Oct. 17, 2024 – The stock of Wave Life Sciences (WVE, Financial), a global biotechnology company, has risen by 74% on Wednesday after a major development. The company has scored a landmark by delivering the essential therapeutic RNA editing in human trials for the first time. This finding may open the door for treatment of many other genetic disorders like Alpha-1 Antitrypsin Deficiency (AATD).
Wave's RNA editing technology appears to have highly potential for the treatment of AATD, a genetic disorder with potential life threatening and implicating the lungs and liver. As recently demonstrated in a Phase I and II concept trial, a single dose of WVE-006, Wave's RNA editing based therapy, boosted the levels of wild type alpha-1 antitrypsin protein which is severely low in patients suffering from AATD. It may be possible that this increase will help to decrease the incidence of AATD associated lung and liver diseases.
The treatment was safe and no severe side effects were described. This is a great improvement because, currently, there are few treatment solutions available for AATD and many of them do not work well.
That means hope for more than 200,000 people in the United States and Europe that suffer from AATD and that this discovery opens the door to a whole new range of therapies. Wave Life Sciences have also partnered with GSK to bring WVE-006 to market, and both firms believe that the drug has the potential to change the lives of patients with AATD. This achievement is a great accomplishment for Wave Life Sciences and for the overall field of RNA editing.
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