Tivanisiran

Tivanisiran (also known by its research code SYL1001) is a small interfering RNA (siRNA) therapeutic agent under investigation for the treatment of dry eye disease and other ocular conditions. It is designed to target and downregulate the expression of the transient receptor potential vanilloid 1 (TRPV1) channel, which is implicated in the pathophysiology of ocular pain and inflammation.

Mechanism of Action

Tivanisiran functions by utilizing the RNA interference (RNAi) pathway to specifically silence the expression of the TRPV1 gene. TRPV1 is a non-selective cation channel that is activated by various stimuli, including heat, protons, and inflammatory mediators. It is expressed in sensory neurons and is involved in the transmission of pain and inflammatory signals. By reducing TRPV1 expression, Tivanisiran aims to alleviate symptoms associated with dry eye disease, such as pain and discomfort.

Clinical Development

Tivanisiran is being developed by Sylentis, a biopharmaceutical company specializing in RNAi-based therapies. The drug has undergone several clinical trials to evaluate its safety and efficacy in patients with dry eye disease.

Preclinical Studies

In preclinical models, Tivanisiran demonstrated the ability to reduce TRPV1 expression and alleviate symptoms of ocular pain and inflammation. These promising results provided the basis for advancing the compound into clinical trials.

Clinical Trials

Tivanisiran has been evaluated in multiple phases of clinical trials:

• Phase I Trials: Initial studies focused on assessing the safety and tolerability of Tivanisiran in healthy volunteers and patients with dry eye disease. The results indicated that the drug was well-tolerated with no significant adverse effects.

• Phase II Trials: These trials aimed to evaluate the efficacy of Tivanisiran in reducing symptoms of dry eye disease. Patients treated with Tivanisiran reported improvements in ocular pain and discomfort compared to placebo.

• Phase III Trials: Ongoing studies are further assessing the long-term safety and efficacy of Tivanisiran in a larger patient population.

Potential Applications

Beyond dry eye disease, Tivanisiran may have potential applications in other ocular conditions where TRPV1 plays a role, such as ocular neuropathic pain and corneal inflammation.

Safety and Side Effects

Tivanisiran has been generally well-tolerated in clinical trials. The most common side effects reported include mild ocular irritation and transient redness at the site of administration. No serious adverse events have been attributed to the drug.

Regulatory Status

As of the latest updates, Tivanisiran is in the late stages of clinical development. Regulatory submissions will depend on the outcomes of ongoing Phase III trials.

Also see

• Dry eye syndrome
• RNA interference
• Transient receptor potential channels
• Ocular pharmacology

References

Template:RNAi drugs

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