Genascence’s GNSC-001, a gene therapy delivering an interleukin-1 receptor antagonist for knee osteoarthritis, secured FDA Regenerative Medicine Advanced Therapy (RMAT) designation. Administered via single intra-articular injection, GNSC-001 aims to provide sustained inhibition of IL-1-mediated inflammation, addressing joint pain and cartilage destruction. Phase I and Ib studies demonstrated safety, tolerability, and biological activity. RMAT status facilitates expedited development, reflecting the potential to transform treatment paradigms for a debilitating and prevalent joint disease.