Our Pipeline
Our Pipeline
Our goal is to discover and develop potentially life-changing therapeutics across a range of neurologic diseases.1
The Ionis Neurology pipeline includes investigational RNA-targeted therapeutics for severe and debilitating neurologic diseases that affect a wide range of patients with unmet needs.1,2
Our RNA-targeted drug discovery and development platform provides a fast, efficient path to identify first-in-class and/or best-in-class therapeutics.2-7
With 3 FDA-approved neurologic therapeutics and more than 10 investigational RNA-targeted therapeutics in mid- or late-stage development, Ionis is potentially transforming the trajectory of serious neurologic diseases.1,2,8
Ionis’ Pipeline Includes Early- and Late-Stage Investigational Therapeutics Designed to Target Genetic Sequences Associated With a Range of Rare and Prevalent Neurologic Conditions1,9-21,a,b
ION582 (Angelman syndrome)
ION582c
(Angelman syndrome)
ION582 (Angelman syndrome)
ION582c
(Angelman syndrome)
ION582a is an investigational RNA-targeted therapeutic (RTT) designed to inhibit the expression of the UBE3A antisense transcript (UBE3A-ATS) and increase production of the UBE3A protein as a potential therapy for Angelman syndrome (AS).1
This investigational RTT is being assessed in REVEAL, a Phase 3, randomized, double-blind, placebo-controlled, global, multicenter, three-part study of ION582 to evaluate the safety and efficacy of ION582 administered intrathecally in patients with AS. The study will include 2 cohorts with mutations and deletions in the UBE3A gene. Cohort 1 will include pediatric participants aged 2-17. Cohort 2 will include adult participants aged 18-50. Participants will be randomized to receive ION582 40 mg, ION582 80 mg, or placebo dosed every 12 weeks during the 60-week double-blind period, 100-week long-term extension (LTE) period, and 32-week post-treatment follow-up period. Participants from both cohorts completing the placebo-controlled treatment period will be eligible to transition into the LTE Treatment Period wherein all trial participants will receive ION582. The study plans to enroll approximately 210 participants.2
Recruitment status2:
Study start 2:
Estimated study completion 2:
UBE3A, ubiquitin protein ligase E3A protein; UBE3A, ubiquitin protein ligase E3A gene.
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://www.ionis.com/science-and-innovation/pipeline/ 2. ClinicalTrials.gov identifier: NCT06914609. Accessed July 15, 2025. https://www.clinicaltrials.gov/study/NCT06914609/ 3. First patient dosed in pivotal Phase 3 REVEAL clinical study of ION582 in Angelman Syndrome. June 11, 2025. Accessed July 15, 2025. https://ir.ionis.com/node/32691/pdfs
| Investigational RNA-Targeted Therapeutic | Disease State | Gene/Target | Phase 1 Phase 2 Phase 3 | |
|---|---|---|---|---|
| Ionis-owned | Zilganersen |
Alexander disease | GFAP | |
ION582c |
Angelman syndrome | UBE3A-ATS | ||
ION717d |
Prion disease | PRNP | ||
ION440 |
MECP2 duplication syndrome |
MECP2 | ||
ION356 |
Pelizaeus-Merzbacher disease |
PLP1 | ||
ION859e |
Parkinson’s disease | LRRK2 | ||
ION464e |
Multiple system atrophy and |
SNCA | ||
ION269d |
Alzheimer's disease |
APP | ||
| Investigational RNA-Targeted Therapeutic | Disease State | Gene/Target | Phase 1 Phase 2 Phase 3 | |
|---|---|---|---|---|
| Ionis-Biogen | Tofersen |
Superoxide dismutase 1 |
SOD1 | |
IONIS-MAPTRxe |
Alzheimer’s disease | TAU | ||
Salanersen |
Spinal muscular atrophy | SMN2 | ||
| Investigational RNA-Targeted Therapeutic | Disease State | Gene/Target | Phase 1 Phase 2 Phase 3 | |
|---|---|---|---|---|
| Ionis-Otsuka | Ulefnersen |
Fused in sarcoma amyotrophic |
FUS | |
| Investigational RNA-Targeted Therapeutic | Disease State | Gene/Target | Phase 1 Phase 2 Phase 3 | |
|---|---|---|---|---|
| Ionis-Roche | Tominersen |
Huntington’s disease | HTT | |
