Biotech firm, Wave Life Sciences Ltd (NASDAQ: WVE) has achieved the first-ever therapeutic RNA editing in humans within their RestorAATion-2 trial.
Announced on Wednesday, the company’s shares jumped over 70 per cent as the trial for WVE-006, a candidate for treating Alpha-1 antitrypsin deficiency (AATD), a genetic condition that can lead to lung and liver disease.
Investors and analysts are interpreting this breakthrough as a strong indicator of the company’s potential in the biotech sector, particularly in the niche of RNA therapeutics. This sector aims to treat diseases at the RNA level, potentially offering curative therapies for conditions previously deemed untreatable.
RNA editing involves modifying RNA to correct or alter protein production in the body. This technique could revolutionize treatment for numerous genetic disorders by addressing them at their root cause, rather than merely managing symptoms. Furthermore, the success in human trials displays Wave’s proprietary platform’s capability, which uses stereopure oligonucleotides to facilitate precise editing.
“Achieving the first-ever therapeutic RNA editing in humans is a significant milestone for our organization, for our GSK collaboration, and for the entire oligonucleotide field,” said Paul Bolno, president and CEO of Wave Life Sciences.
“The level of mRNA editing we are observing with a single dose exceeded our expectations and we expect M-AAT levels to continue to increase with repeat dosing, based on our preclinical data.”
Bolno highlighted that the initial data, along with the durability of WVE-006 and its convenient subcutaneous administration, support its potential to be best-in-class when compared to other gene editors and treatments in the broader AATD field.
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Wave has big deal with pharma giant GSK
Pharmaceutical giant, GSK plc (LON: GSK), holds the exclusive global license for WVE-006.
Subsequently, after Wave completes the RestorAATion-2 study, GSK will take over development and commercialization. Wave is also eligible to receive up to USD$525 million in milestone payments, along with tiered royalties on net sales for WVE-006.
Wave Life Sciences’ partnership with GSK centers around the exclusive global license for WVE-006, a therapeutic designed to address AATD. This collaboration positions Wave to benefit from GSK’s extensive global reach and expertise in advancing innovative treatments.
As part of the deal, Wave Life Sciences stands to receive up to USD$525 million in milestone payments and tiered royalties on the net sales of WVE-006. This alliance allows both companies to collaborate in advancing RNA-based therapies.
An estimated 200,000 individuals in the US and Europe are homozygous for the SERPINA1 Z mutation and live with AATD. Current treatment options are limited to weekly IV augmentation therapy, which only addresses lung disease and generated over USD$1.4 billion in global sales in 2023. There are no approved therapies for AATD-related liver disease, leading many affected individuals to require liver transplants.
The positive market response suggests broader implications for the biotechnology industry. Wave Life Sciences’ success further validates RNA editing as a viable therapeutic approach, which could attract more investment into this area of research.
This breakthrough may also give Wave a competitive advantage in the race to develop RNA-based treatments, although the field remains highly competitive with other companies advancing their technologies. Wave is likely to draw increased interest from larger pharmaceutical companies for potential partnerships and may also see a boost in funding for further research.
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