Year 4 (NCE)
During the last four years we have developed a novel therapeutic candidate for the treatment of severe, non healing, fractures. We have shown that massive fractures can be healed by the attraction of resident stem cells to the fracture site followed by their activation using ultrasound-assisted gene delivery. The treatment resulted in a transient expression (about a week) of the gene in stem cells within the fracture, which was sufficient to bridge all fractured bones in the study, eight weeks post-treatment (compared to the control bones, which did not heal). In the last year of the study we conducted a meeting with the FDA and shared with them our results. Based on the supportive feedback from that meeting we have composed a plan to address all the requirements of the FDA in order to obtain an approval to commence clinical trials. We have also generated new genetic material that would be adequate for safety testing of the therapy prior to human trials. The new material was tested and shown to efficient in bone repair.