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Research programme: lysosomal storage disease gene therapies - JCR Pharmaceuticals/Takeda

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Drug Profile

Research programme: lysosomal storage disease gene therapies - JCR Pharmaceuticals/Takeda

Latest Information Update: 26 Dec 2023

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At a glance

  • Originator JCR Pharmaceuticals
  • Developer JCR Pharmaceuticals; Takeda
  • Class Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Lysosomal storage diseases

Most Recent Events

  • 21 Dec 2023 JCR Pharmaceuticals terminates its R&D agreement with Takeda to develop gene therapies for Lysosomal storage diseases
  • 28 Mar 2023 JCR Pharmaceuticals and Takeda agree to co-develop gene therapies for Lysosomal storage diseases
  • 28 Mar 2023 Preclinical trials in Lysosomal storage diseases in Japan (Parenteral)

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