Drugs / BEAM-302
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BEAM-302

Developed for
alpha 1-antitrypsin deficiency
Investigated by
Beam Therapeutics Inc.

Trials 1

2025202620272028
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1/2 NCT06389877 Jun 2024 → May 2028 expected alpha 1-antitrypsin deficiency Beam Therapeutics Inc. Recruiting No outcome recorded

News releases announcing trial results or a regulatory action · 16

DateIssuerRelease
2026-09-08 Beam Therapeutics Inc. Results Beam Therapeutics Presents Updated Clinical Data from the Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at the European Respiratory Society (ERS) Congress 2026 beamtx.com ↗
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced updated clinical data from the Phase 1/2 trial evaluating BEAM-302 for alpha-1 antitrypsin deficiency (AATD).
2026-05-18 Beam Therapeutics Inc. Results Beam Therapeutics Presents Recently Reported Topline Clinical Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at the American Thoracic Society (ATS) 2026 International Conference beamtx.com ↗
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 1/2 trial in alpha-1 antitrypsin deficiency (AATD) at a symposium on translating scientific discovery in gene editing into clinical progress for patients with lung disease.
2026-05-07 Beam Therapeutics Inc. Results Beam Therapeutics Reports First Quarter 2026 Financial Results and Recent Business Updates beamtx.com ↗
Recent BEAM-302 Topline Data in Alpha-1 Antitrypsin Deficiency (AATD) Demonstrate Strong Single-dose Safety and Efficacy Profile, with 60 mg Selected as Optimal Biological Dose; Global Pivotal Cohort Expected to Initiate in Second Half of 2026
2026-03-25 Beam Therapeutics Inc. Results Beam Therapeutics Announces Compelling Updated Clinical Data from the Ongoing Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) to Support Advancement to Pivotal Development beamtx.com ↗
2026-01-11 Beam Therapeutics Inc. Regulatory Beam Therapeutics Sets Strategic Priorities for its Genetic Disease and Hematology Franchises to Drive Execution of Late-Stage Clinical Programs and Extends its Operating Runway through Commercial Transition beamtx.com ↗
BEAM-302 has also been accepted into the FDA’s Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) program aimed at facilitating CMC development of products with expedited clinical development timeframes.
2025-08-05 Beam Therapeutics Inc. Results Beam Therapeutics Reports Second Quarter 2025 Financial Results and Provides Update on BEAM-302 Development Progress in Alpha-1 Antitrypsin Deficiency (AATD) beamtx.com ↗
With 17 Patients Dosed in the Phase 1/2 Trial, BEAM-302 Continues to Demonstrate Durable Correction of the Disease-causing Mutation, Restoration of AAT Physiology, and a Well Tolerated Safety Profile
2025-05-29 Beam Therapeutics Inc. Regulatory Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) beamtx.com ↗
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD).
2025-05-12 Beam Therapeutics Inc. Regulatory Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) beamtx.com ↗
2025-05-06 Beam Therapeutics Inc. Regulatory Beam Therapeutics Reports First Quarter 2025 Financial Results and Recent Business Highlights beamtx.com ↗
Building on this momentum, we have swiftly advanced the program—initiating the fourth cohort of Part A of the Phase 1/2 BEAM-302 study and securing U.S. FDA clearance for our investigational new drug application, positioning us for continued rapid progress,
2025-04-05 Beam Therapeutics Inc. Results Beam Therapeutics Presents Additional Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency (AATD) at 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress beamtx.com ↗
These previously reported data were included in today’s presentation, alongside updated biomarker data from the 60 mg cohort showing levels of corrected protein (M-AAT) and the mutant form of alpha-1 antitrypsin protein (Z-AAT) out to Day 28 for all three patients.
2025-03-27 Beam Therapeutics Inc. Regulatory Beam Therapeutics Announces Clearance of Investigational New Drug Application for BEAM-302 for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) by the United States (U.S.) Food and Drug Administration beamtx.com ↗
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for BEAM-302 for the treatment of alpha-1 antitrypsin deficiency (AATD).
2025-03-10 Beam Therapeutics Inc. Results Beam Therapeutics Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (AATD), Demonstrating First Ever Clinical Genetic Correction of a Disease-causing Mutation beamtx.com ↗
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced initial safety and efficacy data from its Phase 1/2 trial of BEAM-302, establishing clinical proof-of-concept as a potential treatment for alpha-1 antitrypsin deficiency (AATD) and for in vivo base editing.

All press releases naming this drug 19 releases

DateIssuerRelease

Evidence & citations 1 cited value

Every value below carries the sentence it was read from. 1 source stands behind the page.

FieldValueCited text
Known as BEAM-302 ClinicalTrials.gov intervention name — accepted as the source's own label NCT06389877 ↗