Clinical Trials of
RNA-Targeted Therapeutics
Clinical Trials of
RNA-Targeted Therapeutics
We are transforming genomic insights into RNA‑targeted innovation.
Our ongoing clinical trials are exploring investigational RNA-targeted therapeutics to address the genetic cause of disease in a range of neurologic diseases.1,2
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.
References
- Ionis Pharmaceuticals. Pipeline. Accessed March 3, 2025. https://ionis.com/science-and-innovation/pipeline/
- Bennett CF, Kordasiewicz HM, Cleveland DW. Antisense drugs make sense for neurological diseases. Annu Rev Pharmacol Toxicol. 2021;61:831-852.
