IRAK-M gene therapy

IRAK-M gene therapy is an experimental adeno-associated virus (AAV)-based gene therapy designed for the treatment of age-related macular degeneration (AMD). Developed by researchers at the University of Bristol, the therapy utilizes an AAV2 vector to deliver the human *IRAK3* gene, which encodes the interleukin-1 receptor-associated kinase M (IRAK-M) protein. IRAK-M is an endogenous inhibitory protein that naturally declines in the retinal pigment epithelium (RPE) with age. By restoring IRAK-M levels, the therapy negatively regulates the Myddosome signaling complex, thereby suppressing chronic inflammation, oxidative stress, and cellular senescence. Preclinical data published in 2024 demonstrated that subretinal administration of this therapy protects retinal cells and preserves mitochondrial function, offering a potential disease-modifying approach for AMD.

Other names

AAV2-IRAK3AAV-2-IRAK3AAV 2-IRAK3AAV-IRAK-MIRAK3 gene therapyIRAK-3 gene therapyIRAK 3 gene therapy