Current CIBMTR Gene Therapy Studies
| Company | bluebird bio | bluebird bio |
|---|---|---|
| Product name | Zynteglo | Skysona |
| Scientific name | Betibeglogene autotemcel | Elivaldogene autotemcel |
| Other name | REG-501 | REG-502 |
| FDA approval date | 8/17/2022 | 9/16/2022 |
| Disease | Beta-thalassemia | cALD |
| Study ID | CS22-24 | CS20-51 |
| Package insert | Zynteglo | Skysona |
| Reporting guide? | Yes | Yes |
Enrollment into these gene studies is done when the product name is reported on the F2400. No additional consent is required. These studies fall under the current CIBMTR Research Database Protocol.
Additional reporting guides are available for these studies on the CIBMTR Portal and contain extra information that is product specific and not part of the Forms Instruction Manuals. Some pages also contain recordings of the site initiation meetings.
Last modified:
Mar 25, 2025