EMA/PE/0000232894 - paediatric investigation plan

adeno-associated viral vector serotype 9 containing the human MECP2 gene (TSHA-102)

Key facts

• Active substance:
  adeno-associated viral vector serotype 9 containing the human MECP2 gene (TSHA-102)
• Therapeutic area:
  Congenital, familial and genetic disorders
• Decision number:
  EMA/PE/0000232894
• PIP number:
  EMA/PE/0000232894
• Pharmaceutical form(s):
  Solution for infusion
• Condition(s) / indication(s):
  Treatment of Rett syndrome
• Route(s) of administration:
  Intrathecal use
• Decision type:
  P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
• Decision date:
  14/04/2025
• Compliance check done:
  No

Decision

EMA/PE/0000232894 : EMA decision of 14 April 2025 on the agreement of a paediatric investigation plan and on the granting of a deferral for adeno-associated viral vector serotype 9 containing the human MECP2 gene

Adopted Reference Number: EMADOC-1700519818-2039246