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Fumarylacetoacetate hydrolase gene therapy - Castle Creek Pharmaceuticals

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Drug Profile

Fumarylacetoacetate hydrolase gene therapy - Castle Creek Pharmaceuticals

Alternative Names: Fumarylacetoacetate hydrolase gene therapy

Latest Information Update: 30 May 2022

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At a glance

  • Originator Castle Creek Pharmaceuticals
  • Class Fibroblast cell therapies; Gene therapies
  • Mechanism of Action Cell replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Tyrosinaemia type I
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Tyrosinaemia type I

Most Recent Events

  • 25 May 2022 Castle Creek Biosciences announces it intention to submit an IND application to US FDA for Tyrosinaemia type I
  • 25 Jan 2022 Preclinical trials in Tyrosinaemia type I in USA (Parenteral) (Castle Creek Pharmaceuticals pipeline, January 2022)
  • 25 Jan 2022 Fumarylacetoacetate hydrolase gene therapy receives Orphan Drug status for Tyrosinaemia type I (In adolescents, In children, In infants, In neonates) in USA (Castle Creek Pharmaceuticals pipeline, January 2022)

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