Beam Therapeutics Inc.

This early-stage study is testing a new gene therapy called BEAM-302 in adults with Alpha-1 antitrypsin deficiency, a genetic condition that can damage the lungs and liver. The main goals are to find a safe dose and see if the treatment can increase levels of a missing protective…

This early-stage study is testing a single dose of an experimental gene therapy called BEAM-301 in adults with a specific genetic form of glycogen storage disease type Ia (GSDIa). The main goals are to see if the treatment is safe and to find the best dose. Researchers will measu…

This study will follow patients for 15 years after they received an experimental gene-editing treatment (BEAM-101) for sickle cell disease or similar blood disorders. The main goal is to monitor their long-term health and safety, and see how well the treatment continues to work o…

This is an early-stage study to test the safety of a new drug called BEAM-103 in healthy people. Researchers will give single, increasing doses to small groups of volunteers to see how their bodies react and process the drug. The main goal is to find a safe dose range for future …