Pacira's Gene Therapy Breakthrough Shows Long-Term Relief for Knee Osteoarthritis

April 29, 2025
Pacira's Gene Therapy Breakthrough Shows Long-Term Relief for Knee Osteoarthritis
  • On April 28, 2025, Pacira BioSciences announced promising long-term results from its gene therapy candidate, PCRX-201, for knee osteoarthritis.

  • The Phase I trial demonstrated sustained improvements in knee osteoarthritis symptoms over a two-year period.

  • In the trial, 72 participants aged 30 to 80 were divided into two groups: one received a single dose of PCRX-201, while the other was pre-treated with an intraarticular corticosteroid to enhance treatment effects.

  • Participants who were pre-treated with corticosteroids experienced greater reductions in pain and stiffness compared to the control group.

  • Both groups showed significant improvements in knee pain, function, and stiffness, evaluated using the WOMAC scores over the 104-week study period.

  • Notably, the most significant improvements were observed in subjects classified with K/L grade 2 knee osteoarthritis, with no serious adverse events reported.

  • PCRX-201 employs an inducible promoter that activates IL-1Ra expression in response to inflammation, ceasing once inflammation is controlled.

  • This therapy utilizes a high-capacity adenovirus gene therapy vector platform, administered directly into the knee joint to enhance IL-1Ra production and reduce chronic inflammation.

  • Following these positive Phase I results, Pacira is currently conducting dosing in the Phase II ASCEND study.

  • These findings strengthen Pacira's position in the competitive landscape of osteoarthritis treatments, which includes existing products like EXPAREL and ZILRETTA.

  • With a workforce of 789 employees, Pacira BioSciences focuses on non-opioid treatments for pain management and other debilitating conditions.

  • Pacira's stock price closed at $25.72, with analysts projecting an average target price of $36.00, indicating a potential upside of nearly 40%.

Summary based on 2 sources


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