Immune Cells, Cardiac Tissues and Other Cell Types

We also intend to pursue the development of AOCs in cell types in addition to muscle to continue to add to our expanding pipeline. For example, in the field of immunology, we believe AOCs have the potential to target a broader set of cell types and diseases than traditional oligonucleotide therapies. We believe oligonucleotide therapies have the potential to address the challenges of immune responses at the RNA level; however, the ability to modulate immune responses has been hampered by delivery of these agents to immune cells. By identifying and optimizing antibodies for specific immune cell types, our goal is to leverage our AOC platform to develop product candidates that can deliver siRNAs to disease-driving subsets of immune cells. We are collaborating with Eli Lilly and Company initially on 6 mRNA targets in immunology and other select indications outside of muscle for the delivery, development and commercialization of AOCs.

Beyond immune cells, we are investing in our AOC platform to explore the full potential of our AOCs in cardiomyopathies, and diseases of cells and tissues inaccessible to other oligonucleotide technologies. We also have a collaboration with MyoKardia, a wholly-owned subsidiary of Bristol Myers Squibb, that will help us expand our therapeutic activities to include cardiac-specific indications.

Through internal discovery efforts and partnerships, our goal is to discover, develop and commercialize novel AOC therapeutics that overcome current barriers to the delivery of oligonucleotides and unlock their potential to treat a wide range of serious diseases currently lacking adequate treatment options.