Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-101 for the Treatment of Sickle Cell Disease
Beam Therapeutics (NASDAQ:BEAM) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BEAM-101, its investigational cell therapy for sickle cell disease. The designation follows the previously granted orphan drug status and provides opportunities for accelerated development and enhanced FDA collaboration.
The company's BEACON Phase 1/2 trial has now treated 30 patients, with updated data from 17 patients showing promising results including increased fetal hemoglobin, reduced sickle hemoglobin, and improved markers of hemolysis and oxygen delivery. The treatment demonstrated rapid neutrophil and platelet engraftment, with no VOCs reported post-engraftment. Additional trial data is expected by the end of 2025.
Beam Therapeutics (NASDAQ:BEAM) ha ricevuto la designazione Regenerative Medicine Advanced Therapy (RMAT) dalla FDA per BEAM-101, la sua terapia cellulare sperimentale per la malattia falciforme. La designazione segue il precedente status di farmaco orfano e apre opportunità per uno sviluppo accelerato e una maggiore collaborazione con la FDA.
Il trial BEACON Fase 1/2 ha finora trattato 30 pazienti, con dati aggiornati su 17 pazienti che mostrano risultati promettenti, tra cui aumento dell'emoglobina fetale, riduzione dell'emoglobina falciforme e miglioramento dei marcatori di emolisi e del trasporto di ossigeno. Il trattamento ha mostrato un rapido attecchimento di neutrofili e piastrine, senza crisi vaso-occlusive (VOCs) segnalate dopo l'attecchimento. Ulteriori dati dello studio sono attesi entro la fine del 2025.
Beam Therapeutics (NASDAQ:BEAM) ha recibido la designación Regenerative Medicine Advanced Therapy (RMAT) de la FDA para BEAM-101, su terapia celular en investigación para la enfermedad de células falciformes. La designación sigue al estatus de medicamento huérfano previamente otorgado y ofrece oportunidades para un desarrollo acelerado y una mayor colaboración con la FDA.
El ensayo BEACON Fase 1/2 ha tratado ahora a 30 pacientes, con datos actualizados de 17 pacientes que muestran resultados prometedores, incluidos aumento de la hemoglobina fetal, reducción de la hemoglobina falciforme y mejora de los marcadores de hemólisis y del suministro de oxígeno. El tratamiento mostró una rápida recuperación de neutrófilos y plaquetas, sin crisis vasooclusivas (VOCs) reportadas tras el injerto. Se esperan datos adicionales del ensayo para finales de 2025.
Beam Therapeutics (NASDAQ:BEAM)는 BEAM-101에 대해 FDA로부터 재생의학 고급 치료(RMAT) 지정을 받았습니다. BEAM-101은 겸상 적혈구 질환을 대상으로 하는 연구용 세포치료제입니다. 이 지정은 이전에 부여된 희귀의약품 지정에 이은 것으로, 개발 가속화와 FDA와의 협력 강화 기회를 제공합니다.
BEACON Phase 1/2 임상시험은 현재까지 30명의 환자를 치료했으며, 17명의 환자에 대한 업데이트 데이터는 태아 혈색소 증가, 겸상형 혈색소 감소, 용혈 및 산소 운반 지표 개선 등 유망한 결과를 보여주고 있습니다. 치료는 중성구 및 혈소판의 빠른 회복을 보였고, 이식(정착) 후 VOC(혈관 폐색성 위기)는 보고되지 않았습니다. 추가 연구 데이터는 2025년 말까지 나올 것으로 예상됩니다.
Beam Therapeutics (NASDAQ:BEAM) a reçu la désignation Regenerative Medicine Advanced Therapy (RMAT) de la FDA pour BEAM-101, sa thérapie cellulaire expérimentale contre la drépanocytose. Cette désignation fait suite au statut de médicament orphelin précédemment accordé et offre des opportunités pour un développement accéléré et une collaboration renforcée avec la FDA.
L'essai BEACON de phase 1/2 a désormais traité 30 patients, et des données mises à jour sur 17 patients montrent des résultats prometteurs, notamment une augmentation de l'hémoglobine fœtale, une diminution de l'hémoglobine falciforme et une amélioration des marqueurs d'hémolyse et du transport d'oxygène. Le traitement a montré une prise rapide des neutrophiles et des plaquettes, et aucune crise vaso-occlusive (VOCs) n'a été signalée après la prise. Des données supplémentaires de l'essai sont attendues d'ici la fin 2025.
Beam Therapeutics (NASDAQ:BEAM) hat von der FDA die Regenerative Medicine Advanced Therapy (RMAT)-Einstufung für BEAM-101 erhalten, seine experimentelle Zelltherapie für die Sichelzellenerkrankung. Die Einstufung folgt dem bereits erteilten Orphan-Drug-Status und bietet Chancen für eine beschleunigte Entwicklung sowie eine intensivere Zusammenarbeit mit der FDA.
Die BEACON Phase-1/2-Studie hat inzwischen 30 Patienten behandelt; aktualisierte Daten von 17 Patienten zeigen vielversprechende Ergebnisse, darunter erhöhtes fetales Hämoglobin, reduziertes Sichelzell-Hämoglobin sowie verbesserte Marker für Hämolyse und Sauerstofftransport. Die Behandlung zeigte ein rasches Einwachsen von Neutrophilen und Thrombozyten, und nach dem Einwachsen wurden keine vaso-okklusiven Krisen (VOCs) berichtet. Weitere Studiendaten werden bis Ende 2025 erwartet.
