#3 RNA-based Therapeutics

Overview

This particular innovation uses RNA antisense technology to treat patients who are unable to reach their targeted cholesterol levels with statins alone or who are statin intolerant. The therapy is intended to reduce the production of ApoB-100, a protein that carries certain forms of cholesterol and triglycerides in the bloodstream.
 
This remains a compelling and very active area of research and development. Agents are in various stages of clinical trials for a diverse range of conditions, including viral infections, cancer, macular degeneration, and hereditary hypercholesterolemia.
 

Where Are They Now

This particular innovation uses RNA antisense technology to treat patients who are unable to reach their targeted cholesterol levels with statins alone or who are statin intolerant. The therapy is intended to reduce the production of ApoB-100, a protein that carries certain forms of cholesterol and triglycerides in the bloodstream.

Nearly 40 RNA antisense based therapies are in various stages of clinical trials for cancer, ebola and HIV. In a 2016 market forecast for 2020 researchers predict possible continued growth in RNA-based therapeutics due to its potential to treat chronic diseases such as cancer and diabetes. It remains in the clinical research phase because of its high failure rates and high expenses but forecasts indicate that the gene splicing aspect of this advancement provides the room for future advancement. In 2017, researchers discovered a billion-year-old primordial system designed to protect against viral infection still found in human cells. The ancient system, separate from the powerful defense system humans overwhelmingly depend on today, could be the next wave of precision medicine as it provides RNA-based therapeutics with a mechanism of action free from the typical unwelcomed immune response.

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