C9orf72 ALS Program

About Iris Medicine

Iris Medicine is a private, preclinical-stage biotech founded in 2020 and based in Cambridge, USA, focused on developing allele-selective RNA therapies for repeat expansion disorders. Its core technology, small binding RNA (sbRNA), leverages cooperative binding to selectively inhibit mutant transcripts while sparing healthy ones, using a non-viral delivery system. The company's initial pipeline targets Huntington’s Disease, C9orf72 ALS, and Spinocerebellar Ataxias, aiming to address a large family of diseases with high unmet need. Backed by experienced life science investors and advisors, including pioneers from the RNAi field, Iris is positioned to advance a potentially transformative 'pipeline-in-a-product' platform.

Therapeutic Areas