RP L102

Drug Profile

RP L102

Alternative Names: RP L102; RP-L02

Latest Information Update: 24 May 2018

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At a glance

  • Originator CIEMAT
  • Developer CIEMAT; 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

  • Phase I/II Fanconi's anaemia

Most Recent Events

  • 18 May 2018 Interim safety and efficacy data from a phase I/II trial in Fanconi anaemia released by Rocket Pharmaceuticals
  • 17 May 2018 Rocket Pharmaceuticals and Stanford University School of Medicine agree to co-develop gene therapies in USA for Fanconi's anaemia
  • 17 May 2018 Rocket Pharmaceuticals and Stanford University School of Medicine plan a registrational clinical trial for Fanconi's anaemia in USA, in 2019
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