ARTICLE | Product Development
In first human RNA-editing data, Wave reports positive results from WVE-006 in alpha-1 antitrypsin deficiency
By Lauren Martz, Executive Director, Biopharma Intelligence
October 17, 2024 12:16 AM UTC
The first clinical data from an RNA-editing oligonucleotide therapy demonstrate that it’s possible to change a single base in an RNA to correct a disease-causing mutation in patients. The data represent another step forward in Wave Life Science’s comeback story and provide validation for the broader concept of RNA editing.
On Wednesday, Wave Life Sciences Inc. (NASDAQ:WAVE) reported that WVE-006 increased wild-type AAT protein levels to about 60% of normal in the first two patients with alpha-1 antitrypsin deficiency (AATD) treated in the Phase II RestorAATion-2 study. Reaching at least 50% of normal is the goal, as patients heterozygous for disease-causing mutations in the AAT gene have a relatively normal phenotype…
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- Source: https://www.biocentury.com/article/653892/wave-data-propel-rna-editing-stocks-but-are-all-platforms-created-equal