Beam Therapeutics (BEAM) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Strategic portfolio and technology platform

• Advancing a growing portfolio with commercial potential in gene editing for diseases like sickle cell, alpha-1 antitrypsin deficiency (AATD), PKU, and GSD1A.

• Utilizing next-generation base editing (CRISPR 2.0) for precise single base changes without double-stranded breaks.

• Demonstrated leadership and validation in base editing, with ongoing development in prime editing and targeted delivery technologies.

• Fully integrated manufacturing and delivery capabilities, including internal cell and mRNA production, supporting rapid expansion to multiple indications.

• Platform enables accelerated development cycles and operational efficiency across pipeline programs.

BEAM-302 for alpha-1 antitrypsin deficiency (AATD)

• Targets a large unmet need by correcting the SERPINA1 gene mutation, converting the disease-causing Z allele to the normal M allele.

• Demonstrated durable, one-time correction with patients achieving protective AAT levels and functional protein production.

• Clinical data show normalization of AAT, elimination of toxic Z protein, and restoration of normal gene regulation.

• Safety profile remains strong, with only transient, non-serious grade three liver enzyme elevation in a single patient.

• Pivotal studies underway at 60 mg dose, with a significant lead over competitors and first-mover advantage in regulatory and commercial pathways.

Pipeline progress: PKU and risto-cel

• BEAM-304 for PKU aims for a one-time cure by correcting point mutations, enabling normalization of phenylalanine metabolism and diet.

• IND opened for BEAM-304, with innovative FDA platform approach allowing multiple editors and rapid expansion to other liver disorders.

• Clinical trial for BEAM-304 expected to begin dosing by year-end or early next year, with quick escalation and expansion planned.

• Risto-cel for sickle cell disease targets the sickest 10% of patients, offering best-in-class efficacy, rapid engraftment, and operational advantages.

• All three programs (risto-cel, BEAM-302, BEAM-304) positioned as potential blockbuster franchises with sequential commercial launches.