aContent in the table subject to change pending updates to Ionis pipeline. bSafety and efficacy have not been evaluated by any regulatory authorities for the indications described. cThe US Food and Drug Administration has granted both orphan drug designation and rare pediatric disease designation for its investigational drug ION582.15 dThis investigational antisense therapeutic is in a Phase 1/2a study.1 eThis investigational antisense therapeutic is in a Phase 1 study. The primary purpose of the study is the evaluation of the therapeutic’s safety profile. It is listed here as Phase 2 because the therapeutic is being tested in patients and not healthy volunteers. This study may be categorized by partners or on regulatory sites, such as ClinicalTrials.gov, as a Phase 1 study.1
FUS, fused in sarcoma protein gene; GFAP, glial fibrillary acidic protein gene; HTT, huntingtin gene; LRRK2, leucine-rich-repeat kinase 2 gene; MECP2, methyl-CpG-binding protein 2 gene (human); PLP1, proteolipid protein 1 gene; PRNP, prion protein gene; SCNA, synuclein alpha gene; SMN2, survival of motor neuron 2 gene; SOD1, superoxide dismutase 1 gene; TAU, tau protein gene; UBE3A-ATS, ubiquitin protein ligase E3A antisense transcript.
Zilganersen (Alexander disease)
Zilganersen
(Alexander disease)
Zilganersen,a clinically known as ION373, is an investigational RNA-targeted therapeutic (RTT) designed to inhibit the production of glial fibrillary acidic protein (GFAP). Zilganersen is being developed as a potential therapy for Alexander disease (AxD).1
This investigational RTT is being assessed in a Phase 1-3, double-blind, placebo-controlled, multiple ascending-dose study evaluating the safety and efficacy of zilganersen in improving or stabilizing gross motor function across the affected domains in 54 patients with AxD. Patients will be randomized in a 2:1 ratio to receive zilganersen or matching placebo for a 60-week double-blind treatment period; then all patients will receive zilganersen for a 60-week open-label treatment period, followed by a 120-week open-label, long-term extension period, and a 28-week post-treatment follow-up period.2
Some study sites will include an optional open-label sub-study in patients <2 years of age.2
Study start 2:
Estimated study completion 2:
aThe US Food and Drug Administration has granted both orphan drug designation and rare pediatric disease designation for its investigational drug zilganersen.3
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://ionis.com/science-and-innovation/pipeline/ 2. ClinicalTrials.gov identifier: NCT04849741. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT04849741/ 3. Zilganersen granted U.S. FDA Fast Track designation for people living with Alexander disease. October 1, 2024. Accessed August 21, 2025. https://ir.ionis.com/node/31726/pdf
ION582 (Angelman syndrome)
ION582c
(Angelman syndrome)
ION582a is an investigational RNA-targeted therapeutic (RTT) designed to inhibit the expression of the UBE3A antisense transcript (UBE3A-ATS) and increase production of the UBE3A protein as a potential therapy for Angelman syndrome (AS).1
This investigational RTT is being assessed in REVEAL, a Phase 3, randomized, double-blind, placebo-controlled, global, multicenter, three-part study of ION582 to evaluate the safety and efficacy of ION582 administered intrathecally in patients with AS. The study will include 2 cohorts with mutations and deletions in the UBE3A gene. Cohort 1 will include pediatric participants aged 2-17. Cohort 2 will include adult participants aged 18-50. Participants will be randomized to receive ION582 40 mg, ION582 80 mg, or placebo dosed every 12 weeks during the 60-week double-blind period, 100-week long-term extension (LTE) period, and 32-week post-treatment follow-up period. Participants from both cohorts completing the placebo-controlled treatment period will be eligible to transition into the LTE Treatment Period wherein all trial participants will receive ION582. The study plans to enroll approximately 210 participants.2
Study start 2:
Estimated study completion 2:
UBE3A, ubiquitin protein ligase E3A protein; UBE3A, ubiquitin protein ligase E3A gene.
