There are 7,000 rare diseases but less than 400 approved treatments. It's time for a different approach.
Marina Biotech's approach to the development of compounds to treat rare diseases is to focus on the rare disease itself rather than any one therapeutic approach. By utilizing its diverse nucleic acid drug discovery engine, Marina can rapidly move multiple, novel oligonucleotide compounds into clinical development for a specific rare disorder thus capitalizing on the individual therapeutic advantages of specific RNA-based mechanisms of action and increasing the overall likelihood of clinical trial success, regulatory approval and, ultimately, getting effective treatments to patients with rare disorders.
Our chemistry and delivery technologies provides us with one of the most unique drug discovery engines for the development of nucleic acid-based therapeutics for rare and orphan diseases. In particular, our comprehensive drug delivery technologies are unparalleled in the field and utilize multiple separate and distinct delivery modalities. Two of these technologies are currently in human clinical trials supporting RNAi, microRNA replacement and DNA decoy approaches to the treatment of human diseases. One of these delivery technologies, SMARTICLES®, is the only technology delivering both single-stranded and double-stranded nucleic acid payload.
Familial Adenomatous Polyposis (FAP) is a rare hereditary disease with no existing therapeutic treatment option. Our Phase 1 clinical trial is evaluating our lead therapeutic candidate, CEQ508,which is the only orally administered RNAi-based compound in clinical development.