Jenny Leigh Gonzalez-Armenta's questions to WAVE Life Sciences Ltd (WVE) leadership • Q1 2025
Question
Jenny Leigh Gonzalez-Armenta from Leerink Partners asked about the potential benefits of exceeding the 11 micromolar AAT protein threshold in the Alpha-1 program and the key differentiators between RNA and DNA editing from a patient's perspective.
Answer
President and CEO Dr. Paul Bolno stated that exceeding the 11 micromolar threshold could offer additional benefits, such as faster clearance of liver aggregates, and that upcoming multi-dose data will be crucial. He contrasted RNA editing with DNA editing by highlighting the risks of bystander edits and misfolded proteins with DNA-based approaches. He also noted a key patient-centric advantage: Wave's GalNAc-conjugated WVE-006 avoids the need for LNP delivery, which carries a risk of hepatic injury, a significant concern for patients with liver disease.