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A Phase 1/2 Dose-exploration and Dose-expansion Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and/or Liver Disease

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Trial Profile

A Phase 1/2 Dose-exploration and Dose-expansion Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and/or Liver Disease

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 10 Jun 2026

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At a glance

  • Drugs BEAM 302 (Primary)
  • Indications Alpha 1-antitrypsin deficiency
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Sponsors Beam Therapeutics

Most Recent Events

  • 18 May 2026 According to Beam Therapeutics media release, company expects to present detailed and updated BEAM-302 data at a medical congress in 2026.
  • 18 May 2026 According to Beam Therapeutics media release, top-line clinical data from this study were presented at the American Thoracic Society (ATS) International Conference being held in Orlando, Fla.
  • 25 Mar 2026 According to Beam Therapeutics media release, to support a future biologics licensing application (BLA) submission, the company anticipates enrolling approximately 50 additional patients with AATD-associated lung disease, with or without liver disease, in an expansion of the ongoing open-label Phase 1/2 trial. Beam expects to initiate the pivotal cohort in the second half of 2026.

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