Beam Therapeutics Inc.

This study tests a new gene therapy called BEAM-301 in 36 adults with a specific form of glycogen storage disease type Ia. The goal is to see if it is safe and can help control blood sugar levels, reducing the need for frequent cornstarch doses. Participants must have a confirmed…

This study tests a new gene editing medicine called BEAM-302 in 106 adults with Alpha-1 antitrypsin deficiency, a genetic condition that can cause lung and liver damage. The goal is to see if the treatment is safe and can raise levels of a protective protein in the blood. Partici…

This study follows 50 people with sickle cell disease who received the gene therapy BEAM-101 in an earlier trial. Researchers will monitor them for 13 years to check for long-term side effects, new health problems, and how well the treatment controls their disease. The goal is to…

This early-stage study tests the safety of a new drug, BEAM-103, in 30 healthy adults aged 18 to 55. Participants receive a single infusion of the drug or a placebo and are monitored for up to 4 months. The goal is to gather safety and dosing information, not to treat any disease…