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AT-001
AT-001 is an antisense oligonucleotide that reduces production of lipoprotein(a) by targeting hepatic apolipoprotein(a) synthesis.
AT-001 is an antisense oligonucleotide that reduces production of lipoprotein(a) by targeting hepatic apolipoprotein(a) synthesis. Used for Elevated lipoprotein(a) for reduction of cardiovascular risk.
At a glance
| Generic name | AT-001 |
|---|---|
| Sponsor | NYU Langone Health |
| Drug class | Antisense oligonucleotide |
| Target | Apolipoprotein(a) mRNA |
| Modality | Small molecule |
| Therapeutic area | Cardiovascular |
| Phase | Phase 3 |
Mechanism of action
AT-001 works by binding to messenger RNA encoding apolipoprotein(a) in the liver, leading to degradation of that mRNA and reduced synthesis of lipoprotein(a), a genetically determined risk factor for cardiovascular disease. By lowering circulating lipoprotein(a) levels, the drug aims to reduce atherosclerotic cardiovascular disease risk in patients with elevated baseline levels.
Approved indications
- Elevated lipoprotein(a) for reduction of cardiovascular risk
Common side effects
- Injection site reactions
- Transient liver enzyme elevation
- Flu-like symptoms
Key clinical trials
- Grapiprant and Eribulin for the Treatment of Metastatic Inflammatory Breast Cancer (PHASE1, PHASE2)
- Safety and Efficacy of AT-001 in Patients With Diabetic Cardiomyopathy (PHASE3)
- Cardiovascular Effects of COVID-19 (PHASE2)
- AT-001 for Long-term Preservation of Brain Health in Aging (NA)
- Safety and Tolerability of Antioxidant (AT-001)for Reducing Brain Oxidative Stress (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- AT-001 CI brief — competitive landscape report
- AT-001 updates RSS · CI watch RSS
- NYU Langone Health portfolio CI