BEAM-302
An investigational treatment for Alpha-1 Antitrypsin Deficiency.
The same compound appears under different names depending on the context. Here is how to identify BEAM-302 wherever you encounter it, plus the key facts at a glance.
- Generic name
- BEAM-302
- Development code
- BEAM-302
- Drug class
- In vivo base editing (investigational)
- Manufacturer
- Beam Therapeutics
- How it's taken
- A single intravenous infusion in the trial.
An investigational one-time IV infusion that edits the liver's DNA to correct the most common alpha-1 antitrypsin deficiency mutation, so the liver makes working AAT again. In a Phase 1/2 trial, a single 60 mg dose raised blood AAT from about 5 to about 14 micromolar, above the historical 11 micromolar target level. Not approved.
How BEAM-302 works
Most severe AATD comes from one misspelled letter in the SERPINA1 gene, the PiZ variant. The liver then makes a misfolded protein (Z-AAT) that gets stuck inside liver cells instead of traveling to the lungs. BEAM-302 uses a base editor, a gene-editing tool that swaps a single DNA letter without cutting both strands of DNA, to change the Z letter back to the normal one in liver cells. Corrected cells make normal AAT, which is intended to protect the lungs, and less of the sticky Z-AAT that can damage the liver.
Mechanism: One-time base-editing treatment delivered to the liver in a lipid nanoparticle; it corrects the single DNA letter behind the PiZ (E342K) variant of the SERPINA1 gene
Side effects and safety
In the Phase 1/2 trial update presented in September 2026, infusion reactions occurred in 41% of patients and were mild to moderate. Most liver enzyme increases were Grade 1; one patient with liver disease had a temporary Grade 3 increase in liver enzymes. Long-term effects of permanent DNA editing are still being studied.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking BEAM-302
A single intravenous infusion in the trial. It is designed to be given once rather than weekly like current augmentation therapy.
Availability and cost
Only available as the brand-name product.
Clinical trial results
The Phase 1/2 trial (NCT06389877) is recruiting adults with PiZZ AATD, with lung disease (Part A) or liver disease (Part B). As of August 17, 2026, 38 patients had been dosed. At the 60 mg dose, steady-state total AAT averaged 14.4 micromolar in Part A (from a baseline of 5.0) and 13.5 micromolar in Part B (from 4.7), above 11 micromolar, the level Beam calls a protective threshold; corrected normal AAT made up 93% of circulating AAT, and Z-AAT fell by 84%. Beam said a pivotal cohort of about 50 more patients at 60 mg began dosing in July 2026 and that it plans to seek U.S. accelerated approval based on a primary endpoint of AAT biomarkers evaluated over 12 months.
Development history
Beam Therapeutics presented an updated dataset with up to 18 months of follow-up at the European Respiratory Society congress in September 2026.
Explore Alpha-1 Antitrypsin Deficiency trials
Other Alpha-1 Antitrypsin Deficiency treatments
Common questions about BEAM-302
▸What is BEAM-302?
An investigational one-time IV infusion that edits the liver's DNA to correct the most common alpha-1 antitrypsin deficiency mutation, so the liver makes working AAT again. In a Phase 1/2 trial, a single 60 mg dose raised blood AAT from about 5 to about 14 micromolar, above the historical 11 micromolar target level. Not approved.
▸How does BEAM-302 work?
Most severe AATD comes from one misspelled letter in the SERPINA1 gene, the PiZ variant. The liver then makes a misfolded protein (Z-AAT) that gets stuck inside liver cells instead of traveling to the lungs. BEAM-302 uses a base editor, a gene-editing tool that swaps a single DNA letter without cutting both strands of DNA, to change the Z letter back to the normal one in liver cells. Corrected cells make normal AAT, which is intended to protect the lungs, and less of the sticky Z-AAT that can damage the liver.
▸What are the side effects of BEAM-302?
In the Phase 1/2 trial update presented in September 2026, infusion reactions occurred in 41% of patients and were mild to moderate. Most liver enzyme increases were Grade 1; one patient with liver disease had a temporary Grade 3 increase in liver enzymes. Long-term effects of permanent DNA editing are still being studied.
▸How is BEAM-302 taken?
A single intravenous infusion in the trial. It is designed to be given once rather than weekly like current augmentation therapy.
▸Is BEAM-302 FDA approved?
BEAM-302 is currently in phase 2 clinical trials for Alpha-1 Antitrypsin Deficiency. It has not yet received FDA approval.
▸Is BEAM-302 a cure for alpha-1 antitrypsin deficiency?
Not yet proven. BEAM-302 is an investigational one-time base-editing treatment. In its Phase 1/2 trial, a single 60 mg dose raised average blood AAT from about 5 to about 14 micromolar, above the historical 11 micromolar target level, with up to 18 months of follow-up as of September 2026. Whether it prevents lung or liver damage over the long term has not been shown, and it is not approved.
▸How is BEAM-302 different from augmentation therapy?
Augmentation therapy (Prolastin-C, Aralast NP, Zemaira, Glassia) is a weekly IV infusion of AAT protein collected from donated human plasma. BEAM-302 is designed as a single infusion that edits the liver's own DNA so it produces normal AAT. In its trial it also lowered circulating misfolded Z-AAT by 84%, something augmentation does not do; whether that prevents liver damage is not yet known.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Beam Therapeutics · 2026-09-08. Beam Therapeutics Presents Updated Clinical Data from Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency. https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-presents-updated-clinical-data-phase-12-trial/
- ClinicalTrials.gov. A Study of BEAM-302 in Patients With Alpha-1 Antitrypsin Deficiency. https://clinicaltrials.gov/study/NCT06389877