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://www.ionis.com/science-and-innovation/pipeline/ 2. ClinicalTrials.gov identifier: NCT06914609. Accessed July 15, 2025. https://www.clinicaltrials.gov/study/NCT06914609/ 3. First patient dosed in pivotal Phase 3 REVEAL clinical study of ION582 in Angelman Syndrome. June 11, 2025. Accessed July 15, 2025. https://ir.ionis.com/node/32691/pdfs
ION717 (Prion disease)
ION717d
(Prion disease)
ION717 is an investigational RNA-targeted therapeutic (RTT) designed to inhibit the production of prion protein as a potential therapy for prion disease (PrD).1
This investigational RTT is being studied in PrProfile, a Phase 1/2a, first-in-human, randomized, multicenter study evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of intrathecally administered ION717 in approximately 56 patients with PrD. The study consists of a screening period of up to 6 weeks, a 30-week double-blind treatment period, a 70-week open-label extension period, and a 32-week post-treatment period.2
Study start 2:
Estimated study completion 2:
ION440 (MECP2 duplication syndrome)
ION440
(MECP2 duplication syndrome)
ION440 is an investigational RNA-targeted therapeutic (RTT) designed as a potential therapy for MECP2 duplication syndrome (MDS).1
This investigational RTT is being studied in ATTUNE, a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of intrathecally administered ION440 in pediatric and adult patients with MDS. This is a multicenter, two-part study with multiple dosing cohorts being evaluated in approximately 48 patients.2
During part 1, patients will be randomized in a 3:1 ratio to receive ION440 or sham. Patients who complete part 1 are eligible to enter part 2, an open-label, long-term extension study, where patients will receive ION440 for up to approximately 156 weeks.2
Study start 2:
Estimated study completion 2:
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://ionis.com/science-and-innovation/pipeline/ 2. ClinicalTrials.gov identifier: NCT06430385. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT06430385/
ION356 (Pelizaeus-Merzbacher disease)
ION356
(Pelizaeus-Merzbacher disease)
ION356 is an investigational RNA-targeted therapeutic (RTT) designed to decrease the production of proteolipid protein 1 (PLP1) as a potential therapy for males diagnosed with Pelizaeus-Merzbacher disease (PMD).1
This investigational RTT is being studied in ORBIT, a Phase 2a study to evaluate the safety, pharmacokinetics, and pharmacodynamics of intrathecally administered ION356 in male patients with PMD caused by duplication of PLP1. This is a multicenter, multiple-ascending-dose (MAD), multipart study enrolling approximately 24 pediatric patients.1,2
The study will have two parts: a 48-week MAD part, followed by an open-label, long-term extension (LTE) part of 109 weeks. During the MAD portion of the study, eligible patients will receive doses of ION356 and, upon completion, will transition to the open-label LTE to receive doses of ION356.2
Study start 2:
Estimated study completion 2:
aThis investigational antisense oligonucleotide therapeutic is in a Phase 1b study. It is listed here as Phase 2 because the therapeutic is being tested in patients and not healthy volunteers.1
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://ionis.com/science-and-innovation/pipeline/ 2. ClinicalTrials.gov identifier: NCT06150716. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT06150716
ION859 (Parkinson’s disease)
ION859e
(Parkinson’s disease)
ION859, clinically known as BIIB094, is an investigational RNA-targeted therapeutic (RTT) designed to inhibit the expression of the leucine-rich repeat kinase 2 (LRRK2) protein as a potential therapy for Parkinson’s disease (PD).1,2
This investigational RTT was studied in REASON, a Phase 2a study evaluating the safety, tolerability, and pharmacokinetics of single and multiple doses of ION859 administered via intrathecal injection in patients with PD. This was a randomized, blinded, placebo-controlled, multiple ascending-dose study in patients with PD with verified presence or absence of variation in the leucine-rich repeat kinase 2 gene (LRRK2), as well as to patients without any verified PD-related genetic variant.1,2
Study start 2:
Estimated study completion 2:
