Research programme: gene therapies - Rocket Pharmaceuticals

Drug Profile

Research programme: gene therapies - Rocket Pharmaceuticals

Latest Information Update: 25 Sep 2017

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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.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Haemolytic anaemia; Immunodeficiency disorders; Osteopetrosis; Unspecified
  • Research Fanconi's anaemia

Most Recent Events

  • 12 Oct 2017 Rocket Pharmaceuticals plans a clinical trial for its AAV-based gene therapy in 2018
  • 14 Sep 2017 Early research in Fanconi's anaemia in USA (Parenteral)
  • 12 Sep 2017 Preclinical trials in Haemolytic anaemia in USA (Parenteral)
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