Corporate Presentation
Differentiated Platform Enables Rapid Progress in Advancing
Neuroscience Therapeutics
Differentiated Nucleic
Acid Therapeutics
Rapidly Advancing
Neuroscience Pipeline
Outstanding
R&D Capabilities
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Enable dramatically higher cellular uptake of oligonucleotides in vitro
Achieve desired PD effects in vivo at substantially lower oligonucleotide doses
Broad CNS distribution and longer persistence in the CNS compared to linear
oligonucleotides (rodent & non-human primates)
SNA platform clinically de-risked
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XCUR-FXN for Friedreich's Ataxia entered IND-enabling stage in 4Q'20
Deep pipeline of SNA therapeutics advancing for neurodegenerative disorders
• Hired CMO Doug Feltner (2020) from AveXis to lead clinical development
~60 employees, ~50 in R&D + high-throughput oligonucleotide discovery platform
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