aThis investigational antisense therapeutic was in a Phase 1 study. The primary purpose of the study was the evaluation of the therapeutic’s safety profile. It is listed here as Phase 2 because the therapeutic was being tested in patients and not healthy volunteers. This study may be categorized by partners or on regulatory sites, such as ClinicalTrials.gov, as a Phase 1 study.1
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://ionis.com/science-and-innovation/pipeline/ 2. ClinicalTrials.gov identifier: NCT03976349. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT03976349/
ION464 (Multiple system atrophy)
ION464e
(Multiple system atrophy)
ION464, clinically known as BIIB101, is an investigational RNA-targeted therapeutic (RTT) designed to inhibit the production of the alpha-synuclein protein as a potential therapy for multiple system atrophy (MSA).1-3
This investigational RTT is being assessed in HORIZON, a Phase 2,a multiple-ascending-dose study (part 1) to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ION464 in adult participants diagnosed with MSA with a long-term extension (part 2). This is a first-in-human, randomized, blinded, placebo-controlled study of approximately 40 patients.1,2
Part 1 of the study will consist of a screening period of up to 6 weeks, a treatment period of 12 weeks, and a follow-up period of 24 weeks. The study duration for each patient in part 2 will be approximately 96 weeks, consisting of a 72-week treatment period and a 24-week follow-up period.2
Study start 2:
Estimated study completion 2:
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://ionis.com/science-and-innovation/pipeline/ 2. ClinicalTrials.gov identifier: NCT04165486. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT04165486/ 3. National Health Service. A phase 1 study to assess the safety, tolerability, and pharmacokinetics of BIIB101/ION464 administered intrathecally to adults with multiple system atrophy (HORIZON). Accessed August 4, 2025. https://www.hra.nhs.uk/planning-and-improving-research/application-summaries/research-summaries/262sp101-phase-1-study-of-biib101-in-multiple-system-atrophy/
Tofersen (Superoxide dismutase 1 amyotrophic lateral sclerosis)
Tofersen
(Superoxide dismutase 1
amyotrophic lateral sclerosis)
IONIS-MAPTRx, also known as BIIB080, is an investigational RNA-targeted therapeutic (RTT) designed to selectively inhibit the production of the microtubule-associated protein tau (MAPT), or tau protein in the brain. IONIS-MAPTRx is being developed as a potential therapy for patients with Alzheimer’s disease (AD) and potentially other neurodegenerative disorders characterized by the deposition of abnormal tau protein within nerve cells in the brain.1,2
This investigational RTT was evaluated in a Phase 1-2, randomized, double-blind, placebo-controlled study followed by an open-label extension to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple ascending doses of intrathecally administered IONIS-MAPTRx in patients with mild AD.2,3
Study start 1:
Estimated study completion 1:
1. Ionis Pharmaceuticals. The Ionis Antisense Pipeline. Accessed August 15, 2024. https://www.ionis.com/ionis-technology/antisense-pipeline/ 2. Ionis Pharmaceuticals. Ionis Neurology Pipeline. Accessed August 21, 2024. https://www.ionis.com/ionis-technology/innovation/pipeline/transforming-the-management-of-neurology/ 3. ClinicalTrials.gov identifier: NCT03186989. Accessed August 19, 2024. https://clinicaltrials.gov/study/NCT03186989/
IONIS-MAPTRx (Alzheimer’s disease)
IONIS-MAPTRxe
(Alzheimer’s disease)
IONIS-MAPTRx , clinically known as BIIB080, is an investigational RNA-targeted therapeutic (RTT) designed to inhibit the production of the microtubule-associated protein tau (MAPT), or tau protein in the central nervous system (CNS). IONIS-MAPTRx is being developed as a potential therapy for patients with Alzheimer’s disease (AD).1,2
This investigational RTT was evaluated in a Phase 1/2, randomized, double-blind, placebo-controlled study, followed by an open-label extension to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple ascending doses of intrathecally administered IONIS-MAPTRx in patients with mild AD.1,2
Study start 2:
Study completion 2:
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://ionis.com/science-and-innovation/pipeline/ 2. ClinicalTrials.gov identifier: NCT03186989. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT03186989/
