BEAM-101

Beam Therapeutics' BEAM-302 demonstrated the first-ever clinical genetic correction of the disease-causing PiZ mutation in Alpha-1 Antitrypsin Deficiency patients, with a single dose producing durable increases in functional AAT protein.

Beam Therapeutics will present updated data from the BEACON Phase 1/2 trial of BEAM-101 for sickle cell disease at the 2025 Tandem Meetings.

Major regulatory approvals for gene therapies in hematological disorders marked 2024, including Casgevy's approval in Canada for sickle cell disease and beta thalassemia. However, commercial uptake remains slower than anticipated, with only 24 patients starting treatment across two FDA-approved sickle cell therapies by mid-2024. The field also faced setbacks, including a patient death related to conditioning therapy in a clinical trial.

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Sanofi's rilzabrutinib demonstrated improved platelet response and reduced bleeding in patients with immune thrombocytopenia (ITP) in a Phase 3 trial.