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RP L101

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Drug Profile

RP L101

Alternative Names: Fanconi's anaemia gene therapy - Rocket Pharmaceuticals; LP L01; RP-L101

Latest Information Update: 30 May 2024

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At a glance

  • Originator Fred Hutchinson Cancer Research Center
  • Developer Fred Hutchinson Cancer Research Center; National Cancer Institute (USA); National Heart, Lung and Blood Institute; 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.

    No
  • New Molecular Entity No

Highest Development Phases

  • Discontinued Fanconi's anaemia

Most Recent Events

  • 15 May 2024 Fred Hutchinson Cancer Center terminates phase I FANCA clinical trial in Fanconi's anaemia (In children, In adults, In the elderly) in USA (Parenteral), due to end of funding (NCT01331018)
  • 24 May 2019 Discontinued - Phase-I for Fanconi's anaemia (In children, In the elderly, In adults) in USA (Parenteral)
  • 12 Oct 2017 Rocket Pharmaceuticals in-licenses RP L101 from Fred Hutchinson Cancer Research Center before October 2017

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