MHRA-101740-PIP01-24

Key Facts

Active Substance
  • messenger RNA encoding Cas9
  • single guide RNA targeting the human KLKB1 gene
Invented Name
Not yet available
PIP Number MHRA-101740-PIP01-24
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Dispersion for infusion
Therapeutic area
Therapeutic area:
  • Other: Angioedemas, Hereditary
Conditions / Indications
Conditions / Indications:
  • Treatment of hereditary angioedema (HAE)
Route(s) of administration
Route(s) of administration:
  • Intravenous use
PIP applicant
Decision Type
Decision Type
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s)
Compliance Check
Compliance Check
No
Compliance Check Decision Date
Compliance Check Procedure Number

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):messenger RNA encoding Cas9single guide RNA targeting the human KLKB1 gene.pdf
Published Date 14/07/2025