Beam Therapeutics
Beam Therapeutics works on 2 rare diseases tracked on Trial Friend, including Alpha-1 Antitrypsin Deficiency, Sickle Cell Disease, with 4 active clinical trials (3 recruiting).
Beam Therapeutics develops base-editing therapies, a precision gene-editing approach that makes single-letter DNA changes without cutting both DNA strands. Risto-cel (formerly BEAM-101), a base-edited cell therapy made from the patient's own stem cells for sickle cell disease, completed dosing in the Phase 1/2 BEACON trial, and Beam plans to file for FDA approval as early as the end of 2026. BEAM-302 for alpha-1 antitrypsin deficiency began dosing a pivotal cohort in July 2026 that Beam says could support accelerated approval. BEAM-301 for glycogen storage disease type 1a is in a Phase 1/2 trial.
Beam Therapeutics Drug Pipeline
Beam Therapeutics has 4 active clinical trials across 2 development stages, with 3 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Beam Therapeutics Clinical Trials (4)
Active and recruiting clinical trials sponsored by Beam Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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FDA-Approved Drugs
Beam Therapeutics Trial Locations
Beam Therapeutics clinical trials are running at 51 sites in 6 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (2)
Diseases targeted by Beam Therapeutics's clinical trial and drug development programs
Alpha-1 antitrypsin deficiency is a genetic disorder affecting the lungs and liver, caused by insufficient production of the protective protein alpha-1 antitrypsin. Without adequate protection, neutro...
Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion. Thi...
Patient Resources
Organizations and resources related to Beam Therapeutics's rare disease focus areas
Frequently Asked Questions About Beam Therapeutics
Common questions about Beam Therapeutics's rare disease programs, clinical trials, and treatments.