Beam Therapeutics Inc. (BEAM)

Overview

Beam Therapeutics Inc. is a pioneering biotechnology company focused on advancing the field of genetic medicines to address the needs of patients with serious diseases. Founded in 2017 and headquartered in Cambridge, Massachusetts, Beam Therapeutics stands at the forefront of developing innovative therapies using precise genome editing technologies. The company’s commitment to this mission is evidenced by its active engagement in several strategic collaborations with leading pharmaceutical and biotechnology companies including Pfizer Inc., Apellis Pharmaceuticals, Verve Therapeutics, Inc., Sana Biotechnology, Inc., and Orbital Therapeutics. These partnerships are aimed at leveraging in vivo base editing for targeting rare genetic diseases affecting the liver, muscle, and central nervous system; identifying gene targets within the complement system; enhancing treatments for cardiovascular disease; exploring CRISPR Cas12b for cell therapy programs; and designing RNA for the prevention, diagnosis, or treatment of human diseases, respectively.

Products and Services

Beam Therapeutics Inc. is actively developing a diverse portfolio of precision genetic medicines, including:

• BEAM-101: This forefront candidate is being developed as a potential treatment for sickle cell disease or beta-thalassemia, aiming to address the genetic root causes of these serious hematologic conditions.
• BEAM-302: A liver-targeting lipid nanoparticle (LNP) formulation designed to tackle severe alpha-1 antitrypsin deficiency, a genetic disorder that can cause liver and lung disease.
• BEAM-201: An anti-CD7 CAR-T product candidate currently in Phase 1/2 clinical trials for refractory T-cell acute lymphoblastic leukemia/T cell lymphoblastic lymphoma, showcasing Beam’s commitment to innovating in the field of oncology with targeted cellular therapies.
• BEAM-301: Another liver-targeting LNP formulation, this time for the treatment of glycogen storage disease 1a, a genetic disorder that prevents the body from properly storing and utilizing glucose, leading to a variety of health issues.

In addition to product development, Beam Therapeutics Inc. collaborates on research initiatives aimed at pioneering new treatment paradigms. These include:

• Research collaboration with Pfizer Inc.: Focused on advancing in vivo base editing programs for rare genetic diseases of the liver, muscle, and central nervous system.
• Apellis Pharmaceuticals partnership: To conduct preclinical research targeting specific genes within the complement system across various organs, including the eye, liver, and brain.
• Verve Therapeutics, Inc. collaboration: Aimed at developing treatments for cardiovascular diseases.
• Sana Biotechnology, Inc. agreement: For the research, development, and commercialization rights to CRISPR Cas12b in cell therapy programs.
• Orbital Therapeutics design: Focused on RNA design for the prevention, treatment, or diagnosis of human diseases, augmenting the capabilities and reach of genetic medicine.