Transforming the Trajectory of Serious Neurologic Diseases Through RNA-Targeted Technology
Transforming the Trajectory of Serious Neurologic Diseases Through RNA-Targeted Technology
Ionis is committed to discovering, developing, and delivering potentially life-changing therapeutics for people living with severe, progressive, and fatal neurologic diseases.1
Ionis Has Decades of Experience Developing Therapies for Challenging Genetic Conditions2-5
Ionis Has Decades of Experience Developing Therapies for Challenging Genetic Conditions2-5
Through our years of preclinical studies, we have developed a rigorous process that optimizes the identification and development of RNA-targeted therapeutics with reduced toxicity and a potentially favorable safety and tolerability profile for patients with neurologic diseases.5-10
Transforming the Management of Neurologic Diseases
Transforming the Management of Neurologic Diseases
Ionis is leveraging its RNA-targeted technology platform to create transformative therapies that target the genes associated with the underlying disease pathology to potentially improve patients’ lives.2,11-13
Identifying the Neurologic Therapeutics of Tomorrow
Identifying the Neurologic Therapeutics of Tomorrow
Ionis is continually advancing the design, screening, and development of its investigational RNA-targeted therapeutics to optimize the selection of investigational therapeutics for neurologic diseases.5,6,9,10
Ionis builds upon foundational knowledge to improve and advance therapeutics using new approaches to target proteins that result from gene expression that are associated with the underlying disease pathology.6,14
With a history of major breakthroughs in RNA-targeted therapeutics, Ionis’ robust pipeline is filled with potential.2,6,10,15-18
References
- Ionis Pharmaceuticals. Ionis Innovation Day. October 4, 2023. Accessed March 3, 2025. https://ir.ionis.com/static-files/8b71dc65-dad9-4368-9014-604c5b203ca1/
- Crooke ST, Baker BF, Crooke RM, Liang XH. Antisense technology: an overview and prospectus. Nat Rev Drug Discov. 2021;20(6):427-453.
- 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.
- 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.
- 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.
- Crooke ST, Liang XH, Baker BF, Crooke RM. Antisense technology: a review. J Biol Chem. 2021;296:100416.
- Bajan S, Hutvagner G. RNA-based therapeutics: from antisense oligonucleotides to miRNAs. Cells. 2020;9(1):137.
- Shen W, De Hoyos CL, Migawa MT, et al. Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index. Nat Biotechnol. 2019;37(6):640-650.
- Crooke ST, Witztum JL, Bennett CF, Baker BF. RNA-targeted therapeutics [published correction appears in Cell Metab. 2019;29(2):501. doi: 10.1016/j.cmet.2019.01.001]. Cell Metab. 2018;27(4):714-739.
- Ionis Pharmaceuticals. The creation of RNA targeting technology. December 2019. Accessed March 3, 2025. https://ir.ionispharma.com/static-files/1cada39c-0fa4-4e90-afdf-e85fa258e03b/
- Ionis Pharmaceuticals. Data on file.
- Ionis Pharmaceuticals. Pipeline. Accessed March 3, 2025. https://www.ionis.com/science-and-innovation/pipeline/
- Mummery CJ, Börjesson-Hanson A, Blackburn DJ, et al. Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer's disease: a phase 1b, randomized, placebo-controlled trial [published correction appears in Nat Med. 2024;30(1):304]. Nat Med. 2023;29(6):1437-1447.
- 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.
- Bennett CF, Kordasiewicz HB, Cleveland DW. Antisense drugs make sense for neurological diseases. Annu Rev Pharmacol Toxicol. 2021;61:831-852.
- Qalsody. Package insert. Biogen MA Inc. Updated April 2023. Accessed March 3, 2025. https://www.biogencdn.com/us/pdfs/qalsody-prescribing-information.pdf
- Wainua. Package insert. AstraZeneca. Updated September 2024. Accessed March 3, 2025. https://www.azpicentral.com/pi.html?product=wainua
- Ionis Pharmaceuticals. Medicines. Accessed March 3, 2025. https://www.ionis.com/medicines/
