Beam Therapeutics (BEAM) Barclays 27th Annual Global Healthcare Conference summary

Key data and technology updates

• Presented first clinical data for BEAM-302 in alpha-1 antitrypsin deficiency (AATD), showing dose-responsive increases in AAT protein and significant reduction of toxic protein, with strong safety profile across all dose cohorts.

• Achieved above-therapeutic threshold AAT levels, moving patients out of the disease category and into a carrier-like state, with up to 78% reduction in toxic Z protein and 88% of circulating protein being functional M type.

• Base editing technology enables precise, single-base gene correction without DNA cutting, offering versatility for gene activation, knockdown, and correction.

• Lipid nanoparticle (LNP) delivery system is proprietary, manufactured in-house, and validated for safety and efficacy, supporting a platform approach for future liver-targeted programs.

• BEAM-101 sickle cell program shows best-in-class hematology results, full anemia resolution, and rapid engraftment; adult trial fully enrolled, adolescent enrollment ongoing, with new data expected at EHA and ASH.

Clinical development and regulatory strategy

• Dose escalation for BEAM-302 will continue, with potential to reach higher AAT levels (15–20 μM) due to strong safety, aiming for even greater efficacy.

• Functional and total protein assays confirm the therapeutic effect; FDA expected to focus on these biomarkers and functional outcomes for regulatory approval.

• Accelerated approval pathways are being explored, leveraging biomarker and genetic evidence of disease reversal; additional studies may target long-term lung and liver function endpoints.

• Part B of the AATD study will include patients with heavier liver involvement, using a similar dose escalation approach but may not require lowest doses due to established safety.

• Platform validation of LNP delivery enables rapid expansion into additional liver programs, with a streamlined process for new product filings.