Gene Editing Drugs Developed to Combat Cholesterol

One of these treatments employs gene-editing techniques, while the other utilizes mRNA. The first, developed by Verve Therapeutics, uses base editing to target the PCSK9 gene responsible for producing LDL, commonly known as “bad” cholesterol.

Verve’s co-founder and CEO, Dr. Sekar Kathiresan, describes the medication as a permanent eraser, eliminating the gene’s ability to elevate cholesterol.


The second medication, reported by NBC News, is a pioneering therapy addressing lipoprotein(a) or Lp(a) — a perilous form of cholesterol. Individuals with elevated Lp(a) levels face a heightened risk of arterial fat buildup.

This genetic condition, as noted by Dr. Steven Nissen, the study’s author, is traditionally challenging to treat. Nissen’s team has developed lepodisiran, a drug akin to COVID-19 vaccines, targeting mRNA to stimulate the production of antibodies.

Awaiting Approval and Public Availability

Despite these promising developments, it will be some time before these medications are accessible to the public. Neither treatment has undergone human testing, necessitating further research and examination before potential approval by the Food and Drug Administration.

Despite this cautious approach, experts at the meeting lauded the research findings as nothing short of revolutionary. Dr. Hugh Cassiere, director of critical care services at South Shore University Hospital, Northwell Cardiovascular Institute in New York, emphasized, “There is no way to categorize this other than revolutionary.” Cassiere was not involved in either research project.

Reference :

  1. A single infusion of a gene-editing medicine may control inherited high LDL cholesterol – ( )
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Source: Medindia


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