Beam Therapeutics said it agreed with FDA reviewers that validated biomarkers assessed over a year could support an accelerated approval pathway for BEAM‑302, its in vivo base‑editing candidate for alpha‑1 antitrypsin deficiency (AATD). The arrangement enables the company to rely on surrogate functional biomarkers as near‑term evidence of clinical benefit. Beam’s CEO framed the approach as a classic accelerated‑approval strategy: secure earlier market access based on biomarkers, then complete confirmatory outcomes studies. BEAM‑302 employs base editing delivered via liver‑targeting lipid nanoparticles to correct the PiZ mutation underlying many severe AATD cases. Regulatory acceptance of biomarker endpoints for gene editing would mark a material shift for the space and could shorten time‑to‑market for other genome‑editing programs that establish robust surrogate–outcome linkages.
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