Salanersen (Spinal muscular atrophy)
Salanersen
(Spinal muscular atrophy)
Salanersen, clinically known as BIIB115, is an investigational RNA-targeted therapeutic (RTT) being developed as a potential therapy for patients with spinal muscular atrophy (SMA). Salanersen is designed to increase the production of functional survival motor neuron protein.1,2
This investigational RTT is being studied in a Phase 2,a randomized, blinded, placebo-controlled, single ascending-dose study (SAD) in healthy adult male volunteers and an open-label multiple ascending-dose study in pediatric patients with SMA previously treated with onasemnogene abeparvovec. This study will evaluate the safety, tolerability, and pharmacokinetics of salanersen.1,2
Part A consists of screening, treatment, and follow-up periods. The treatment and follow-up period will last for 13 months. Part B will consist of a treatment and follow-up period for children with SMA and will last for 24 months.2
Study start 2:
Estimated study completion 2:
aThis investigational antisense therapeutic is being tested in a Phase 1 study. The primary purpose of the study is the evaluation of the therapeutic’s safety profile, tolerability, and pharmacokinetics. It is listed here as Phase 2 because the therapeutic is being tested in healthy adult male volunteers and pediatric patients with SMA.1,2
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://ionis.com/science-and-innovation/pipeline/ 2. ClinicalTrials.gov identifier: NCT05575011. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT05575011/
Ulefnersen (Fused in sarcoma amyotrophic lateral sclerosis)
Ulefnersen
(Fused in sarcoma
amyotrophic lateral sclerosis)
Ulefnersen, clinically known as ION363, is an investigational RNA-targeted therapeutic (RTT) designed to reduce the production of the fused in sarcoma (FUS) protein as a potential therapy for people with amyotrophic lateral sclerosis caused by mutations in the FUS gene.1,2
This investigational RTT is being studied in FUSION, a Phase 1-3, multicenter, three-part study evaluating the pharmacokinetics, pharmacodynamics, efficacy, and safety of ulefnersen in up to 95 participants. Part 1 will consist of participants randomized in a 2:1 ratio to receive a multidose regimen of ulefnersen or placebo for a period of 60 weeks, followed by part 2, in which participants will receive open-label ulefnersen for a period of 84 weeks. Participants may continue to receive open-label ulefnersen in part 3 for up to 3 additional years or until it becomes commercially available in the participant's country or until discontinuation of the development program, whichever occurs earlier.2
Study start 2:
Estimated study completion 2:
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://ionis.com/science-and-innovation/pipeline/ 2. ClinicalTrials.gov identifier: NCT04768972. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT04768972/
Tominersen (Huntington’s disease)
Tominersen
(Huntington’s disease)
Tominersen, clinically known as IONIS-HTTRx and RG6042, is an investigational RNA-targeted therapeutic (RTT) being evaluated as a potential therapy for patients with Huntington’s disease (HD).1,2
This investigational RTT is being studied in GENERATION HD2, a Phase 2 randomized, double-blind, placebo-controlled, dose-finding study to evaluate the safety, biomarkers, and efficacy of tominersen in patients with prodromal and early manifest HD.2
Study start 2:
Estimated study completion 2:
1. Ionis Pharmaceuticals. Pipeline. Accessed July 15, 2025. https://ionis.com/science-and-innovation/pipeline 2. ClinicalTrials.gov identifier: NCT05686551. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT05686551/
ION269 (Alzheimer's diseasein Down syndrome)
ION269d
Alzheimer's disease in Down
syndrome
ION269 is an investigational RNA-targeted therapeutic (RTT) designed to lower the amount of amyloid precursor protein for the potential treatment of Alzheimer's disease (AD), which is initially being studied in adults with Down syndrome (DS) who have a genetic risk of developing AD.1
This investigational RTT is being studied in HERO, a Phase 2,a multicenter, open-label, single ascending dose study in adult participants with DS who have evidence of brain amyloid positivity. Participants will be examined in 3 separate cohorts and will receive a single dose of the study drug during the 36-week treatment period, followed by a 4-week follow-up period.1,2
