Beam Therapeutics

Beam Therapeutics specializes in gene editing to develop novel precision therapeutics for a variety of genetic diseases using a suite of gene editing technologies including base editing, nuclease editing, ribonucleic acid (RNA) editing, and prime editing. The company’s base editing technology targets a single base in the genome, without creating unnecessary DNA modifications, thereby designing precisely targeted therapies. It has also obtained an exclusive license for the prime editing technology from Prime Medicine. The technology was developed by David Liu (co-founder of Beam and Prime) and was intended for Beam’s sickle cell disease indication.

Beam develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. Its pipeline (as of March 2024) is classified based on the company’s delivery methods and consists of two ex-vivo programs based on hematopoietic stem cells (HSC), one ex-vivo programs based on T cells, and four in-vivo programs based on lipid nanoparticles (LNP). Two of the programs. “BEAM-101” and “BEAM-201”, are under Phase I/II clinical trials for the treatment of sickle-cell disease. In August 2023, “BEAM-201” was dosed to the first patient with T (CAR T)-cell therapy for relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma (T-ALL/T-LL).

Beam acquired biotechnology company Guide Therapeutics for an undisclosed amount in February 2021 to leverage its non-viral drug delivery technologies for genetic medicines. It owns a good manufacturing practices (GMP)-standard manufacturing facility, which was expected to open in 2023.

In October 2023, Beam agreed to sell its opt-in rights to Verve Therapeutics’ Base Editing Programs for Cardiovascular Disease to Eli Lilly and Company for a combined USD 250 million in combined upfront payment and equity investment. Under the terms of the agreement, Beam received USD 200 million in an upfront payment and a USD 50 million equity investment. Beam was also eligible to receive up to USD 350 million in potential future development-stage payments upon the completion of certain clinical, regulatory and alliance events for a total of up to USD 600 million in potential total deal consideration.

Key customers and partnerships

The company secured a four-year research partnership with big pharma Pfizer in January 2022 to develop in vivo base editing therapeutics for three undisclosed rare genetic diseases of the liver, muscle, and central nervous system. Similarly, in June 2021, it signed a five-year research partnership with Apellis Pharmaceuticals to conduct six programs for complement-driven diseases.

Beam’s other partnerships include a collaboration and licensing agreement with Verve Therapeutics (2019) to develop therapeutics for cardiovascular diseases; a research collaboration with Institute of Molecular and Clinical Ophthalmology Basel (IOB) (2020) to explore base editing therapies for ocular diseases; option and license agreement with Sana Biotechnology (2021) to license its CRISPR-Cas12b technology; and a non-exclusive research and clinical trial collaboration with Magenta Therapeutics (2020). 

Funding and financials

Beam Therapeutics went public on the Nasdaq Global Select Market in February 2020, raising gross proceeds of USD 207 million. The net proceeds were earmarked for the advancement of Beam’s clinical development pipeline toward IND-enabling stage and potential clinical trials. For 2021, it recorded revenue of USD 51.8 million, earned through licensing and collaborations.