BEAM-302 for Alpha-1 Antitrypsin Deficiency
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called BEAM-302 for individuals with Alpha-1 Antitrypsin Deficiency (AATD), a condition that can lead to lung and liver problems. The study aims to determine the safety and effectiveness of BEAM-302 and identify the optimal dose. It seeks participants diagnosed with AATD who have specific lung or liver issues, such as emphysema or certain levels of liver fibrosis. Eligible participants should not have undergone a lung or liver transplant or have severe lung or liver conditions. This trial might be suitable for those meeting these criteria. As a Phase 1, Phase 2 trial, the research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.
Is there any evidence suggesting that BEAM-302 is likely to be safe for humans?
Research has shown that BEAM-302 has a good safety record in clinical trials. Patients who took doses up to 75 mg mostly experienced mild to moderate side effects. Importantly, no reports indicated serious side effects or issues that would prevent higher dosing. This suggests that participants tolerated the treatment well. These findings imply that BEAM-302 could be a safe option for treating alpha-1 antitrypsin deficiency, although further research is needed to confirm these results.12345
Why do researchers think this study treatment might be promising?
Most treatments for Alpha-1 Antitrypsin Deficiency rely on augmenting the body's deficient enzyme with infusions of alpha-1 antitrypsin protein derived from human plasma. But BEAM-302 offers something different. It uses gene editing technology to precisely target and correct the faulty gene responsible for the deficiency, potentially providing a more permanent solution. Researchers are excited because this approach could reduce the need for frequent infusions and directly address the root cause of the condition, rather than just managing its symptoms.
What evidence suggests that BEAM-302 might be an effective treatment for Alpha-1 Antitrypsin Deficiency?
Research has shown that BEAM-302 holds promise for treating alpha-1 antitrypsin deficiency (AATD). In studies, a single dose of BEAM-302 increased levels of healthy alpha-1 antitrypsin protein and reduced the harmful mutated protein by 84%. Patients who received 60 mg of BEAM-302 consistently maintained healthy protein levels above the target, averaging 16.1 µM. After treatment, 94% of the alpha-1 antitrypsin in the blood was corrected. These results suggest that BEAM-302 may effectively address the root cause of lung and liver disease related to AATD by improving protein levels.12567
Who Is on the Research Team?
Medical Information
Principal Investigator
Beam Therapeutics
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Phase 1 Dose Exploration
Participants receive BEAM-302 to evaluate safety, tolerability, PK/PD, and efficacy
Phase 2 Dose Expansion
Participants receive BEAM-302 to determine the optimal biological dose
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- BEAM-302
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Find a Clinic Near You
Who Is Running the Clinical Trial?
Beam Therapeutics Inc.
Lead Sponsor
Citations
1.
investors.beamtx.com
investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-compelling-updated-clinical-data/Press Release
Treatment with 60 mg of BEAM-302 Led to Mean Steady-state Total AAT Level of 16.1 µM and All Patients Consistently and Durably Above the 11 ...
Beam Therapeutics Announces Update on BEAM-302
Mutant Z-AAT was durably and significantly reduced after treatment with BEAM-302. The steady-state mean reduction in Z-AAT was 84% in the 60 mg ...
A cure for alpha-1? Novel therapeutics in alpha-1 antitrypsin ...
In early results from an ongoing phase 1/2 clinical trial (NCT06389877), a single intravenous dose of BEAM-302 led to durable, dose-dependent increases in total ...
A Study to Evaluate the Safety and Efficacy of BEAM-302 in ...
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD) · Overview · Trial locations.
5.
crisprmedicinenews.com
crisprmedicinenews.com/news/beam-therapeutics-reports-clinical-proof-of-concept-data-for-beam-302-in-alpha-1-antitrypsin-deficie/Beam Therapeutics Reports Clinical Proof-Of-Concept ...
In patients with alpha-1 antitrypsin deficiency, a single treatment of BEAM-302 increased the levels of healthy protein and reduced harmful mutated protein by ...
6.
investors.beamtx.com
investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-reports-first-quarter-2026-financial-results/Beam Therapeutics Reports First Quarter 2026 Financial ...
Recent BEAM-302 Topline Data in Alpha-1 Antitrypsin Deficiency (AATD) Demonstrate Strong Single-dose Safety and Efficacy Profile, with 60 mg ...
Initial Clinical Data for BEAM-302 in Alpha-1 Antitrypsin ...
302 in alpha-1 antitrypsin deficiency. (AATD) → Initial data today. • Potential one-time treatment for. AATD lung and liver manifestations.
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