Safety, biodistribution, and exploratory clinical outcomes of a novel intra-articular IL-1Ra gene therapy (PCRX-201) in moderate-to-severe knee osteoarthritis: 2-year results of a phase 1 study.
A gene therapy for knee arthritis showed sustained pain and function improvements over two years with a strong safety profile in early trials.
PCRX-201 (high-capacity adenoviral vector expressing IL-1Ra under an inflammation-inducible promoter) demonstrated safety with very limited systemic biodistribution in a Phase 1 trial of 72 moderate-to-severe knee OA patients. Exploratory patient-reported outcomes showed sustained pain, stiffness, and function improvements at 2 years, supporting further investigation of this disease-modifying gene therapy for OA—a disease with no currently approved DMOAD.
What the study was
- Study design
- clinical_trial
- Population
- Adults aged 30-80 with symptomatic radiographic knee OA (WOMAC pain 5-9, KL grade 2-4)
- Sample size
- 72
- Category
- Gene Therapy / Musculoskeletal
- Maturity
- Exploratory
- Journal
- Ann Rheum Dis
Why it surfaced
Phase 1 gene therapy for OA with inflammation-inducible promoter enabling smart dosing; 2-year safety and sustained clinical benefit; very limited systemic distribution; no currently approved DMOAD (500M+ OA patients globally); Ann Rheum Dis; Pacira BioSciences.
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