The First Clinical Trial for RNA Editing Begins in the UK


According to information in a recent press release from Wave Life Sciences, dosing has begun in a clinical trial investigating the RNA editing candidate, WVE-006. The drug treats alpha-1 antitrypsin deficiency (ATTD) and began in the UK in December 2023. WVE-006 has been designed to remedy the RNA mutation in AATD. In anticipation of RNA’s increasing popularity, it might be worth noting the difference between RNA editing and DNA editing.

Both editing types maintain the same goal, which is to alter the quantity and structure of a protein. However, there is a difference. Deoxyribonucleic acid (DNA) is the molecule that holds the genetic information for the functioning and development of an organism. It defines who we are. Comparing any two individuals, their DNA is 99% identical. The 0.1% difference contains variations influencing our uniqueness.

Science is only beginning to understand DNA while the study of RNA has thus far been neglected.
Adding the definition of RNA, which is found in every cell, deepens the mystery. Ribonucleic acid (RNA) is a molecule found in most living viruses and organisms in single stranded forms. The RNA virus uses RNA rather than DNA as its genetic material. It can cause multiple human diseases. A major advantage is that results are temporary.

One example of RNA’s application as a tool is CRISPR, the “gene scissor.” If mistakes are made during DNA editing, off target effects occur. Cells continuously turn out copies of RNA. Instead of being cemented in the blueprint of a cell, any genetic changes that occur in short-lived RNA may be reversed or even halted. This would be safer than DNA editing. Another comparison shows that editing with RNA is less complicated. CRISPR uses “machines” from bacterial proteins capable of cutting genes. In addition, it uses an RNA molecule that directs the editing.

The Cup is Half Full

Every DNA cell has only two chromosomes that DNA editors need to attend to compared to many more targets in RNA editing. He also said that when the life of an RNA editor is expanded, it creates the same risks as off-target incidents. Therefore, the lifespan of RNA editing must be extended and continue to be active in order for it to be effective. If there is a mistake in editing, problems similar to those in DNA editing will occur. However, RNA editing is new to the clinic. As RNA editing receives more attention these issues may be resolved.

About WVE-006

WVE-006, a synthetic RNA molecule, enlists the human enzyme adenosine deaminase (ADAR) when editing RNA. Wave anticipates delivering data in 2024 showing how WVE-006 interacts with its target plus other requisite study results.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

Follow us