Beam Therapeutics

This is a Phase 1/2, multicenter, open-label study conducted in 3 parts (Part A, Part B, and Part C) to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease. Parts A and B are designed to evaluate safety and tolerability and identify the optimal biological dose (OBD) in patients with AATD-associated lung disease (Part A) and/or liver disease (Part B), with optional expansion cohorts to further evaluate a potential OBD. Part C is designed to demonstrate the efficacy and safety of BEAM-302 at the OBD in patients with AATD-associated lung disease with or without liver disease in the pivotal cohort.Show more

This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing Study BTX-AUT-001. Patients who enter into this study will be followed for 13 years (a total of 15 years after receiving BEAM-101).Show more

This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygous for the G6PC1 c.247C\>T (p.R83C) variant and to determine the optimal biological dose.Show more

This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD

Base editing changes a single DNA letter in place; standard CRISPR cuts the DNA.

CRISPR: cuts both strands of DNA and relies on the cell to repair the break.

Base editing: uses a disabled CRISPR enzyme to carry a chemical editor to one spot and swap one letter, without cutting both strands.

Why it matters: diseases caused by a single-letter mutation, which is why Beam's lead programs are in sickle cell disease and alpha-1 antitrypsin deficiency.

A one-time infusion that edits the liver's DNA to correct the Z mutation so the liver makes working AAT protein again.

Results: at the 60mg dose, blood AAT rose from about 5 to about 14 micromolar, above the 11 micromolar level associated with protection from lung disease, and 93% of the circulating AAT was the corrected normal form.

Next: a pivotal cohort of about 50 more patients at 60mg began dosing in July 2026, Beam said on November 3, 2026 that it expects to finish dosing that cohort in the first half of 2027, and Beam plans to seek accelerated approval on AAT biomarkers measured over 12 months.

Source: Beam's updated Phase 1/2 data, September 8, 2026 and the BEAM-302 drug page. Our alpha-1 treatments compared post places it against the weekly infusions and the RNA drugs.

Risto-cel, short for ristoglogene autogetemcel and formerly called BEAM-101, is a base-edited version of the patient's own blood stem cells, and Beam expects to submit its application for FDA approval as early as the end of 2026.

Process: the cells are collected, edited to switch fetal hemoglobin back on, and returned after chemotherapy conditioning, the same overall path as the approved gene therapies Casgevy and Lyfgenia.

Trial: the Phase 1/2 BEACON trial (NCT05456880) has finished dosing its adult and adolescent patients and is no longer recruiting.

Pivotal result: Beam said on November 10, 2026 that BEACON met its main goal, no severe pain crises for at least 12 months in a row, at a planned interim analysis, and met all its main safety goals and all its secondary efficacy goals. By the October 20, 2026 data cutoff, 49 patients had been treated, 39 adults and 10 adolescents, and 31 had been followed long enough to be judged on the main goal. Beam has not yet said how many of those 31 reached it, and plans to present detailed results at a medical meeting in 2027.

Earlier results: in The New England Journal of Medicine in April 2026, an early look at 31 treated patients followed for an average of 6.6 months found fetal hemoglobin above 60% of total hemoglobin on average at 6 months in the 13 patients measured then, and no severe pain crises reported starting more than 60 days after the last blood transfusion.

Safety: a patient died from idiopathic pneumonia syndrome, a lung complication, and Beam has said the trial doctor judged that death likely related to the busulfan chemotherapy given before infusion, not to the edited cells. Of the 31 patients, 87% had a side effect of grade 3 or higher and 39% had a serious side effect.

FDA status: orphan drug and Regenerative Medicine Advanced Therapy designations.

How it differs: risto-cel changes single DNA letters in the HBG1 and HBG2 genes so the BCL11A protein can no longer switch fetal hemoglobin off there, while Casgevy cuts both DNA strands in a BCL11A control region to lower the amount of BCL11A in red blood cell precursors; no trial has compared the 2.

Source: Beam's November 10, 2026 release, Gupta et al., New England Journal of Medicine, 2026, Beam's April 1, 2026 release and Beam's December 7, 2024 release.

No. Beam is a clinical-stage company.

Founded: 2017, in Cambridge, Massachusetts.

Ticker: BEAM on the Nasdaq.

Most advanced programs: risto-cel for sickle cell disease, which Beam said on November 10, 2026 met the main goal of its pivotal trial, with an FDA filing planned as early as the end of 2026, and BEAM-302 for alpha-1 antitrypsin deficiency, now dosing a pivotal cohort aimed at accelerated approval.

Source: Beam's second quarter 2026 results, August 4, 2026 and Beam's November 10, 2026 release.

Start from the disease page, then the trial record.

Recruiting trials: the list on this page comes live from ClinicalTrials.gov, and the sickle cell disease and alpha-1 antitrypsin deficiency pages show eligibility rules and site locations.

What to expect: gene editing trials have strict entry criteria and a long screening process; our guide to what happens after you express interest is a useful read before contacting a site.

None as of November 2026. Beam Therapeutics has no FDA-approved drug yet; its programs are still in trials.

Most advanced: BEAM-302 (Phase 2).

Where to watch: the FDA calendar lists any Beam Therapeutics application once a decision date is public, and the trials list on this page shows what is recruiting now.