- Received FDA RMAT designation for BEAM-101, enabling enhanced FDA collaboration and potential accelerated approval
- Strong clinical results from 17 patients showing robust improvements in key disease markers
- No VOCs (vaso-occlusive crises) reported post-engraftment in treated patients
- Advanced manufacturing process showing consistently high yields and viability
- None.
Insights
BEAM-101's RMAT designation accelerates its regulatory pathway, enhancing its competitive position in the lucrative sickle cell disease market.
Beam Therapeutics has secured a crucial Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BEAM-101, their base editing cell therapy for sickle cell disease. This designation, following their earlier orphan drug designation, significantly accelerates their regulatory pathway by providing enhanced FDA collaboration opportunities, potential surrogate endpoints for accelerated approval, and possibilities for rolling review and priority review.
The clinical data looks particularly compelling. With 30 patients now dosed in the BEACON Phase 1/2 trial, the treatment has demonstrated robust increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS) - precisely the molecular changes needed to address the underlying disease mechanism. Importantly, patients showed rapid neutrophil and platelet engraftment and required a median of just one mobilization cycle, suggesting a potentially more efficient treatment process than competing therapies.
The absence of vaso-occlusive crises (VOCs) post-engraftment is particularly significant, as these painful episodes are the hallmark clinical manifestation of sickle cell disease. Their manufacturing process, described as "largely automated" with "consistently high yields and viability," could provide crucial commercial advantages in cell therapy, where manufacturing challenges often create bottlenecks.
With additional data expected later this year and the regulatory advantages conferred by both RMAT and orphan designations, Beam is positioning BEAM-101 for an accelerated path to market in a condition affecting approximately 100,000 Americans and millions globally.
CAMBRIDGE, Mass., Aug. 14, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD).
“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 one-time, best-in-class therapy for severe sickle cell disease,” said Giuseppe Ciaramella, Ph.D., president of Beam Therapeutics. “These designations not only recognize the promise of BEAM-101 but also enable enhanced collaboration with the FDA as we advance toward a BLA 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 sickle cell disease.”
The FDA’s RMAT designation is designed to support the development and evaluation of regenerative medicines, including genetic therapies, with the intention of addressing serious or life-threatening diseases that have unmet medical needs. RMAT designation provides opportunities for early interactions with the FDA to discuss potential surrogate or intermediate endpoints to support accelerated approval, organizational commitment from senior staff at the agency, opportunities to participate in novel review and development programs, and the potential for a rolling review and priority review of a product’s future biologics license application.
Updated clinical data from the BEACON Phase 1/2 clinical trial of BEAM-101 were presented at the European Hematology Association (EHA) 2025 Congress in June, providing further demonstration of the strong clinical profile for BEAM-101, as initially established in previously announced data at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2024. Updated data from 17 patients treated with BEAM-101 demonstrated robust and durable increases in fetal hemoglobin (HbF) and reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet engraftment, and normalized or improved markers of hemolysis and oxygen delivery. Patients required a median of one mobilization cycle. No VOCs were reported post-engraftment. BEAM-101 is manufactured using an advanced, largely automated process that has demonstrated consistently high yields and viability.
Beam previously announced that the FDA granted orphan drug designation to BEAM-101 in June, and the company plans to present updated data from the BEACON Phase 1/2 trial by the end of 2025.
About BEAM-101
BEAM-101 is an investigational genetically modified cell therapy for the treatment of severe sickle cell disease (SCD). The one-time therapy consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that have been base-edited in the promotor regions of the HBG1/2 genes and are administered via a hematopoietic stem cell transplant procedure. The BEAM-101 edit is designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A expression, leading to increased production of non-sickling and anti-sickling fetal hemoglobin (HbF) and thus mimicking the effects of naturally occurring variants seen in hereditary persistence of fetal hemoglobin. HbF is the predominant hemoglobin variant during development and early life. The safety and efficacy of BEAM-101 is being evaluated in the ongoing BEACON Phase 1/2 study, an open-label, single-arm, multicenter trial in adult patients with SCD with severe vaso-occlusive crises (VOCs).
About Sickle Cell Disease
Sickle cell disease (SCD), a severe inherited blood disease, is caused by a single point mutation, E6V, in the beta globin gene. This mutation causes the mutated form of sickle hemoglobin (HbS) to aggregate into long, rigid molecules that bend red blood cells into a sickle shape under conditions of low oxygen. Sickled cells obstruct blood vessels and die prematurely, ultimately resulting in anemia, severe pain (crises), infections, stroke, organ failure and early death. SCD is the most common inherited blood disorder in the United States (U.S.), affecting an estimated 100,000 individuals within the U.S. and approximately eight million people worldwide.
About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform with integrated gene editing, delivery and internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: the therapeutic applications and potential of our technology, including with respect to SCD; our plans, and anticipated timing, to advance our programs, including the clinical trial design and expectations for BEAM-101; our plans and anticipated timing to present data from ongoing clinical trials; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the uncertainty that our product candidates will receive regulatory approval necessary to advance human clinical trials; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates or the delivery modalities we rely on to administer them may cause serious adverse events; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; and the other risks and uncertainties identified under the headings “Risk Factors Summary” and “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2024, our Quarterly Reports on Form 10-Q, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.
Contacts:
Investors:
Holly Manning
Beam Therapeutics
hmanning@beamtx.com
Media:
Josie Butler
1AB
josie@1abmedia.com
FAQ
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