Expert Interview
Examining novel candidates for alpha-1 antitrypsin deficiency, including Beam Therapeutics’ BEAM-302 and Wave Life Sciences’ WVE-006
Ticker(s): WVE, BEAMInstitution - Blessing Health System/ Hannibal Clinic
- Pulmonologist at Blessing Heath Hannibal clinic
- Manages over 400 patients at an Alpha-1 certified center and research center
- Primary research in AATD
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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