Wave Life Sciences has reported promising early results from its RNA editing treatment for alpha-1 antitrypsin deficiency (AATD), showing significant increases in AAT protein levels in two patients. This marks a milestone in therapeutic RNA editing, with no serious side effects observed. The findings have boosted investor confidence, leading to a 75% rise in Wave's shares, while GSK prepares to take over development under their partnership.
Takeda has terminated its partnership with Wave Life Sciences regarding the investigational antisense oligonucleotide WVE-003 for Huntington’s disease, after investing $260 million since their 2018 agreement. Despite this unexpected withdrawal, Wave retains full responsibility for WVE-003 and anticipates regulatory feedback on accelerated approval by the end of 2024. The biotech continues to advance its pipeline, including successful RNA editing demonstrations and improvements in treatments for other conditions.
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