IRAK-M Gene Therapy

While most AMD treatments address only single pathways in this multifactorial disease, our IRAK-M gene therapy targets a central driver of AMD disease biology. Cirrus scientists identified IRAK-M as a central regulator of immune homeostasis in the eye. As people age, IRAK-M levels fall, leaving retinal cells exposed to chronic inflammation and metabolic stress that drive dry AMD. Carriers of rare variants in IRAK3, the gene encoding IRAK-M, have an increased propensity to develop AMD, further underscoring its importance in AMD risk.

Our AAV-based therapy delivers genetic instructions directly to the eye to restore IRAK-M production toward youthful levels. By rebalancing immune signaling upstream, this approach is designed to reduce harmful inflammation, normalize cellular metabolism, and protect both retinal pigment epithelium and photoreceptors from ongoing injury. Instead of targeting a single downstream pathway, we aim to stabilize multiple disease-driving pathophysiological pathways at once.

The anticipated outcome is a one-time, durable intervention for patients with earlier stages of GA and dry AMD. Our goal is to slow or halt disease progression and, in some patients, improve visual function by enhancing the cells that support sharp central vision. If successful, IRAK-M gene therapy could shift dry AMD from a chronic, deteriorating condition to a controllable disease with a markedly improved trajectory.