial joints and other appplications, such as in a minimally invasive method of prophylatically treating patients at high risk for bone fractures.

For project information:

Quenta Vettel, (386) 418-8888
qvettel@rtix.com

ATP Project Manager

Mrunal Chapekar, (301) 975-6846
mrunal.chapekar@nist.gov