Current CIBMTR Gene Therapy Studies
| Company | bluebird bio | bluebird bio | Vertex |
|---|---|---|---|
| Product name | Zynteglo | Skysona | Casgevy |
| Scientific name | Betibeglogene autotemcel | Elivaldogene autotemcel | Exagamglogene autotemcel |
| Other name | REG-501 | REG-502 | n/a |
| FDA approval date | 8/17/2022 | 9/16/2022 | 12/8/2023 (SCD) and 1/16/2024 (Thal) |
| Disease | Beta-thalassemia | cALD | Sickle Cell Disease and Beta-thalassemia |
| CIBMTR Study ID | CS22-24 | CS20-51 | CS20-55 |
| Package insert | Zyntelgo | Skysona | Casgevy |
| Reporting guide? | Yes | Yes | Yes |
Centers must opt-in to participate in gene therapy studies. Enrollment into these gene studies is automated in FormsNet3 based on form completion. No additional consent is required. These studies fall under the current CIBMTR Research Database Protocol.
There is additional study specific training on the CIBMTR Portal. There are recordings of the site initiation meeting (when applicable), study synopsis (when applicable) and copies of the reporting guides.
Last modified:
Mar 09, 2026