EMEA-002730-PIP04-21
Key facts
| Active substance |
autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene
|
| Therapeutic area |
Haematology-Hemostaseology
|
| Decision number |
P/0549/2021
|
| PIP number |
EMEA-002730-PIP04-21
|
| Pharmaceutical form(s) |
Dispersion for infusion
|
| Condition(s) / indication(s) |
Treatment of beta-thalassemia intermedia and major
|
| Route(s) of administration |
Intravenous use
|
| Contact for public enquiries |
Vertex Pharmaceuticals (Ireland) Limited
E-mail: vertexmedicalinfo@vrtx.com |
| Decision type |
P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
|