A Pipeline With the Potential to Impact Neurologic Diseases

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The neurology pipeline includes investigational RNA-targeted medicines for severe and debilitating neurologic diseases that affect a wide range of people.5,6

Investigational RNA-targeted medicines (RTMs) are designed to target genetic sequences associated with neurologic diseases.1,7

Disease pipeline chart

All agents listed above are investigational and have not been approved by the US Food and Drug Administration (FDA).1

aContent above subject to change pending updates to Ionis pipeline.
bThis investigational antisense medicine is in a Phase 1/2a study.2
cThe US Food and Drug Administration has granted both orphan drug designation and rare pediatric disease designation for its investigational drug ION582.3
dThis investigational antisense medicine is in a Phase 1/2 study. The primary purpose of the study is the evaluation of the medicine’s safety profile. It is listed here in Phase 2 because the medicine is being tested in patients and not healthy volunteers. This study may be categorized by partners or on regulatory sites, such as clinicaltrails.gov, as a Phase 1 study2
MECP2, methyl CpG binding protein-2.

Investigational RNA-Targeted Medicines in Clinical Trials

Ionis Neurology has several ongoing clinical trials applying investigational RNA-targeted medicines to a range of neurologic diseases.6

Current clinical trials are exploring investigational RNA-targeted medicines to target the genes associated with neurologic disease.4,6

dna chart
dsDNA, double-stranded DNA; mRNA, messenger RNA; RTM, RNA-targeted medicine.

If you are interested in learning more about the trial design, outcomes, and/or participating centers for one of the following diseases, please download the appropriate information sheet below.

If you are interested in more disease state education, please download the appropriate information sheet below:


  1. Crooke ST, Liang XH, Baker BF, Crooke RM. Antisense technology: a review. J Biol Chem. 2021;296:100416.
  2. Ionis Pharmaceuticals. Annual Report 2022. Accessed January 31, 2024.
  3. Partridge W, Xia S, Kwoh TJ, Bhanot S, Geary RS, Baker BF. Improvements in the tolerability profile of 2′-O-methoxyethyl chimeric antisense oligonucleotides in parallel with advances in design, screening, and other methods. Nucleic Acid Ther. 2021;31(6):417-426.
  4. Bennett CF, Kordasiewicz HB, Cleveland DW. Antisense drugs make sense for neurological diseases. Annu Rev Pharmacol Toxicol. 2021;61:831-852.
  5. Ionis Pharmaceuticals. Ionis Innovation Day 2023. Accessed February 5, 2024.
  6. Ionis Pharmaceuticals. The Ionis antisense pipeline. Accessed February 5, 2024.
  7. Quemener AM, Bachelot L, Forestier A, Donnou-Fournet E, Gilot D, Galibert MD. The powerful world of antisense oligonucleotides: from bench to bedside. Wiley Interdiscip Rev RNA. 2020;11(5):e1594.
  8. Data on file. Ionis Pharmaceuticals.
  9. Ionis reports second quarter financial results and recent business achievements. US Securities and Exchange Commission. August 9, 2022. Accessed February 5, 2024.
  10. Dhuri K, Bechtold C, Quijano E, et al. Antisense oligonucleotides: an emerging area in drug discovery and development. J Clin Med. 2020;9(6):2004.

Contact Us

For questions or to request information, please contact us.

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Ionis is built on a foundation of transformative technologies that target the underlying causes of neurologic diseases.

Learn more about innovation in neurology »

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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