Beam Therapeutics
Beam Therapeutics works on 2 rare diseases tracked on Trial Friend, including Alpha-1 Antitrypsin Deficiency, Sickle Cell Disease, with 3 recruiting clinical trials.
Beam Therapeutics develops base-editing therapies, a precision gene-editing approach that makes single-letter DNA changes without cutting both DNA strands. BEAM-101, a base-edited autologous cell therapy for sickle cell disease, is in Phase 1/2 (BEACON trial) with FDA RMAT designation. BEAM-302 for alpha-1 antitrypsin deficiency is also in Phase 1/2, with clinical proof-of-concept showing durable mutation correction.
Focus areas at Beam Therapeutics
As a rare disease specialist, Beam Therapeutics has active clinical trial programs and drug development efforts across 2 rare diseases, including Alpha-1 Antitrypsin Deficiency, Sickle Cell Disease. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Beam Therapeutics, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Beam Therapeutics is headquartered in Cambridge, United States, founded in 2017, publicly traded under the ticker symbol BEAM. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Beam Therapeutics Drug Pipeline
Beam Therapeutics has 3 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 includes all of Beam Therapeutics's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Beam Therapeutics Clinical Trials (3)
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.
FDA-Approved Drugs
Beam Therapeutics Trial Locations
Beam Therapeutics clinical trials are running at 47 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 enzyme alpha-1 antitrypsin. Without adequate protection, neutrop...
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.