ONYX-101

ONYX-101 is a kidney-targeted dual-vector adeno-associated virus (AAV) gene therapy candidate developed by Nionyx Bio for the treatment of X-linked Alport syndrome. The therapy utilizes proprietary NYX AAV capsids engineered for efficient renal delivery and specific targeting of glomerular podocytes. Because the *COL4A5* gene exceeds the standard AAV packaging capacity, ONYX-101 employs a dual-vector strategy with an RNA-trans splicing approach to reconstitute the full-length COL4A5 protein. By restoring functional type IV collagen assembly in the glomerular basement membrane, ONYX-101 aims to address the underlying genetic defect of Alport syndrome and prevent progression to end-stage kidney disease.