Wave Life Sciences submits first clinical application for RNA editing candidate

Wave Life Sciences submitted its first clinical trial application for WVE-006 in alpha-1 antitrypsin deficiency, or AATD. WVE-006 is a first-in-class, GalNAc-conjugated RNA editing oligonucleotide and is designed to correct the single base mutation in messenger RNA coded by the SERPINA1 Z allele, thereby enabling restoration and circulation of functional, wild-type alpha-1 antitrypsin protein. Wave expects to initiate dosing in healthy volunteers in 4Q23 and deliver proof-of-mechanism data in individuals with AATD in 2024. Earlier this year, Wave commenced its strategic collaboration with GSK to advance transformative RNA medicines using Wave’s multimodal RNA platform, including WVE-006. Wave received $170M in upfront cash and equity and is also receiving research funding. Wave is eligible for up to $3.3B in potential milestone payments, including near-term preclinical and clinical milestones, as well as royalties, for WVE-006 and GSK’s eight collaboration programs. For WVE-006, Wave is eligible to receive up to $225M in development and launch milestone payments, and up to $300,M in sales-related milestone payments, as well as double-digit tiered royalties as a percentage of net sales up to the high teens. Wave expects that its cash and cash equivalents will be sufficient to fund operations into 2025.