Theater Session: The First Potential DMOAD Therapy, TG-C: Intra-Articular Cell-Based Gene Therapy with Long-Term Safety and Insight into Delaying Total Knee Arthroplasty

Presenter: 

• David Hunter

Abstract:

Osteoarthritis (OA) is a progressive degenerative joint disease with limited treatment options that address the underlying pathology. TissueGene-C (TG-C) is an investigational intra-articular cell-based gene therapy currently under clinical development for the treatment of knee OA. It is being evaluated for its potential as a disease-modifying osteoarthritis drug (DMOAD), with a mechanism of action designed to deliver sustained therapeutic activity through localized gene expression following a single injection.

This theater session highlights long-term safety data from a U.S.-based long-term safety study (LTS), including study design, subject demographics, and adverse event (AE) findings. Malignancy incidence was evaluated in comparison to age-adjusted population data from the Surveillance, Epidemiology, and End Results (SEER) Program and did not indicate an increased risk. In addition, total knee arthroplasty (TKA) outcomes were evaluated, providing preliminary insights into the potential of TG-C to delay surgical intervention.

TG-C has demonstrated encouraging therapeutic effect in a previous Phase 2 study, with symptomatic benefits lasting up to two years post-injection. Currently, two pivotal Phase 3 studies are underway to further evaluate its efficacy and safety. Annual safety data updates over the past five years have revealed no new safety signals to date, and AEs observed have been consistent with expectations in the OA population.

In conclusion, current findings support the continued development of TG-C as a potential DMOAD. The favorable long-term safety profile, along with observed trends in symptom improvement and delayed TKA, reinforce its therapeutic potential. Ongoing Phase 3 studies are expected to further characterize these benefits. Patient safety remains a top priority, with robust monitoring integrated throughout the development program.