SRP 4053

Drug Profile

SRP 4053

Alternative Names: Exon 53 PMO; SRP-4053

Latest Information Update: 23 Mar 2017

Price : $50

At a glance

  • Originator Sarepta Therapeutics
  • Class Antisense oligonucleotides; Morpholines
  • Mechanism of Action Dystrophin expression modulators; Genetic transcription inhibitors
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Phase III Duchenne muscular dystrophy

Most Recent Events

  • 28 Feb 2017 Sarepta Therapeutics has patent protection for SRP 4053 in USA and EU (Sarepta Therapeutics Form 10-K, March 2017)
  • 01 Aug 2016 Phase-III clinical trials in Duchenne muscular dystrophy (In adolescents, In children) in USA (IV, Infusion) (NCT02500381)
  • 21 Jul 2015 Sarepta Therapeutics plans a phase III trial in Duchenne muscular dystrophy (In Children, In adolescents) in USA (IV, Infusion) (NCT02500381)
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