New research results show that a new form of gene editing is efficient in correcting a mutation in patient cells with the monogenic disease Alpha-1 antitrypsin deficiency (AATD), a common inherited ...
AIRNA is expected to file clinical trial application for optimized product candidate (AIR-001) with potential best-in-class profile for AATD in 2H 2025 CAMBRIDGE, Mass. &, TÜBINGEN, Germany, May 13, ...
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today presented the recently reported clinical data from the BEAM-302 Phase ...
Recent studies attempted to improve the risk of recurrence while reducing the risk of adverse effect Alvelestat is an investigational oral neutrophil elastase inhibitor designed to protect AATD ...
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Beam Therapeutics (BEAM) Is Up 17.6% After Advancing BEAM-302 Toward Accelerated Approval in AATD
Earlier in May 2026, Beam Therapeutics presented updated Phase 1/2 BEAM-302 data in alpha-1 antitrypsin deficiency, including detailed safety, durability and human neutrophil elastase findings, and ...
Potential best-in-class therapeutic delivered subcutaneously (GalNAc-conjugated oligonucleotide) for a debilitating genetic condition impacting ...
Chronic obstructive pulmonary disease (COPD) remains highly prevalent among veterans, but the contribution of alpha-1 antitrypsin deficiency (AAT deficiency or AATD) to this patient population remains ...
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