Harnessing Gene Therapy for Osteoarthritis


Flexion Therapeutics believes that local administration of a gene therapy may not only improve pain management but potentially slow disease progression.

Despite the more than 15 million Americans with osteoarthritis, treatment of the degenerative musculoskeletal disorder had been at a standstill, with short-term pain management with steroid injections considered standard care. However, in 2017, a new therapeutic approach was approved by the FDA that promised more sustained pain relief over time.1

That therapy, triamcinolone acetonide extended-release injectable suspension, marketed as Zilretta by Flexion Therapeutics, resulted in significant reductions in knee pain for 12 weeks in patients with osteoarthritis, with some patients reporting pain relief through 16 weeks.

Although Zilretta represents an improvement over current standards of care for OA, Flexion has continued to push to address the burgeoning unmet need in this population, now with a gene therapy approach.

The company is currently investigating FX201, an intra-articular, helper-dependent adenoviral gene therapy that responds to inflammation by producing interleukin-1 receptor antagonist. Data from its first in-human trial presented in May 2021 at the American Society for Gene and Cell Therapy virtual annual meeting showed good safety and tolerability, with substantial improvements in WOMAC-A pain intensity observed in 3 patients at week 8 and 2 patients at weeks 12 and 24.2

To learn more about this endeavor, GeneTherapyLive spoke with Flexion’s chief executive officer and co-founder, Michael D. Clayman, MD.

1. Flexion Therapeutics announces FDA approval of Zilretta (triamcinolone acetonide extended-release injectable suspension) for osteoarthritis (OA) knee pain. News release. Flexion Therapeutics. October 6, 2017. https://ir.flexiontherapeutics.com/news-releases/news-release-details/flexion-therapeutics-announces-fda-approval-zilrettatm
2. Kelley S, Kivitz A, Senter B, et al. Interim data from the first-in-human phase 1 trial of FX201, an intra-articular, helper-dependent adenoviral gene therapy for osteoarthritis - safety, tolerability, biodistribution, and preliminary evaluation of clinical activity in 5 patients. Presented at: ASGCT 2021 Annual Meeting.
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