Advancing Nucleic Acid Therapies for ALS: Delivery Innovations, Multiomic Insights, & Next-Generation Target Discovery

  • Learnings from full-cycle oligonucleotide development in ALS, spanning target validation, lead identification, and clinical candidate nomination
  • Delivery innovations and nucleic-acid–based strategies that may improve therapeutic reach and durability in neurodegenerative disease
  • How integrated data science, including bioinformatics, computational biology, and biomarker discovery, is shaping next-generation ALS programs
  • The role of multiomic and spatial biology platforms in enabling precision medicine approaches for ALS and other neurodegenerative disorders