BEAM-101
- Generic Name
- BEAM-101
Beam Therapeutics Reports Breakthrough in Alpha-1 Antitrypsin Deficiency Treatment with BEAM-302 Base Editing Therapy
• 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.
• The highest dose level (60mg) achieved mean total AAT of 12.4μM, exceeding the protective therapeutic threshold of 11μM, while reducing harmful mutant Z-AAT by up to 78% in circulation.
• BEAM-302 showed a favorable safety profile with no serious adverse events across all dose levels, supporting continued dose escalation with updated data expected in the second half of 2025.
Beam Therapeutics' BEAM-101 Shows Promise in Sickle Cell Disease Trial
• 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.
• BEAM-101 demonstrated robust increases in fetal hemoglobin and reductions in sickle hemoglobin in treated patients.
• The therapy's safety profile aligns with expectations for busulfan conditioning and autologous hematopoietic stem cell transplantation.
• Beam Therapeutics anticipates dosing 30 patients in the BEACON trial by mid-2025 and presenting updated data.
Hematology Gene Therapy Adoption Faces Challenges Despite Regulatory Advances in 2024
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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ASH 2024: Sanofi's Rilzabrutinib, Novo Nordisk's Etavopivat, and Beam's CRISPR Therapy Show Promise
• Sanofi's rilzabrutinib demonstrated improved platelet response and reduced bleeding in patients with immune thrombocytopenia (ITP) in a Phase 3 trial.
• Novo Nordisk's etavopivat reduced pain crises in sickle cell disease patients by 46% compared to placebo in a Phase 2 study.
• Beam Therapeutics' CRISPR therapy, BEAM-101, showed consistent results in increasing fetal hemoglobin production in sickle cell patients.
ASH 2024: Advances in Sickle Cell Disease and Immune Thrombocytopenia Therapies
• Researchers presented promising data on new therapies for sickle cell disease (SCD) and immune thrombocytopenia (ITP) at the ASH Annual Meeting.
• Etavopivat significantly reduced vaso-occlusive crises in SCD patients in a Phase II trial, suggesting a novel approach to managing the condition.
• Rilzabrutinib demonstrated durable platelet response in refractory ITP patients in a Phase III trial, offering a new option for challenging cases.
• A gene therapy trial (BEAM-101) showed robust results in SCD patients, with high fetal hemoglobin production and no vaso-occlusive crises post-treatment.
BEAM-101 Demonstrates Promising Results in Sickle Cell Disease Trial
• BEAM-101, an investigational base-editing therapy, induced HbF levels >60% and reduced HbS to <40% in all seven treated sickle cell disease patients.
• The BEACON Phase 1/2 trial showed rapid neutrophil and platelet engraftment with normalization of hemolysis markers post BEAM-101 treatment.
• The therapy's safety profile was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation.
• Beam Therapeutics plans to advance BEAM-101 and ESCAPE programs for sickle cell disease, expanding treatment access.
FDA Investigates Blood Cancer Risk Linked to Bluebird Bio's Skysona Gene Therapy
• The FDA is investigating Bluebird Bio's Skysona gene therapy due to reports of hematologic malignancies, including myelodysplastic syndrome and acute myeloid leukemia.
• These adverse events were observed in clinical trials, with diagnoses occurring 14 to 92 months post-treatment, raising concerns about long-term safety.
• The FDA advises healthcare providers to consider alternative treatments like allogeneic stem cell transplants before using Skysona, especially for patients with suitable donors.
• Patients treated with Skysona should undergo lifelong monitoring for malignancies, including regular blood counts and bone marrow evaluations.
FDA Approves Autolus' Obecabtagene Autoleucel (Aucatzyl) for R/R B-cell ALL
• The FDA has approved obecabtagene autoleucel (obe-cel), a CD19-directed CAR-T therapy, for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-cell ALL).
• Approval was based on the Phase 1b/2 FELIX trial, which demonstrated overall complete remission rates above 60% and durable remissions exceeding 12 months.
• Clinical holds on CARsgen's BCMA-directed CAR-T therapy zevorcabtagene autoleucel and Claudin18.2-directed CAR-T satricabtagene autoleucel have been removed by the FDA.
CRISPR Weekly Roundup: Advancements in Gene Editing and Clinical Trials
• Beam Therapeutics reported a patient death in their BEAM-101 sickle cell disease trial, likely due to the conditioning regimen.
• AstraZeneca scientists engineered PsCas9 for therapeutic genome editing in mouse liver, showing promise for hypercholesterolemia treatment.
• YolTech Therapeutics' novel LNP system delivers base editor mRNA to bone marrow cells, activating foetal haemoglobin production for blood disorder treatment.
Beam Therapeutics Reports Patient Death in BEAM-101 Sickle Cell Disease Trial
• Beam Therapeutics reported a patient death in their Phase I/II trial of BEAM-101 for sickle cell disease, attributed to busulfan conditioning, not the therapy.
• Sana Biotechnology is refocusing on autoimmune assets, leading to workforce reductions and program cuts to extend its cash runway into 2026.
• OSE Therapeutics announced positive Phase II data for lusvertikimab in ulcerative colitis, marking a significant step for the anti-IL-7 therapy.
• Ring Therapeutics is partnering with Singaporean institutions to advance a new class of viral vectors based on commensal anelloviruses.
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