Research programme: neurodegenerative disorders therapeutics - Spark Therapeutics

Drug Profile

Research programme: neurodegenerative disorders therapeutics - Spark Therapeutics

Alternative Names: SPK TPP1

Latest Information Update: 14 Oct 2016

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At a glance

  • Originator University of Iowa Research Foundation
  • Developer Spark Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene modulators
  • Orphan Drug Status

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

    Yes - Neuronal ceroid lipofuscinosis
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Huntington's disease; Neurodegenerative disorders; Neuronal ceroid lipofuscinosis

Most Recent Events

  • 14 Oct 2016 Preclinical trials in Huntington's disease in USA (Parenteral) before October 2016 (Spark Therapeutics pipeline, October 2016)
  • 16 Mar 2016 Preclinical trials in Neuronal ceroid lipofuscinosis in USA (Parenteral) (9195357; FDA website, May 2016)
  • 16 Mar 2016 SPK TPP1 - Spark Therapeutics receives Orphan Drug status for Neuronal ceroid lipofuscinosis in USA (9195357; FDA website, May 2016)
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