Beam Therapeutics (BEAM) investor relations material

Recent progress and strategic vision

• Advanced base editing technology enables precise, permanent gene edits without double-strand breaks, expanding therapeutic possibilities in hematology and liver diseases.

• Lead program for sickle cell disease (risto-cel) is progressing toward a BLA filing by year-end, with plans to expand in vivo approaches using LNP delivery.

• Alpha-1 antitrypsin deficiency program (BEAM-302) achieved functional cures at 60 mg dosing, with robust, predictable editing and strong safety profile confirmed in trials.

• Additional liver programs, including BEAM-301 for glycogen storage disease and a PKU-targeting candidate, are advancing with IND filings and data expected this year.

• Platform predictability and scalability support confidence in expanding to new indications and patient populations.

Clinical data and safety insights

• 60 mg dose of BEAM-302 delivers Alpha-1 levels consistent with healthy carriers, with no added benefit at higher doses; redosing at lower levels planned for subtherapeutic patients.

• Safety profile shows transient, manageable liver enzyme elevations typical of LNP therapies, with no significant long-term liver function impact.

• Bystander editing produces a known, functional protein variant, confirmed by structural and functional assays, supporting therapeutic efficacy.

• Patient baseline characteristics influence serum Alpha-1 response, but all treated patients reached therapeutic thresholds.

• Ongoing and future studies will include biopsies and imaging to directly assess liver benefit and fibrosis resolution.

Regulatory and market outlook

• Accelerated approval pathway for Alpha-1 program is supported by stable FDA review teams and robust biomarker data, with 12-month follow-up required for 50 patients.

• Pricing strategy for one-time gene therapies will exceed annual augmentation therapy costs, justified by long-term pharmacoeconomic value and payer acceptance in similar indications.

• Sickle cell program targets both severe and broader patient populations, with ex vivo and in vivo approaches in development to maximize reach and scalability.

• Commitment to ongoing innovation and global patient access, leveraging both ex vivo and in vivo gene editing platforms.