Gene Therapies Aim to Treat Knee Osteoarthritis With Single Injections

Developers of gene therapies are taking the intra-articular route to deliver proteins and molecules directly to the site of knee osteoarthritis.

Published on Mar. 9, 2026

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Researchers are exploring several gene therapy approaches to treat knee osteoarthritis, including delivering anti-inflammatory proteins like IL-1 receptor antagonist and TGF-beta 1 directly into the affected joint through a single injection. While some therapies have shown promise in early trials, experts say a combination of approaches targeting multiple pathways may be needed to effectively treat this complex joint disease.

Why it matters

Knee osteoarthritis is a common and debilitating condition, and current treatments like corticosteroid injections only provide temporary relief. Gene therapies that can continuously reduce inflammation and potentially restore joint function with a single injection could be a major advancement in managing this chronic disease.

The details

The gene therapy approaches in development for knee osteoarthritis include viral vectors like adeno-associated virus (AAV) that deliver anti-inflammatory proteins directly into the joint, as well as non-viral plasmid therapies that target nerve endings. While some have shown acceptable safety and sustained therapeutic protein expression in early trials, experts say combining therapies that address multiple pathways involved in osteoarthritis may be necessary for optimal results.

  • In 2024, the FDA granted Fast Track designation to GNSC-001, an IL-1 receptor antagonist gene therapy delivered via an AAV vector.
  • Results from a phase 2 trial of the plasmid-based therapy XT-150 were reported in 2023, showing lower response rates compared to placebo.
  • Top-line data from two phase 3 trials of the cellular gene therapy TissueGene-C are expected to be announced in July 2026.

The players

Christopher Evans

A researcher at the Mayo Clinic who has developed a gene therapy for osteoarthritis.

Daniel Grande

A biomedical researcher at the Feinstein Institutes for Medical Research at Northwell Health who is excited about the resurgence of gene therapy.

Nancy Lane

A rheumatologist and researcher at the University of California Davis School of Medicine who says getting something into the joint that continuously reduces inflammation would be a major step forward.

Howard Rutman

The chief medical officer of Xalud Therapeutics, the company developing the plasmid-based gene therapy XT-150.

Jeon Seung-ho

The CEO of Kolon TissueGene, the company developing the cellular gene therapy TissueGene-C.

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What they’re saying

“Many people have had a shot of cortisone in their knee, except we're not injecting the steroid. We're injecting a virus that carries a therapeutic gene or a gene that will promote and direct the synthesis of a therapeutic protein.”

— Christopher Evans, Researcher, Mayo Clinic (Medscape)

“Trying to raise money for gene therapy was nonexistent for many years.”

— Daniel Grande, Biomedical Researcher, Feinstein Institutes for Medical Research (Medscape)

“The extra steroid actually increases the viral replication in the joint, which then usually means that more gene is inserted into the DNA and potentially more activity.”

— Nancy Lane, Rheumatologist and Researcher, University of California Davis School of Medicine (Medscape)

What’s next

Kolon TissueGene is expected to announce top-line data from two phase 3 trials of its cellular gene therapy TissueGene-C in July 2026.

The takeaway

While no gene therapy for knee osteoarthritis has been approved yet, the field is showing promise with several approaches in clinical trials. Experts believe a combination of therapies targeting multiple pathways involved in this complex joint disease may be needed to achieve the ultimate goal of reducing pain and restoring joint function with a single injection.