Corporate Presentation

Extensive Proof of Concept in Neurological Disorders Biogen IONIS Spinraza Ⓡ (nusinersen) Commercially available linear oligo therapeutic to treat spinal muscular atrophy (SMA) Survival data presented at 2018 Annual Cure SMA Conference 100 80 60 40 20 0 0 PROOF OF CONCEPT (MICE) 20 Control 40 60 80 Days Survival 100 120 exicure SNA 2x levels of healthy, full-length SMN2 mRNA and protein in SMA patient fibroblasts 4x longer survival vs. nusinersen and mitigated nusinersen toxicity exicure | 13

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