Expert Interview
A Second View: Examining novel candidates for alpha-1 antitrypsin deficiency, including Beam Therapeutics’ BEAM-302 and Wave Life Sciences’ WVE-006
Ticker(s): WVE, BEAMInstitution: University College Dublin
- Associate Professor of Pulmonology and rare lung disease specialist at University College Dublin School of Medicine; Formerly at University of Cincinnati Medical Center and Cincinnati Children's Hospital
- Manages 100 patients a year with AATD
- Research focuses on Pulmonary alveolar proteinosis, alpha-1 antitrypsin deficiency, alveolar macrophage function, surfactant lung diseases, cystic fibrosis, and nocardia infection with a current interest in the role of cholesterol in the pathogenesis of surfactant lung diseases.
How do you currently manage AATD in clinical practice (lung vs liver manifestations), and what are the biggest unmet needs that today’s augmentation therapy and supportive care do not address?
Added By: sara_adminWhat is your interpretation of the BEAM-302 Phase 1/2 biomarker data (e.g., corrected M-AAT levels, proportion of corrected protein, Z-AAT reduction), and what threshold would you consider “clinically meaningful” for improving outcomes?
Added By: sara_adminHow compelling is the WVE-006 RestorAATion-2 update showing durable serum AAT production with repeat dosing, and how do you view RNA editing as a modality compared with gene editing/base editing for AATD?
Added By: sara_adminAre You Interested In These Questions?
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