FDA Backs Single-Trial Pathway for Wave's AATD RNA Editor
Wave Life Sciences announced on October 1, 2026 that the FDA has accepted its LC-MS assay for distinguishing M-AAT from Z-AAT in alpha-1 antitrypsin deficiency (AATD) patients.
The FDA also expressed support for a single-trial pathway for Wave's RestorAATion-2a trial, potentially streamlining clinical development for the RNA editor therapy.
Data from the 600 mg monthly cohort of the Phase 1b/2a RestorAATion-2 trial are expected in the fourth quarter of 2026.
Most severe AATD is caused by the Z allele, a G-to-A point mutation in SERPINA1 that leads to misfolded Z-AAT protein accumulation in hepatocytes and lung dysfunction.
Around 200,000 people in the US and Europe are PiZZ homozygotes suffering from severe AATD, which currently requires lifelong intravenous augmentation therapy.
Wave's RNA editor approach aims to edit the disease-causing mutation in the SERPINA1 gene to restore functional AAT protein production.
The news was reported in a press release by Wave Life Sciences and covered by CRISPR Medicine News.
Source: CRISPR Medicine News. This article summarizes the linked reporting and distinguishes announced plans from demonstrated results.