Accro Bioscience Secures NMPA Pilot Status for Blau Syndrome Therapy

This regulatory milestone follows a similar endorsement from the U.S. Food and Drug Administration, which granted AC-101 Rare Pediatric Disease designation earlier this June. The drug, a selective RIPK2 inhibitor developed by the Suzhou-based Accro Bioscience, targets the NOD signaling pathway to mitigate the systemic inflammation characteristic of the disease.

Blau syndrome, a hereditary condition triggered by NOD2 gene variants, typically manifests in early childhood through a triad of granulomatous dermatitis, arthritis, and uveitis. Without effective intervention, the disease often progresses to severe joint deformity and permanent vision loss. Accro Bioscience intends to showcase safety and efficacy data from its ongoing clinical trials at the upcoming UEG Week 2026. The company’s broader pipeline focuses on leveraging regulatory cell death mechanisms to treat autoimmune disorders, including moderate-to-severe ulcerative colitis.