Beam secures USD 500m financing as Pfizer licenses in vivo base editing candidate

Beam Therapeutics announced a USD 500 million strategic financing alongside Pfizer’s opt-in to an exclusive global license for a liver-targeted in vivo base editing development candidate originating from the companies’ four-year research collaboration.

Financing for risto-cel

The financing is based on an agreement with investment firm Sixth Street for long-term, non-dilutive capital in the form of senior secured credit facility, focused on funding the potential launch of ristoglogene autogetemcel (risto-cel) in sickle cell disease (SCD). The USD 500 million facility includes USD 100 million provided at the close of the deal, up to USD 300 million contingent upon achievement of certain clinical, regulatory, and commercial milestones for risto-cel, and a further USD 100 million available to Beam as an option. The financing deal has a seven-year term.

The prospective sickle cell therapy risto-cel is an investigational one-time therapy consisting of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that have been base-edited in the promoter regions of the HBG1/2 genes and are administered via a hematopoietic stem cell transplant procedure. The aim is to induce pancellular fetal hemoglobin expression in sickle cell disease by disrupting BCL11A binding at the HBG promoter. The molecule is currently being evaluated in the Phase I/II BEACON trial, while Beam has said it has completed BLA readiness discussions with the US with the potential for an approval filing later this year. Risto-cel is wholly owned by Beam and being developed independently.

Pfizer’s option exercise

Pfizer is acting to option the Beam-discovered drug candidate following the December 2025 completion of the companies’ four-year research collaboration, which was initiated in 2021 to advance in vivo base editing programs across multiple therapeutic areas using lipid nanoparticle (LNP) delivery technologies.

Pfizer has opted in on a candidate that utilizes Beam’s proprietary liver-targeting LNP platform to deliver base editing reagents designed to enable precise genetic modification in hepatocytes. Under the agreement, Pfizer will assume responsibility for all development activities, as well as potential regulatory approvals, manufacturing, and commercialization of the program.

Beam will remain eligible to receive development, regulatory, and commercial milestone payments tied to the asset. Beam is able to exercise its option to participate in global co-development and co-commercialization with Pfizer at the end of Phase I/II clinical trials. If exercised, the parties would share net profits, as well as development and commercialization costs, in a 35%/65% ratio (Beam/Pfizer).

Pfizer’s opt-in represents the first exclusive licensing decision to emerge from the companies’ research partnership, under which Pfizer previously committed up to USD 1.05 billion in potential milestone payments in addition to USD 300 million upfront funding and research support to advance Beam’s in vivo base editing pipeline.

Proceeds from the USD 500 million financing are expected to support continued clinical development across Beam’s wholly owned pipeline, including programs targeting hematologic, hepatic, and genetic diseases using its base editing platform.

Beyond its collaboration with Pfizer, Beam has established multiple platform-driven partnerships spanning a range of therapeutic areas and modalities. In 2021, the company entered into a research collaboration with Apellis Pharmaceuticals to develop base editing therapies targeting complement pathway-driven diseases, with Apellis holding options to license up to six programs. Beam has also granted Verve Therapeutics exclusive rights to its base editing technologies for liver-directed cardiovascular targets including PCSK9 and ANGPTL3, and in October 2023 monetized certain downstream opt-in rights to those programs through a separate agreement with Eli Lilly valued at up to USD 600 million. Additional partnerships include a non-exclusive license agreement with Sana Biotechnology covering use of Beam’s Cas12b nuclease for ex vivo engineered cell therapies, as well as a collaboration with Orbital Therapeutics focused on advancing RNA-based approaches and non-viral delivery technologies for in vivo genetic medicines.