Drugs / BEAM-101
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05456880 | Aug 2022 → Feb 2028 expected | sickle cell disease | Beam Therapeutics Inc. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 10
| Date | Issuer | Release |
|---|---|---|
| 2026-04-01 | Beam Therapeutics Inc. | Results Beam Therapeutics Announces Publication of BEACON Phase 1/2 Data for risto-cel in Patients with Sickle Cell Disease (SCD) in The New England Journal of Medicine beamtx.com ↗
Initially Presented at ASH, Data from 31 Patients with SCD Demonstrated Deep Resolution of Red Blood Cell Dysfunction and Reduced Time in Hospital Driven by a Median of One Cell Collection Cycle and Rapid Engraftment |
| 2025-12-06 | Beam Therapeutics Inc. | Results Beam Therapeutics Reports Updated Data from BEACON Phase 1/2 Trial of ristoglogene autogetemcel (risto-cel) Highlighting Durable, Differentiated Profile in Sickle Cell Disease (SCD) at American Society of Hematology (ASH) Annual Meeting beamtx.com ↗ |
| 2025-08-14 | Beam Therapeutics Inc. | Regulatory Beam Therapeutics Announces U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to BEAM-101 for the Treatment of Sickle Cell Disease beamtx.com ↗
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). |
| 2025-06-13 | Beam Therapeutics Inc. | Results 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 beamtx.com ↗
New data from the BEACON trial with more patients and longer follow-up provide further demonstration of the strong clinical profile for the investigational base-editing therapy BEAM-101, as initially established in previously announced data at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2024. |
| 2025-06-03 | Beam Therapeutics Inc. | Regulatory Beam Therapeutics Announces U.S. FDA Orphan Drug Designation Granted to BEAM-101 for the Treatment of Sickle Cell Disease beamtx.com ↗
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 orphan drug designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD). |
| 2024-12-07 | Beam Therapeutics Inc. | Results Beam Therapeutics Announces New Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 in Sickle Cell Disease at American Society of Hematology (ASH) Annual Meeting beamtx.com ↗
today announced new safety and efficacy data from its BEACON Phase 1/2 clinical trial of BEAM-101 in patients with sickle cell disease (SCD) with severe vaso-occlusive crises (VOCs). |
| 2024-11-05 | Beam Therapeutics Inc. | Results Beam Therapeutics to Present Data Across Hematology Franchise, Including First Clinical Data for BEAM-101 in Sickle Cell Disease and ESCAPE Non-human Primate Data, at American Society of Hematology (ASH) Annual Meeting beamtx.com ↗
Initial Results from BEACON Phase 1/2 Clinical Trial Demonstrate Potential for Differentiation of Base Editing and BEAM-101 |
| 2024-06-14 | Beam Therapeutics Inc. | Results Beam Therapeutics Reports Data Highlighting Optimized Manufacturing Process for BEAM-101, an Investigational Base Editing Therapeutic for Sickle Cell Disease beamtx.com ↗
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported data highlighting its optimized, closed and automated manufacturing process for its base-edited CD34+ hematopoietic stem and progenitor cell (HSPC) genetic medicines in a poster presentation at the European Hematology Association (EHA) Hybrid Congress. |
| 2021-11-08 | Beam Therapeutics Inc. | Regulatory Beam Therapeutics Provides Business and Pipeline Updates and Reports Third Quarter 2021 Financial Results beamtx.com ↗
As part of today’s update, Beam announced that its Investigational New Drug (IND) application for BEAM-101 for the treatment of sickle cell disease was cleared by the U.S. Food and Drug Administration (FDA). |
| 2020-12-05 | Beam Therapeutics Inc. | Results Beam Therapeutics Presents Updated Data from Novel Base Editing Programs for Sickle Cell Disease at ASH 2020 beamtx.com ↗
The new data for BEAM-101 highlight the precise targeting of our base editor with no off-target editing observed and will support our planned Investigational New Drug application submission in the second half of 2021. |
All press releases naming this drug 16 releases
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | BEAM-101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05456880 ↗ |
| Known as | risto-cel | “Ristoglogene autogetemcel (risto-cel) includes autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited to target the HBG1 and HBG2 promoters and...” PMID 41931046 ↗ Apr 2026 |
| Known as | Ristoglogene autogetemcel | “Ristoglogene autogetemcel (risto-cel) includes autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited to target the HBG1 and HBG2 promoters and...” PMID 41931046 ↗ Apr 2026 |
| Action | Restore | “yielding a switch in hemoglobin production from sickle hemoglobin (HbS) to antisickling fetal hemoglobin (HbF)” PMID 41931046 ↗ Apr 2026 |
| Modality | Cell therapy | “autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited” PMID 41931046 ↗ Apr 2026 |
| Route | Intravenous | “administered as a single dose by IV infusion” NCT05456880 ↗ |
| Target | HBG1 | “base-edited to target the HBG1 and HBG2 promoters” PMID 41931046 ↗ Apr 2026 |
| Target | HBG2 | “base-edited to target the HBG1 and HBG2 promoters” PMID 41931046 ↗ Apr 2026 |