BEAM

Beam Therapeutics Inc. is a leading biotechnology company pioneering precision genetic medicines through base editing, a novel CRISPR-based technology that enables single nucleotide changes in DNA and RNA without making double-stranded breaks. Headquartered in Cambridge, Massachusetts, Beam was founded by leaders in genome editing including CRISPR pioneers and has built a comprehensive platform for developing transformative genetic medicines. Base editing represents a potentially safer and more precise form of gene editing compared to traditional CRISPR-Cas9, which cuts both strands of DNA. Beam's technology can make four types of single nucleotide changes: adenine to guanine (A→G) and cytosine to thymine (C→T) in DNA, and adenine to inosine (A→I, read as G) and cytosine to uracil (C→U, read as T) in RNA. These capabilities enable correction of disease-causing mutations or introduction of beneficial genetic changes without generating double-strand DNA breaks that could cause unintended chromosomal rearrangements. Beam's pipeline includes programs for sickle cell disease, beta-thalassemia, alpha-1 antitrypsin deficiency, glycogen storage disease type 1a, T cell cancers, and other genetic diseases. The company's lead programs are advancing through clinical development with encouraging safety and preliminary efficacy data. Beam is developing both ex vivo therapies (where cells are edited outside the body and reinfused) and in vivo therapies (where editing components are delivered directly into the body). The company has established strategic partnerships with pharmaceutical companies including Verve Therapeutics for cardiovascular disease programs. Beam benefits from its foundational intellectual property position, experienced management and scientific team, and comprehensive platform that addresses a broad range of genetic diseases. The company aims to deliver curative genetic medicines for patients with serious diseases caused by single nucleotide mutations.