EMEA-002730-PIP03-21 - paediatric investigation plan

autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene

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/0548/2021
• PIP number:
  EMEA-002730-PIP03-21
• Pharmaceutical form(s):
  Dispersion for infusion
• Condition(s) / indication(s):
  Treatment of sickle cell disease
• Route(s) of administration:
  Intravenous use
• Decision type:
  P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
• Decision date:
  31/12/2021
• Compliance check done:
  No

Decision

P/0548/2021 : EMA decision of 31 December 2021 on the agreement of a paediatric investigation plan and on the granting of a deferral and on the granting of a waiver for autologous CD34+ hematopoietic stem cells with... (EMEA-002730-PIP03-21)

Adopted Reference Number: EMA/706930/2021