Zilebesiran

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An investigational RNA interference therapeutic agent


Zilebesiran
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Zilebesiran is an investigational RNA interference (RNAi) therapeutic agent designed to treat hypertension. It is being developed by Alnylam Pharmaceuticals, a company known for its work in RNAi therapeutics.

Mechanism of Action[edit | edit source]

Zilebesiran works by targeting the angiotensinogen (AGT) mRNA, which is a precursor to the renin-angiotensin-aldosterone system (RAAS). By silencing the AGT mRNA, zilebesiran reduces the production of angiotensin II, a peptide hormone that causes blood vessels to constrict, leading to increased blood pressure. This mechanism allows for the reduction of blood pressure in patients with hypertension.

Development and Clinical Trials[edit | edit source]

Zilebesiran is currently undergoing clinical trials to evaluate its safety and efficacy. The drug is administered via subcutaneous injection, and its effects are being studied in various populations with hypertension. The trials aim to determine the optimal dosing regimen and to assess the long-term effects of the drug on blood pressure control.

Potential Benefits[edit | edit source]

The use of RNAi technology in zilebesiran offers several potential benefits over traditional antihypertensive medications. These include:

  • Long-lasting effects: Due to its mechanism of action, zilebesiran may provide sustained blood pressure control with less frequent dosing.
  • Targeted action: By specifically targeting AGT mRNA, zilebesiran may reduce the risk of off-target effects and improve safety.
  • Novel approach: As a first-in-class RNAi therapeutic for hypertension, zilebesiran represents a novel approach to managing this common condition.

Challenges and Considerations[edit | edit source]

While zilebesiran shows promise, there are challenges and considerations in its development and potential use:

  • Delivery method: The requirement for subcutaneous injection may be a barrier for some patients compared to oral medications.
  • Long-term safety: The long-term safety profile of zilebesiran needs to be established through ongoing and future clinical trials.
  • Cost and accessibility: As a novel therapeutic, the cost and accessibility of zilebesiran will be important factors in its adoption.

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Contributors: Prab R. Tumpati, MD