Director of Pathology Ionis Pharmaceuticals Carlsbad, California, United States
This presentation will focus on nucleic acid-based therapeutics such as antisense oligonucleotides (ASOs) and mRNA therapies that are used for the treatment of neurologic or neuromuscular diseases. Mechanisms of action, in-vitro and in-vivo characterization, and pathology considerations unique to the safety assessment of these modalities will be discussed.
