| Size | Price | Stock | Qty |
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| 1mg |
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| 5mg |
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| Other Sizes |
| Targets |
TRPV1 mRNA. Tivanisiran is a double-stranded RNA molecule that targets the TRPV1 transcript through RNA interference, leading to decreased translation of TRPV1 protein, which is involved in pain and inflammation pathways in the ocular surface.
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| ln Vitro |
Tizanisiran transfection in vitro has the ability to slash TRPV1 mRNA levels by 50–60%[1][2].
In vitro studies show that tivanisiran silences TRPV1 expression in human corneal epithelial cells and trigeminal ganglia neurons at concentrations in the low nanomolar range. TRPV1 protein knockdown of approximately 50-70% has been demonstrated. |
| ln Vivo |
In animal models of dry eye, topical ocular administration of tivanisiran reduced corneal staining, increased tear production, and decreased inflammatory markers. In clinical trials, tivanisiran ophthalmic solution (1.125% or 1.5%) demonstrated significant improvement in dry eye symptoms and signs, including corneal fluorescein staining scores, after 28 days of treatment.
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| Enzyme Assay |
No cell-free enzyme/receptor binding assays are applicable for tivanisiran as it is an RNAi agent. Target engagement is assessed via knockdown of TRPV1 mRNA. For determination of nuclease stability, tivanisiran is incubated in human tear fluid or corneal homogenates at 37degC, and remaining intact siRNA is measured by HPLC or gel electrophoresis at various time points.
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| Cell Assay |
Immortalized human corneal epithelial cells or primary trigeminal ganglion neurons are seeded and treated with tivanisiran complexed with transfection reagent or formulated for topical delivery (0.1-10 uM) for 24-72 hours. TRPV1 mRNA levels are quantified by RT-qPCR and normalized to housekeeping genes (e.g., GAPDH). TRPV1 protein is measured by Western blot or immunofluorescence. Cell viability is assessed by MTT.
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| Animal Protocol |
In vivo studies are typically conducted in a mouse model of dry eye induced by desiccating stress or scopolamine administration. Tivanisiran ophthalmic solution (1.125-1.5% w/v) is administered topically, 1-2 drops per eye, 2-4 times daily for 7-28 days. Tear production is measured by phenol red thread test. Corneal fluorescein staining is scored to assess ocular surface damage. Eyes are harvested for histology and TRPV1 expression analysis.
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| ADME/Pharmacokinetics |
PK data are limited for tivanisiran. Following topical ocular administration, systemic absorption is minimal (<5% of dose). Ocular PK studies in rabbits show detectable siRNA levels in cornea and conjunctiva for up to 24 hours post-administration. Degradation products are eliminated renally. No formal human PK studies are publicly available.
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| Toxicity/Toxicokinetics |
Preclinical toxicology studies in rabbits and dogs demonstrated that topical ocular administration of tivanisiran is well-tolerated, with no significant adverse ocular findings at clinical doses (up to 1.5%). In clinical trials, the most common adverse event was mild, transient eye irritation (burning/stinging) upon instillation. No systemic toxicities reported.
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| References | |
| Additional Infomation |
Other information: Tivanisiran completed Phase 3 clinical trials for dry eye disease but did not meet all primary endpoints. Further development may be ongoing. Tivanisiran was granted Orphan Drug designation for dry eye syndrome. Not FDA- or EMA-approved as of 2026. It represents a first-in-class siRNA approach targeting the TRPV1 pathway for ocular surface diseases.
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| CAS # |
1848224-71-4
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| Appearance |
White to off-white solid powder
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment (e.g. under nitrogen), avoid exposure to moisture. |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
H2O: 100 mg/mL
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| Solubility (In Vivo) |
Solubility in Formulation 1: 25 mg/mL (Infinity mM) in PBS (add these co-solvents sequentially from left to right, and one by one), clear solution; with sonication.
(Please use freshly prepared in vivo formulations for optimal results.) |
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution concentration mg/mL). Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug.
Method for preparing in vivo formulation:：Take μL DMSO stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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