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SYL1001 ophthalmic solution
SYL1001 is a small interfering RNA (siRNA) that silences the production of transforming growth factor-beta 2 (TGF-β2) to reduce intraocular pressure in glaucoma.
SYL1001 is a small interfering RNA (siRNA) that silences the production of transforming growth factor-beta 2 (TGF-β2) to reduce intraocular pressure in glaucoma. Used for Open-angle glaucoma or ocular hypertension.
At a glance
| Generic name | SYL1001 ophthalmic solution |
|---|---|
| Sponsor | Sylentis, S.A. |
| Drug class | siRNA (small interfering RNA) |
| Target | TGF-β2 (transforming growth factor-beta 2) |
| Modality | Small molecule |
| Therapeutic area | Ophthalmology |
| Phase | Phase 3 |
Mechanism of action
SYL1001 works by targeting and degrading TGF-β2 messenger RNA in ocular tissues, thereby reducing TGF-β2 protein levels. Elevated TGF-β2 is associated with increased intraocular pressure and glaucomatous damage. By reducing TGF-β2 expression, the drug aims to lower intraocular pressure and slow or prevent glaucomatous progression.
Approved indications
- Open-angle glaucoma or ocular hypertension
Common side effects
- Ocular irritation or discomfort
- Conjunctival hyperemia
- Eye pain
Key clinical trials
- Tivanisiran for Dry Eye in Subjects With Sjögren's Syndrome (PHASE3)
- Safety Study of Tivanisiran to Treat Dry Eye (PHASE3)
- HELIX, a Double-masked Study of SYL1001 in Patients With Moderate to Severe Dry Eye Disease (DED) (PHASE3)
- Dose-finding Study to Assess the Safety and Effect of SYL1001 in Patients With Ocular Pain (PHASE2)
- Pilot Study to Evaluate SYL1001 Safety and Effect in Patients With Ocular Pain (PHASE1, PHASE2)
- Study to Evaluate the Ocular Tolerance of SYL1001 in Healthy Volunteers (PHASE1)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |