GSK 2696274

Drug Profile

GSK 2696274

Alternative Names: ARSA gene therapy - GlaxoSmithKline/Fondazione Telethon/Ospedale San Raffaele; ARSA transduced autologous CD34+ stem cells - GlaxoSmithKline/Fondazione Telethon/Ospedale San Raffaele; Arylsulfatase A gene therapy -GlaxoSmithKline/Fondazione Telethon/Ospedale San Raffaele; Ex vivo stem cell gene therapy - GlaxoSmithKline/Fondazione Telethon/Ospedale San Raffaele; GSK2696274

Latest Information Update: 11 Jan 2018

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At a glance

  • Originator Fondazione Telethon; GlaxoSmithKline; Ospedale San Raffaele
  • Class Gene therapies; Stem cell therapies
  • Mechanism of Action Gene expression stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Metachromatic leukodystrophy
  • New Molecular Entity No

Highest Development Phases

  • Phase III Metachromatic leukodystrophy

Most Recent Events

  • 15 Dec 2017 Phase-III clinical trials in Metachromatic leukodystrophy (In children, In infants) in Italy (IV) (NCT03392987)
  • 29 Jul 2015 Phase-II clinical trials in Metachromatic leukodystrophy (In children, In infants) in Italy (IV)
  • 20 Apr 2015 GlaxoSmithKline, Fondazione Telethon and Ospedale San Raffaele complete enrolment in phase I/II trial for Metachromatic leukodystrophy (In infants, In children) in Italy (NCT01560182)
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