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BEAM-101 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| BEAM-101 | EXPERIMENTAL | BEAM-101 manufactured with autologous CD34+ hematopoietic stem cells collected by plerixafor mobilization and edited ex vivo. No maximum dose has been set for BEAM-101; all of the gene edited cells that pass release specifications will be administered to the patient. BEAM 101 will be administered as a single dose by IV infusion. |
| Name | Type | Description |
|---|---|---|
| BEAM-101 | BIOLOGICAL | Single dose of BEAM-101 administered by IV following myeloablative conditioning with busulfan |
Key Inclusion Criteria Include: 1. Age ≥12 years to ≤35 years 2. Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes. 3. Severe SCD defined by the occurrence of at least 4 severe VOCs in the 24 months prior to screening despite receiving hydroxyurea or other supportive...
Top 12 of 13 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 6 | PHASE3 | Etavopivat |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab, OTQ923, ITU512 |
| Agios Pharmaceuticals, Inc. | AGIO | 3 | PHASE3 | Mitapivat, Tebapivat |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, rilzabrutinib |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Travere Therapeutics, Inc. | TVTX | 1 | PHASE3 | L-Citrulline |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470 |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
BEAM-101 is an investigational therapy being studied for the treatment of severe sickle cell disease. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The therapy is being evaluated in the BEACON study, which is assessing its safety and efficacy in patients with this condition.
BEAM-101 targets the HBG1 and HBG2 genes, which are involved in the production of fetal hemoglobin. By modulating these targets, the therapy aims to address the underlying pathophysiology of sickle cell disease. This mechanism is being investigated in early-stage clinical trials to determine its potential therapeutic benefit.
BEAM-101 is being developed by Beam Therapeutics Inc., a biotechnology company publicly traded under the ticker symbol BEAM. The company is conducting clinical research on this investigational therapy for sickle cell disease, with the therapy currently in Phase 1 development.
BEAM-101 is in Phase 1 clinical development. It is an investigational therapy and has not received regulatory approval. The ongoing Phase 1 study, known as BEACON, is actively enrolling or has enrolled participants to evaluate the safety and efficacy of BEAM-101 in patients with severe sickle cell disease.
BEAM-101 is being studied in the BEACON trial, registered as NCT05456880. This Phase 1 study is evaluating the safety and efficacy of BEAM-101 in patients with severe sickle cell disease. The trial is active but not recruiting, with an enrollment target of 15 participants in the United States.