New drugs for treating dyslipidemias. From small molecules to small interfering RNAs

Lluís Masana1, Daiana Ibarretxe1

  • 1Unitat de Recerca en Lípids i Arteriosclerosi, Unitat de Medicina Vascular i Metabolisme, Hospital Universitari Sant Joan, Universitat Rovira i Virgili, IISPV, CIBERDEM, Reus, Tarragona, España.

Insights

New cardiovascular drugs target lipoprotein disorders to reduce persistent cardiovascular events. Research explores novel pathways and mechanisms like RNA interference, including inclisiran, for better patient outcomes.

Area of Science:

  • Cardiovascular Medicine
  • Pharmacology
  • Lipid Metabolism

Background:

  • Cardiovascular diseases (CVDs) remain a leading cause of mortality.
  • Current therapies for lowering LDL-C do not fully eliminate residual cardiovascular risk.
  • Persistent cardiovascular events occur even when LDL-C targets are met.

Purpose of the Study:

  • To review emerging therapeutic targets and pharmacological mechanisms for lipoprotein metabolism disorders.
  • To provide a comprehensive overview of novel drug development in cardiovascular medicine.
  • To assess the potential clinical impact of new treatments for dyslipidemia.

Main Methods:

  • Literature review of recent research on novel cardiovascular drug development.
  • Analysis of pharmacological mechanisms targeting lipoprotein metabolism.
  • Discussion of clinical trial data and regulatory approvals for new agents.

Main Results:

  • Inclisiran represents a first-in-class therapy utilizing RNA interference for cardiovascular risk reduction.
  • Multiple novel pharmacological approaches are under investigation, including small molecules and monoclonal antibodies.
  • These new therapies target different pathways in lipoprotein metabolism, offering alternative treatment strategies.

Conclusions:

  • Novel therapeutic strategies are crucial to address unmet needs in cardiovascular disease prevention.
  • Advancements in understanding lipoprotein metabolism are driving the development of innovative pharmacological agents.
  • The reviewed therapies hold promise for significantly impacting the management of dyslipidemia and reducing cardiovascular events.

Related Concept Videos

siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the...
16.6K
Lipid-Lowering Drugs: Statins and Miscellaneous Agents01:20

Lipid-Lowering Drugs: Statins and Miscellaneous Agents

Hyperlipidemia, a medical condition often referred to as high cholesterol, is characterized by abnormally elevated levels of lipids in the bloodstream. When present in excess, these lipids, specifically cholesterol and triglycerides, can lead to serious health complications, often involving cardiovascular diseases. Illnesses like atherosclerosis, heart attacks, and pancreatitis have all been linked to untreated hyperlipidemia. This means controlling and regulating cholesterol and triglyceride...
532
Small interfering RNAs (siRNA)02:30

Small interfering RNAs (siRNA)

3.5K
RNA Interference01:23

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
25.9K
Experimental RNAi02:15

Experimental RNAi

RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
6.1K
Targets for Drug Action: Overview01:26

Targets for Drug Action: Overview

Drugs target macromolecules to modify ongoing cellular processes. Primary drug targets include receptors, ion channels, transporters, and enzymes.
Receptors are either membrane-spanning or intracellular proteins, which upon binding a ligand, get activated and transmit the signal downstream to elicit a response. Drugs bind receptors, either mimicking the action of endogenous ligands or blocking the receptor activity to bring about a modified response. Nearly 35% of approved drugs target the G...
6.0K