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.  To date we have generated over 22 unique compounds developed to target knock-down of specific genes across a wide variety of therapeutics such as rare diseases, oncology, autoimmune disorders, metabolic conditions and diseases driven by a single point mutation.  We are currently completing the pre-clinical activities for our first undisclosed selected liver target related to a rare disease.  Our goal is to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) in the early part of 2018 with plans to then commence a clinical trial with a goal of delivering biological proof of concept also within 2018.

In addition to our own clinical aspirations, we also have a collaboration with Glaxo Smith Kline (GSK) to develop an undisclosed 3GA gene target for renal conditions.

  • Dermatomyositis

    Learn more about the debilitating effects of dermatomyositis.