Research programme: gene therapies - Spark Therapeutics/University of Massachusetts Medical School

Drug Profile

Research programme: gene therapies - Spark Therapeutics/University of Massachusetts Medical School

Latest Information Update: 11 Nov 2016

Price : $50

At a glance

  • Originator Spark Therapeutics
  • Developer Spark Therapeutics; University of Massachusetts Medical School
  • Class 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
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity No

Highest Development Phases

  • Research Liver disorders; Neurological disorders; Retinal disorders

Most Recent Events

  • 20 Oct 2016 Early research in Liver disorders in USA (Parenteral)
  • 20 Oct 2016 Early research in Neurological disorders in USA (Parenteral)
  • 20 Oct 2016 Early research in Retinal disorders in USA (Intraocular)
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