What’s Next: Innovation In Neurology

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Delivering RNA-Targeted Medicines Deep Within the Nervous System

Preliminary research has demonstrated that investigational RNA-targeted medicines can penetrate tissues throughout the central nervous system (CNS) and are active across a wide range of cell types in the central nervous system.1-6 This offers the opportunity to develop RNA-targeted medicines for many types of neurologic diseases.1-4

RNA-targeted investigational medicines can be delivered to the spinal cord and deep regions within the brain, including the hippocampus, pons, and amygdala.1-4

graphs of cortex, cerebellu, hypothalamus and spinal cord
aEach of the symbols (ie, circle, square, diamond, triangle) represents data from a single mouse at the dose plotted. bRTM used in the study targets Malat1 RNA.
CNS, central nervous system; PBS, phosphate-buffered saline; RTID, RNA-targeted investigational drug; RTM, RNA-targeted medicine.

Research in animal models has shown investigational RNA-targeted medicines are active in neurons, microglia, astrocytes, and oligodendrocytes.1-4

In human patients, investigational RNA-targeted medicines have been shown to distribute within the CNS.7

graph of neurons, oligodendroncytes, astrocytes, microglia
aRTM used in the study targets Malat1 RNA.
CNS, cnetral nervous system; ED50 effective dose; mRNA, messenger RNA; PBS, phosphate-buffered saline; RTM, RNA-targeted medicine.

To see how this technology functions in the CNS, please watch the video below:

With 4 FDA-approved neurologic medicines and more than 10 investigational RNA-targeted medicines in mid- or late-stage development, Ionis is potentially transforming the trajectory of serious neurologic diseases.8-11

Learn more about Ionis’ approach to screening investigational RNA-targeted medicines and its pipeline candidates.


  1. Jafar-Nejad P, Powers B, Soriano A, et al. The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration. Nucleic Acids Res. 2021;49(2):657-673.
  2. Korobeynikov VA, Lyashchenko AK, Blanco-Redondo B, Jafar-Nejad P, Shneider NA. Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis. Nat Med. 2022;28(1):104-116.
  3. Mazur C, Powers B, Zasadny K, et al. Brain pharmacology of intrathecal antisense oligonucleotides revealed through multimodal imaging. JCI Insight. 2019;4(20):e129240.
  4. Mortberg MA, Gentile JE, Nadaf NM, et al. A single-cell map of antisense oligonucleotide activity in the brain. Nucleic Acids Res. 2023;51(14):7109-7124.
  5. Edwards AL, Collins JA, Junge C, et al. Exploratory tau biomarker results from a multiple ascending-dose study of BIIB080 in Alzheimer disease: a randomized clinical trial. JAMA Neurol. 2023;80(12):1344-1352.
  6. Finkel RS, Chiriboga CA, Vajsar J, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016;388(10063):3017-3026.
  7. Bennett CF, Krainer AR, Cleveland DW. antisense oligonucleotide therapies for neurodegenerative diseases. Annu Rev Neurosci. 2019;42:385-406.
  8. Ionis Pharmaceuticals. The Ionis antisense pipeline. Access March 13, 2024.
  9. Ionis Pharmaceuticals. Data on file.
  10. Ionis Pharmaceuticals. Ionis Innovation Day. October 4, 2023. Accessed February 5, 2024.
  11. Ionis Pharmaceuticals. Our medicines. Accessed February 5, 2024.

Contact Us

For questions or to request information, please contact us.

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Ionis continues to build upon its pioneering platform and foundational knowledge to develop medicines that can alter disease trajectory.

Learn more about the Ionis pipeline and candidates »

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We are committed to addressing the significant unmet needs across a spectrum of neurologic diseases.

Learn more about Ionis’ involvement in the community »

US-GEN-2400029 v1.0 03/2024