The latest announcement is out from Wave Life Sciences ( (WVE) ).
Wave Life Sciences Ltd. has achieved a breakthrough in therapeutic RNA editing, unveiling positive proof-of-mechanism data from their RestorAATion-2 study targeting alpha-1 antitrypsin deficiency (AATD). This landmark development marks the first clinical demonstration of RNA editing in humans, showcasing the potential of WVE-006 as a promising treatment option. With a single subcutaneous dose showing significant increases in functional M-AAT protein, the company anticipates further advancements with repeated dosing, solidifying their leadership in RNA medicine innovation.
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