Tag: Gene silencing

ONPATTRO (patisiran) was the first-ever small interfering RNA (siRNA) therapeutic approved by the U.S. Food and Drug Administration (FDA) in 2018 for treating a genetic disease. This groundbreaking treatment, which works by "silencing" a gene, has raised a crucial question in the field of genetic medicine: is ONPATTRO a gene therapy?

In 2018, ONPATTRO became the first FDA-approved medication using RNA interference technology, ushering in a new era of gene-based medicines. The groundbreaking therapy acts as a gene silencer, specifically targeting and reducing the production of a protein responsible for a rare, debilitating genetic disease known as hATTR amyloidosis.

Hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) is a rare and progressive genetic disorder, estimated to affect between 10,000 and 40,000 people globally. This condition is caused by a gene mutation that results in abnormal protein deposits that damage the nervous system and other vital organs.

In 2022, the FDA approved Amvuttra (vutrisiran) as a major therapeutic advance for specific types of amyloidosis. Often mistakenly searched for as 'ambutra,' this medication represents a powerful form of gene-silencing therapy that targets the root cause of hereditary transthyretin-mediated (hATTR) amyloidosis.

As the first FDA-approved small interfering RNA (siRNA) therapy, Onpattro (patisiran) represents a significant advance in treating hereditary transthyretin-mediated (hATTR) amyloidosis. This groundbreaking medication uses a unique gene-silencing mechanism to address the root cause of the disease, rather than just managing its symptoms.

First approved in 2022 for hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy, **vutrisiran** (brand name Amvuttra) is a gene-silencing medication that offers a new approach to treatment. It works by targeting the root genetic cause of the disease, preventing the production of the toxic protein that causes nerve and heart damage.