A Unique Approach to Rare Diseases
Marina Biotech is a leading nucleic acid-based drug discovery and development company focused on rare diseases. Our drug discovery platform, the broadest in the sector, allows us to develop compounds which best counter the underlying pathophysiology associated with a specific rare disease. Using this platform, we are developing proprietary single and double-stranded nucleic acid therapeutics including siRNAs, microRNA mimics, antagomirs, and antisense compounds intended to trigger various RNA-based mechanisms of action including RNA interference, microRNA replacement therapy, microRNA inhibition and mRNA translational inhibition, respectively.
Currently, we are advancing CEQ508, for which we have received FDA orphan drug designation, in a Phase 1 clinical program for patients with Familial Adenomatous Polyposis. In addition, we are expanding our rare disease focus to include myotonic dystrophy and Duchenne’s muscular mystrophy and believe that our platform is a unique and potentially powerful approach that is aptly positioned to treat the multiple anatomical systems - muscle, heart and CNS, associated with these diseases.