Insights and Medicines (original) (raw)
Ionis Neurology: Pipeline
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Ionis continues to build upon its pioneering platform and foundational knowledge to develop medicines that can alter disease trajectory.1-4
Transforming Genomic Insights Into RNA-Targeted Innovation
Ionis’ RNA-targeted technology translates genomics insights into potential transformative medicines.5
Once a causative gene is identified, researchers can use this knowledge to develop antisense oligonucleotides (ASOs) that target the associated mRNA and modulate protein production.4,6,7 Ionis has used knowledge from the genomics revolution to develop FDA-approved medicines for the treatment of spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), and polyneuropathy in patients with hereditary transthyretin (hATTR) amyloidosis.7-13
Case Study: SMA
Prior to the 1990s, the fundamental cause of SMA was unknown.7,14 Research throughout the 1990s identified the cause of SMA—the deletion of survival motor neuron gene 1 (SMN1)—and a potential therapeutic target: alternative splicing to include exon 7 of SMN2.7,9
ASOs were screened for the optimal target along exon 7 of SMN2, marking a shift in drug discovery.5,7-9,15
Ionis Has Decades of Experience Developing Therapies
for Hard-to-Treat Genetic Conditions3,5,7,9,15
ASO, antisense oligonucleotide; FDA, US Food and Drug Administration; RTM, RNA-targeted medicine; SMA, spinal muscular atrophy.
Identifying the Neurologic Medicines of Tomorrow
Years of preclinical studies have yielded a rigorous process to optimize the identification and development of RNA-targeted medicines with reduced toxicity and a potentially favorable safety and tolerability profile for patients with neurologic diseases.1,3,16-19
Ionis is continually advancing the design, screening rigor, and development of its RNA-targeted medicines to optimize the selection of investigational medicines.1,3,18
Transforming Emerging Insights Into Investigational Medicines
Ionis Is Continually Advancing the Design, Screening, and Development of Its Investigational RNA-Targeted Medicines for Neurologic Diseases1,3,18,19
MD, multiple dose; SD, single dose.
Click on the figure above to learn more about the rigorous screening process that allows the identification of an investigational RNA-targeted medicine from thousands of oligonucleotides.18,19
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 care.24