pharmaphorum October 17, 2024
Shares in Wave Life Sciences rose by 74% after it reported clinical data with a pioneering new RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD).
Wave claims this is the first clinical data with a therapeutic RNA-editing drug in humans, an achievement that boosted its stock, even as the news also emerged that Takeda had pulled out of a Huntington’s disease alliance with the biotech.
The results come from the phase 1b/2a RestorAATion-2 study of WVE-006, a GalNAc-conjugated oligonucleotide that is designed to correct a mutation in mRNA associated with AATD that prevents patients from producing the normal ‘wild-type’ form of the enzyme alpha-1 antitrypsin (M-AAT).
AATD is an inherited disorder whose primary consequence is life-threatening pulmonary emphysema, resulting in irreversible...
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