Gene Editing Drugs Developed to Combat Cholesterol

Amid the growing concerns about the side effects of weight-loss drugs, groundbreaking research presented at the annual meeting of the American Heart Association has unveiled two experimental medications showing significant promise in reducing bad cholesterol levels (1^✔ ✔Trusted Source
A single infusion of a gene-editing medicine may control inherited high LDL cholesterol

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New Solutions for High Cholesterol

According to NBC News, these medications are not intended for weight loss but rather cater to individuals with a specific genetic predisposition. People genetically prone to high cholesterol can potentially use these drugs in conjunction with a balanced diet and regular exercise to better manage their risk of heart attack and stroke. 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 - (https://newsroom.heart.org/news/a-single-infusion-of-a-gene-editing-medicine-may-control-inherited-high-ldl-cholesterol )

Source-Medindia