Beam Therapeutics is a clinical-stage biotech developing genetic medicines based on proprietary base editing technology. Base editing chemically converts a single DNA base at a targeted genomic location without cutting both strands of DNA — Beam describes this as a "pencil" approach that is more precise and predictable than conventional CRISPR-based gene editing. Beam has no approved products and generates no product revenue; it funds itself through equity raises and partnership deals. Beam's lead program is risto-cel, an ex vivo cell therapy for sickle cell disease in which patient stem cells are edited outside the body to increase fetal hemoglobin production and then reinfused. Beam is targeting a BLA submission for risto-cel as early as year-end 2026. Beyond risto-cel, Beam is developing a pipeline of in vivo therapies delivered via lipid nanoparticles, targeting the liver: BEAM-302 for alpha-1 antitrypsin deficiency (AATD), BEAM-301 for glycogen storage disease type 1a, and BEAM-304 for phenylketonuria. If approved, Beam's therapies would likely be priced as high-cost, potentially one-time curative treatments targeting rare disease populations. Beam also licenses its base editing platform to pharma partners — including Pfizer, Eli Lilly (via Verve Therapeutics), and Apellis — in exchange for upfront fees, milestones, and royalties. Beam operates a cGMP manufacturing facility in Research Triangle Park, NC to support clinical and eventual commercial supply.