TLDR:
- Wave Life Sciences reported successful RNA editing in humans for the first time
- A single dose of WVE-006 increased wild-type M-AAT to over 60% of total AAT in AATD patients
- Total AAT levels reached 10.8 micromolar, meeting regulatory approval standards for AAT therapies
- RNA-editing stocks surged on the news, with Wave Life Sciences up 74%
- Wave plans to share multi-dose data from the RestorAATion-2 trial in 2025
Wave Life Sciences has achieved a significant milestone in the field of RNA editing. The company reported the first-ever successful therapeutic RNA editing in humans through its RestorAATion-2 trial of WVE-006, a treatment for alpha-1 antitrypsin deficiency (AATD).
AATD is a genetic disorder that can cause lung and liver diseases. People with the condition lack a protein called alpha-1 antitrypsin (AAT), which protects the lungs and liver from damage. The trial focused on patients with the “ZZ” genotype of AATD, who do not naturally produce the normal, or “wild-type,” form of AAT known as M-AAT.
In the trial, a single dose of WVE-006 was given to two patients. The results were impressive. The treatment increased the levels of wild-type M-AAT to more than 60% of the total AAT in the patients’ blood.
This is a significant achievement because it brings the patients’ AAT profile closer to that of people with the “MZ” genotype, who have a lower risk of developing AATD-related lung and liver diseases.
The total AAT levels in the patients’ blood reached 10.8 micromolar. This is an important threshold because it meets the level that has been the basis for regulatory approval of other AAT therapies. The increase in AAT was observed as early as three days after treatment and lasted through day 57 of the trial.
Paul Bolno, the President and CEO of Wave Life Sciences, expressed enthusiasm about the results. He noted that the level of mRNA editing observed with a single dose exceeded their expectations. The company believes that with repeat dosing, the levels of M-AAT could continue to increase.
The treatment appears to be well-tolerated so far. All adverse events reported in both the RestorAATion-2 trial and an ongoing trial with healthy volunteers have been mild to moderate. No serious adverse events have been reported.
This breakthrough has significant implications for the field of RNA editing and for patients with AATD. Currently, treatment options for AATD are limited. The only approved therapy for lung disease requires weekly intravenous infusions. There are no approved therapies for AATD-related liver disease, which often leads to liver transplantation.
The news of Wave Life Sciences’ success had a major impact on the stock market. Shares of Wave Life Sciences surged by 74%, reaching their highest level since September 2020. This jump added nearly $950 million to the company’s market value.
Other companies working on RNA-editing therapies also saw their stocks rise dramatically. ProQR Therapeutics shares more than doubled, increasing by 122%. Korro Bio’s shares climbed by 94%. These were record one-day gains for both companies.
Analysts responded positively to the news. Madison El-Saadi, an analyst at B Riley Securities, highlighted the significance of seeing the treatment work in humans for the first time. Luca Issi from RBC Capital Markets described the data as “fantastic news for the broader field” of RNA editing.
Wave Life Sciences plans to continue its research. The company expects to share data from a multi-dose trial of WVE-006 in 2025. They will also present more information about the trial results and discuss new RNA editing programs at their Annual Research Day on October 30.
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