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Research programme: gene therapies - Rocket Pharmaceuticals

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Drug Profile

Research programme: gene therapies - Rocket Pharmaceuticals

Latest Information Update: 28 Jun 2022

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At a glance

  • Originator Rocket Pharmaceuticals
  • Class Antianaemics; 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.

    Yes - Osteopetrosis
  • New Molecular Entity No

Highest Development Phases

  • No development reported Fanconi's anaemia; Glycogen storage disease type II; Haemolytic anaemia

Most Recent Events

  • 28 Jun 2022 No recent reports of development identified for preclinical development in Haemolytic-anaemia in Europe (Parenteral, Infusion)
  • 28 Oct 2021 No recent reports of development identified for preclinical development in Glycogen-storage-disease-type-II in USA (Parenteral, Injection)
  • 28 Oct 2021 No recent reports of development identified for preclinical development in Haemolytic-anaemia in USA (Parenteral, Infusion)

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