Estimated study start 2:
Estimated study completion 2:
aThis investigational antisense oligonucleotide therapeutic is in a Phase 1b study. It is listed here in Phase 2 because the therapeutic is being tested in people living with Down syndrome.1
1. Ionis Pharmaceuticals. Pipeline. Accessed March 3, 2025. https://www.ionis.com/science-and-innovation/pipeline/
2. ClinicalTrials.gov identifier: NCT06673069. Accessed February 26, 2025. https://www.clinicaltrials.gov/study/NCT06673069/
aContent in the table subject to change pending updates to Ionis pipeline. bSafety and efficacy have not been evaluated by any regulatory authorities for the indications described. cThe US Food and Drug Administration has granted both orphan drug designation and rare pediatric disease designation for its investigational drug ION582.15 dThis investigational antisense therapeutic is in a Phase 1/2a study.1 eThis investigational antisense therapeutic is in a Phase 1 study. The primary purpose of the study is the evaluation of the therapeutic’s safety profile. It is listed here as Phase 2 because the therapeutic is being tested in patients and not healthy volunteers. This study may be categorized by partners or on regulatory sites, such as ClinicalTrials.gov, as a Phase 1 study.1
FUS, fused in sarcoma protein gene; GFAP, glial fibrillary acidic protein gene; HTT, huntingtin gene; LRRK2, leucine-rich-repeat kinase 2 gene; MECP2, methyl-CpG-binding protein 2 gene (human); PLP1, proteolipid protein 1 gene; PRNP, prion protein gene; SCNA, synuclein alpha gene; SMN2, survival of motor neuron 2 gene; SOD1, superoxide dismutase 1 gene; TAU, tau protein gene; UBE3A-ATS, ubiquitin protein ligase E3A antisense transcript.
Learn more about the current clinical trials for Ionis’ RNA-targeted therapeutics.
Learn more about the neurologic diseases in Ionis’ pipeline.
References
- Ionis Pharmaceuticals. Pipeline. Accessed March 3, 2025. https://ionis.com/science-and-innovation/pipeline
- Ionis Pharmaceuticals. Ionis Innovation Day. October 4, 2023. Accessed March 3, 2025. https://ir.ionis.com/static-files/8b71dc65-dad9-4368-9014-604c5b203ca1/
- 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.
- 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/
- Crooke ST, Liang XH, Baker BF, Crooke RM. Antisense technology: a review. J Biol Chem. 2021;296:100416.
- Crooke ST, Witztum JL, Bennett CF, Baker BF. RNA-targeted therapeutics [published correction appears in Cell Metab. 2019 Feb 5;29(2):501. doi: 10.1016/j.cmet.2019.01.001]. Cell Metab. 2018;27(4):714-739.
- Ionis Pharmaceuticals. Data on file.
- Ionis Pharmaceuticals. Medicines. Accessed March 3, 2025. https://www.ionis.com/medicines/
- ClinicalTrials.gov identifier: NCT04849741. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT04849741/
- ClinicalTrials.gov identifier: NCT06914609. Accessed July 15, 2025. https://www.clinicaltrials.gov/study/NCT06914609/
- ClinicalTrials.gov identifier: NCT06153966. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT06153966/
- ClinicalTrials.gov identifier: NCT06430385. Accessed July 15, 2025. https://clinicaltrials.gov/study/ NCT06430385/
- ClinicalTrials.gov identifier: NCT06150716. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT06150716/
- ClinicalTrials.gov identifier: NCT03976349. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT03976349/
- ClinicalTrials.gov identifier: NCT04165486. Accessed July 15, 2025. https://clinicaltrials.gov/study/ NCT04165486/
- ClinicalTrials.gov identifier: NCT02623699. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT02623699/
- ClinicalTrials.gov identifier: NCT03186989. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT03186989/
- ClinicalTrials.gov identifier: NCT05575011. Accessed July 15, 2025. https://clinicaltrials.gov/ study/NCT05575011/
- ClinicalTrials.gov identifier: NCT04768972. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT04768972/
- ClinicalTrials.gov identifier: NCT05686551. Accessed July 15, 2025. https://clinicaltrials.gov/study/NCT05686551/
- First patient dosed in pivotal Phase 3 REVEAL clinical study of ION582 in Angelman syndrome. June 11, 2025. Accessed July 15, 2025. https://ir.ionis.com/node/32691/pdf
