Evolving landscape of small-molecule immunotherapeutics for uveitis: a scoping review

In this scoping review, we highlight advances in small-molecule immunotherapeutics for non-infectious uveitis (NIU), a sight-threatening inflammatory disease. Unlike traditional corticosteroids and biologics, emerging small molecules promise enhanced ocular penetration and precise intracellular modulation. Analysing randomised trials, observational studies, and animal models, we identified 16 drug classes and 31 novel compounds. Notably, new-generation calcineurin inhibitors, Janus kinase (JAK) inhibitors, mammalian target of rapamycin (mTOR) inhibitors, corticosteroids, and reactive aldehyde species (RASP) inhibitors demonstrated encouraging clinical efficacy. These agents could redefine treatment paradigms by minimising systemic toxicity and overcoming current therapeutic limitations. However, larger studies are needed to establish their long-term safety and effectiveness across diverse NIU patient populations.