Committed to serving patients in key areas of unmet medical need.

Rare diseases can afflict anyone, at any age. They can be acute or chronic. Many are debilitating, while others are unavoidably fatal, given current medical options. Approved therapies are available to treat hundreds of these conditions, but most have no curative or disease-modifying treatment options.

This is the case in dermatomyositis, a rare disease that is the lead clinical target for our IMO-8400 clinical development program. Our therapeutic approach to treating these diseases is to design compounds to block the over-activation of toll-like receptors (TLRs) and inhibit multiple cytokines without affecting their normal function. Our ultimate goal is to develop effective new medicines with improved long-term safety profiles and positive impact on disease remission.

We are also assessing third-generation antisense targets in rare diseases. We believe that, based on its validation in pre-clinical models, our third-generation antisense technology has the potential to overcome the remaining hurdles of current antisense technologies, including efficient delivery without a carrier, reduced immunotoxicity, and increased potency. The first two gene targets that have been selected to advance into clinical development from this platform are NLRP3 (NOD-like receptor family, pyrin domain containing protein 3) and DUX4 (Double Homeobox 4). Potential disease indications related to these two gene targets include, but are not limited to interstitial cystitis, uveitis, and facioscapulohumeral muscular dystrophy (FSHD), respectively.

  • Dermatomyositis

    Learn more about the debilitating effects of dermatomyositis.