Beam Therapeutics Inc.

Beam Therapeutics Inc. (NASDAQ: BEAM) is a biotechnology company committed to establishing an integrated platform for precision genetic medicines. The company's website is www.beamtx.com. Beam focuses on developing life-long curative therapies for patients with serious genetic diseases through its proprietary base editing technology, which enables precise modification of single DNA bases without creating double-stranded breaks in the genome.

The company's core technology platform combines base editing capabilities with advanced delivery modalities and internal manufacturing infrastructure. Base editing represents a differentiated approach to gene editing that allows for highly precise and predictable genetic outcomes across both dividing and non-dividing cells. Beam's platform includes cytosine base editors and adenine base editors, each utilizing engineered deaminase enzymes to create specific chemical modifications at targeted genomic locations.

Beam's clinical pipeline spans hematology and genetic disease indications. The lead program, ristoglogene autogetemcel (risto-cel), is an autologous hematopoietic stem cell therapy in Phase 1/2 development for sickle cell disease, with a biologics license application expected as early as year-end 2026. BEAM-302, a liver-targeting lipid nanoparticle formulation, targets alpha-1 antitrypsin deficiency and has reached alignment with the FDA on an accelerated approval pathway. Additional programs include BEAM-301 for glycogen storage disease type 1a, BEAM-304 for phenylketonuria, and BEAM-103, an anti-CD117 monoclonal antibody supporting the company's ESCAPE platform.

Beam operates a 100,000 square foot current Good Manufacturing Practice facility in Research Triangle Park, North Carolina, designed to support manufacturing for both ex vivo cell therapy and in vivo non-viral delivery programs. The company has established strategic collaborations with major pharmaceutical partners including Pfizer, Apellis Pharmaceuticals, Eli Lilly and Company, and Bristol Myers Squibb, leveraging its technology platform across multiple therapeutic areas and disease targets beyond its core focus areas.