Pfizer Exercises Option for Beam’s Liver-Targeted Base Editing Program

Pfizer has exercised its option to secure global rights to a liver-targeted in vivo base editing candidate from Beam Therapeutics, marking a notable return to genetic medicine after stepping back from gene therapy last year.

The move follows Pfizer’s discontinuation of its hemophilia gene therapy Beqvez in early 2025, less than a year after FDA approval. Despite that exit, the New York pharma giant has now opted to advance an unnamed liver disease program developed under its four-year collaboration with Beam Therapeutics.

The original 2021 agreement included a $300 million upfront payment and up to $1.05 billion in potential milestone payments tied to candidate selection and development progress. The collaboration focused on in vivo base editing programs using Beam’s proprietary technology platform.

Under the newly exercised option, Pfizer will assume responsibility for development, manufacturing and potential commercialization of the liver-targeted candidate. The therapy uses Beam’s lipid nanoparticle (LNP) formulation to deliver base editing reagents directly to hepatocytes, enabling precise gene correction without introducing double-strand DNA breaks.

Beam retains the right to opt into a global codevelopment and co-commercialization agreement after completion of Phase 1/2 trials. If exercised, Pfizer would cover 65% of development costs and receive 65% of profits, with Beam sharing the remainder.

The announcement comes amid evolving regulatory dynamics, including new draft guidance from the FDA aimed at accelerating development pathways for genome editing and RNA-based therapies targeting rare diseases.

In a separate disclosure, Beam also announced a $500 million loan agreement with Sixth Street, strengthening its financial position ahead of a planned FDA submission for risto-cel, its investigational sickle cell disease therapy. Analysts at William Blair noted that the financing should extend Beam’s runway into mid-2029 and support continued investment in its in vivo pipeline.

Pfizer’s decision signals renewed confidence in precision gene editing approaches—particularly liver-directed programs leveraging LNP delivery—as the field moves beyond traditional AAV gene therapy strategies.