Kolon’s cell-based gene therapy misses key endpoints in phase 3 osteoarthritis trial

Kolon TissueGene reported that its phase 3 study of TG‑C, a cell‑based gene therapy for knee osteoarthritis, failed to meet its co‑primary endpoints. Patients receiving the therapy showed no statistically significant improvement in pain or function compared with placebo after one year.

TG‑C combines human allogeneic chondrocytes with an irradiated human embryonic kidney cell line that produces TGF‑β1. The product had received South Korean approval in 2017, but that clearance was withdrawn two years later after a mix‑up over the cell type used.

The U.S. trial enrolled 531 participants across 27 sites after a delayed start caused by an FDA hold. Both the primary and all key secondary measures were missed, indicating the therapy did not demonstrate the expected clinical benefit.

Kolon now awaits results from a second, similar phase 3 trial scheduled for release in October, which will determine the company’s next steps for the osteoarthritis program.