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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...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 5 | PHASE3 | Etavopivat Low dose |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, 20vPCV |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Agios Pharmaceuticals, Inc. | AGIO | 2 | PHASE2 | Mitapivat |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470, Famotidine, Pantoprazole |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
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 drug is being evaluated in the BEACON study, which is assessing its safety and efficacy in patients with this condition.
BEAM-101 is being developed by Beam Therapeutics Inc., a biotechnology company publicly traded under the ticker symbol BEAM on the NASDAQ stock exchange. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational therapy for Sickle Cell Disease.
BEAM-101 is currently in Phase 1 clinical development. It is an investigational drug and has not yet received approval from regulatory agencies. The ongoing Phase 1 study, known as BEACON, is actively evaluating the safety and efficacy of BEAM-101 in patients with severe Sickle Cell Disease.
BEAM-101 is being studied in a Phase 1 clinical trial with the identifier NCT05456880. This trial, named BEACON, is evaluating the safety and efficacy of BEAM-101 in patients with severe Sickle Cell Disease. The study is active but not recruiting, with an enrollment target of 15 participants in the United States.
No, BEAM-101 is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for the treatment of severe Sickle Cell Disease. The drug is being evaluated in the BEACON clinical trial to assess its safety and efficacy, but it has not yet received regulatory approval.
The BEACON trial, identified as NCT05456880, is a Phase 1 study evaluating the safety and efficacy of BEAM-101 in patients with severe Sickle Cell Disease. This single-arm, open-label trial is being conducted in the United States and is currently active but not recruiting. The study aims to enroll 15 participants aged 12 years and older.