Trial Name: BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (BEACON)
ClinicalTrials.gov ID: NCT05456880
Sponsor: Beam Therapeutics Inc.
Completion Date (Estimated): February 1, 2027
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BEAM-101 Receives RMAT for the Treatment of Sickle Cell Disease
Key Takeaways
- BEAM-101, a genetically modified cell therapy, received RMAT designation for treating sickle cell disease, following promising BEACON trial results.
- The therapy involves base-editing CD34+ hematopoietic stem cells to increase fetal hemoglobin, reducing sickle hemoglobin levels.
The designation is supported by promising results from the ongoing phase 1/2 BEACON clinical trial.
The FDA granted a regenerative medicine advanced therapy (RMAT) designation to BEAM-101 (Beam Therapeutics) for the treatment of patients with sickle cell disease (SCD). The designation follows positive results from the ongoing BEACON phase 1/2 clinical trial (NCT05456880), which were presented at the 2025 European Hematology Association (EHA) Congress.1,2
Image credit: tussik | stock.adobe.com
BEAM-101 is an investigational, genetically modified cell therapy that consists of autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited in the promoter regions of the HBG1/2 genes. This one-time therapy is administered via a hematopoietic stem cell transplant (HSCT) procedure, and it is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without completely disrupting BCL11A expression. This then leads to an increased production of nonsickling and antisickling fetal hemoglobin, therefore mimicking the effects of naturally occurring variants that are seen in the hereditary persistence of fetal hemoglobin. In June 2025, BEAM-101 was granted an orphan drug designation for this same indication.1,3
SCD is characterized by sickled cells, which obstruct blood vessels and die prematurely, ultimately resulting in anemia, severe pain, stroke, infections, organ failure, and early death. It is reported in an estimated 100,000 individuals, making it the most common inherited blood disorder in the US. Worldwide, about 8 million people have the disease.1,3
At the 2025 EHA Congress, data from the open-label, single-arm, multicenter BEACON trial demonstrated that 17 patients with SCD who were treated with BEAM-101 experienced robust and durable increases in fetal hemoglobin and reductions in sickle hemoglobin, rapid neutrophil (16.5 days [range: 12–30]) and platelet engraftment (19.5 days [range: 11–34]), and normalized or improved markers of hemolysis and oxygen delivery. The enrolled patients required a median of 1 mobilization cycle (range: 1–3 cycles). Of note, there were no severe vaso-occlusive crises reported by patients post-engraftment.2,4
Specifically, the data presented were consistent with those previously presented, with fetal hemoglobin levels exceeding 60% and a durable reduction in corresponding sickle hemoglobin below 40%. A pancellular distribution of fetal hemoglobin-expressing cells, with fetal hemoglobin levels per cell above the sickling threshold, was maintained through follow-up. Increases in fetal hemoglobin, decreases in sickle hemoglobin, and resolution of anemia were durable for up to 15 months.4
The safety profile of BEAM-101 was observed to be consistent with busulfan conditioning, autologous HSCT, and underlying SCD. The most common treatment-emergent adverse events (TEAEs) were consistent with busulfan conditioning (eg, stomatitis, febrile neutropenia, and anemia). One patient died 4 months after receiving a BEAM-101 infusion, but this was due to respiratory failure, which was determined to be unrelated to BEAM-101.4
“We are thrilled that the FDA has granted RMAT designation to BEAM-101, following orphan drug designation earlier this year, reinforcing its potential as a 1-time, best-in-class therapy for severe SCD,” Giuseppe Ciaramella, PhD, president of Beam Therapeutics, said in the news release. “These designations not only recognize the promise of BEAM-101 but also enable enhanced collaboration with the FDA as we advance toward a biologics license application filing. With 30 patients now dosed in the BEACON phase 1/2 trial and additional data expected later this year, we remain focused on delivering a transformative treatment to people living with SCD.”1
REFERENCES
GlobeNewswire. Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-101 for the Treatment of Sickle Cell Disease. News release. August 14, 2025. Accessed August 14, 2025. https://www.globenewswire.com/news-release/2025/08/14/3133299/0/en/Beam-Therapeutics-Announces-U-S-FDA-Regenerative-Medicine-Advanced-Therapy-RMAT-Designation-Granted-to-BEAM-101-for-the-Treatment-of-Sickle-Cell-Disease.html
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (BEACON). ClinicalTrials.gov identifier: NCT05456880. Updated January 10, 2025. Accessed August 14, 2025. https://clinicaltrials.gov/study/NCT05456880
Beam Therapeutics. Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-101 for the Treatment of Sickle Cell Disease. June 3, 2025. Accessed August 14, 2025. https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-us-fda-orphan-drug-designation-0
Beam Therapeutics. Beam Therapeutics Announces New Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 Supporting Differentiated Profile in Sickle Cell Disease (SCD) at European Hematology Association (EHA) 2025 Congress. News release. June 13, 2025. Accessed August 14, 2025. https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-new-data-beacon-phase-12-clinical-0
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