Wave Life Sciences Ltd. has announced the approval of its first clinical trial application (CTA) for the RestorAATion-2 trial of WVE-006, a novel RNA editing oligonucleotide designed to treat alpha-1 antitrypsin deficiency (AATD). This approval signifies a significant milestone for both the company and the AATD community. WVE-006, which is administered subcutaneously and employs GalNAc conjugation without the use of a lipid nanoparticle delivery system, presents a promising therapeutic approach.

Wave Life Sciences, highlighted the importance of this milestone, emphasizing WVE-006's potential to address both AATD liver and lung diseases. The rapid progression in dosing healthy volunteers allowed for the swift identification of a starting dose level expected to effectively engage the target in patients. The RestorAATion-2 trial, a Phase 1b/2a study, will evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of WVE-006 in individuals with AATD carrying the homozygous Pi*ZZ mutation.

Wave remains dedicated to advancing its RNA editing pipeline, utilizing its proprietary "edit-verse" to efficiently identify new targets and edit sites. With GSK holding the exclusive global license for WVE-006, development and commercialization responsibilities will transition post the completion of the RestorAATion-2 study.

The preclinical data for WVE-006 demonstrate its potential in addressing AATD-related liver and lung diseases through potent and specific editing of the SERPINA1 Z transcript. This advancement represents a step forward in the field of RNA medicines and offers hope for patients grappling with AATD.

 

Source: globenewswire.com

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