Our Science
Our Science
Ionis is a leader in developing transformational RNA-targeted medicines for hard-to-treat neurologic diseases.1,2
For decades, Ionis has worked to advance RNA-targeted technology to create medicines for rare and prevalent neurologic diseases.3-6
Ionis’ investigational therapeutics are designed to interact precisely with target RNA, resulting in investigational RNA-targeted medicines that may target the underlying cause of neurologic disease without altering patients’ genes.7,8
Ionis Is Continually Refining and Optimizing Its RNA-Targeted Medicine Platform to Positively Impact the Lives of Patients With Neurologic Diseases, Creating New Standards of Care9
Bioinformatics
In vitro screening
in human cells
Human
neuron
Rodent
Rodent with
human gene
Non-human
primate
Candidate for human study
MD, multiple dose; SD, single dose.
Ionis has used knowledge from the genomics revolution to develop FDA-approved medicines for the treatment of spinal muscular atrophy, amyotrophic lateral sclerosis, and polyneuropathy in patients with hereditary transthyretin amyloidosis.5,10-15
References
- Ionis Pharmaceuticals. Ionis’ antisense technology. Accessed August 19, 2024. https://www.ionis.com/ionis-technology/technology/
- Crooke ST. RNA-directed therapeutics at Ionis. Nature. 2019;574(7778):1-3.
- Ionis Pharmaceuticals. The creation of RNA targeting technology. December 2019. Accessed August 19, 2024. https://ir.ionispharma.com/static-files/1cada39c-0fa4-4e90-afdf-e85fa258e03b/
- Crooke ST, Baker BF, Crooke RM, Liang XH. Antisense technology: an overview and prospectus. Nat Rev Drug Discov. 2021;20(6):427-453.
- Hua Y, Vickers TA, Baker BF, et al. Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon. PLoS Biol. 2007;5(4):e73.
- Bennett CF, Krainer AR, Cleveland DW. Antisense oligonucleotide therapies for neurodegenerative diseases. Annu Rev Neurosci. 2019;42:385-406.
- 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.
- Crooke ST, Liang XH, Baker BF, Crooke RM. Antisense technology: a review. J Biol Chem. 2021;296:100416.
- Ionis Pharmaceuticals. The Ionis antisense pipeline. Accessed August 19, 2024. https://www.ionis.com/ionis-technology/antisense-pipeline/
- AstraZeneca. Press release. December 7, 2021. Accessed August 19, 2024. https://www.astrazeneca.com/media-centre/press-releases/2021/astrazeneca-ionis-to-collaborate-on-eplontersen.html#/
- FDA approves first drug for spinal muscular atrophy. US Food and Drug Administration. December 23, 2016. Accessed August 19, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-spinal-muscular-atrophy/
- Hua Y, Vickers TA, Okunola HL, Bennett CF, Krainer AR. Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice. Am J Hum Genet. 2008;82(4):834-848.
- Qiu J, Wu L, Qu R, et al. History of development of the life-saving drug "Nusinersen" in spinal muscular atrophy. Front Cell Neurosci. 2022;16:942976.
- Miller TM, Cudkowicz ME, Genge A, et al. Trial of antisense oligonucleotide tofersen for SOD1 ALS. N Engl J Med. 2022;387(12):1099-1110.
- Wainua. Package insert. AstraZeneca. Updated September 2024. Accessed October 21, 2024. https://www.azpicentral.com/pi.html?product=wainua/
