Our Clinical Studies
Receiving a devastating diagnosis can be overwhelming, especially when treatment options are limited or not available. It’s important to know that you are not alone.
Discover Trial Opportunitiesfeatured Studies now enrolling
Angelman Syndrome
The purpose of this study is to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.
Studies Now Enrolling
Through scientific research and educational resources, we are committed to pioneering new markets and changing the standard of care. With a novel RNA-targeted drug discovery platform called “antisense,” we are tackling a range of diseases across a number of therapeutic areas.
Amyotrophic Lateral Sclerosis
The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).
PHASE 3
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
The purpose of the study is to prospectively assess longitudinal changes in biomarkers (MECP2, potential biomarkers of target engagement and disease activity) in cerebrospinal fluid (CSF) and blood; characterize longitudinal changes in performance on clinical scales (clinician-reported measures of neurodevelopment and functioning) and caregiver-reported outcome assessments (communication, gastrointestinal, social-emotional-adaptive behavioral measures); evaluate longitudinal changes in caregiver-reported health-related quality-of-life measures; and assess the frequency, type, and severity of seizures over time.
Observational
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
PHASE 1/2
Pelizaeus-Merzbacher Disease
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
PHASE 1
Alzheimer’s Disease
The primary purpose of this study is to evaluate the safety and tolerability of ION269 in adults with Down syndrome with evidence of brain amyloid positivity.
PHASE 1
Pelizaeus-Merzbacher Disease
The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to Pelizaeus-Merzbacher disease (PMD) and longitudinal changes in performance on clinical, participant, and caregiver-reported outcome assessments to inform the development of therapies for PMD.
Observational
Angelman Syndrome
The purpose of this study is to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.
PHASE 3
Alexander Disease
The purpose of this study is to evaluate the safety and efficacy of zilganersen (ION373) in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD.
PHASE 3
Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
PHASE 3
Severe Hypertriglyceridemia
The purpose of this study is to evaluate the safety and tolerability of olezarsen in participants with SHTG.
PHASE 3
Hereditary Angioedema
The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).
PHASE 3
