Last reviewed · How we verify
BETIBEGLOGENE AUTOTEMCEL
ZYNTEGLO adds functional β-globin genes to patient HSCs, producing RBCs with modified β A-T87Q -globin to correct β/α-globin imbalance and increase Hb levels.
At a glance
| Generic name | BETIBEGLOGENE AUTOTEMCEL |
|---|---|
| Target | β-globin gene |
| Modality | Gene therapy |
| Phase | FDA-approved |
| First approval | 2019 |
Mechanism of action
ZYNTEGLO introduces functional copies of a modified β-globin gene into a patient's hematopoietic stem cells. These modified cells then produce red blood cells containing a functional form of β-globin, which helps balance the β/α-globin ratio and increases the production of functional adult hemoglobin, potentially eliminating the need for regular blood transfusions.
Approved indications
Common side effects
- Mucositis
- Febrile neutropenia
- Vomiting
- Pyrexia (fever)
- Alopecia (hair loss)
- Epistaxis (nose bleed)
- Abdominal pain
- Musculoskeletal pain
- Cough
- Headache
- Diarrhea
- Rash
Serious adverse events
- Pyrexia (fever)
- Thrombocytopenia
- Liver veno-occlusive disease
- Febrile neutropenia
- Neutropenia
- Stomatitis
- Neutropenia (Grade 3 or 4)
- Thrombocytopenia (Grade 3 or 4)
- Leukopenia (Grade 3 or 4)
- Anemia (Grade 3 or 4)
Key clinical trials
- A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
- Study to Assess the Efficacy and Safety of CLBS12 in Patients with Critical Limb Ischemia (CLI) (PHASE2)
- A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia (PHASE3)
- A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype (PHASE3)
- Study of Intracoronary CD34+ Cell Administration in Patients With Early Coronary Atherosclerosis (PHASE1)
- Autologous Followed by Non-myeloablative Allogeneic Transplantation for Non-Hodgkin's Lymphoma (PHASE2)
- Peripheral Stem Cell Transplant in Treating Patients With Multiple Myeloma (PHASE2)
- An Efficacy and Safety Study of Autologous Cluster of Differentiation 34 (CD34+) Hematopoietic Progenitor Cells Transduced With Placebo or an Anti- Human Immunodeficiency Virus Type 1 (HIV-1) Ribozyme (OZ1) in Participants With HIV-1 Infection (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- BETIBEGLOGENE AUTOTEMCEL CI brief — competitive landscape report
- BETIBEGLOGENE AUTOTEMCEL updates RSS · CI watch RSS