Research programme: alternative splicing therapeutics - Sarepta Therapeutics

Drug Profile

Research programme: alternative splicing therapeutics - Sarepta Therapeutics

Alternative Names: AVI-5038; DMD EXON 35; DMD EXON 43; DMD EXON 44; DMD EXON 50; DMD EXON 52; DMD EXON 55; DMD EXON 8; SRP 4044; SRP 4050; SRP 4052; SRP 4055; SRP-4008

Latest Information Update: 17 Oct 2016

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At a glance

  • Originator AVI BioPharma; Ercole Biotech
  • Developer Ercole Biotech; Murdoch University; Sarepta Therapeutics
  • Class Antisense oligonucleotides; Morpholines
  • Mechanism of Action Dystrophin expression modulators; Protein synthesis modulators; RNA interference
  • Orphan Drug Status

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

    Yes - Duchenne muscular dystrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Duchenne muscular dystrophy
  • Research Multiple sclerosis
  • Discontinued Thalassaemia

Most Recent Events

  • 23 Oct 2015 Preclinical development for Duchenne muscular dystrophy is ongoing in USA
  • 01 Oct 2015 Early research in Multiple sclerosis in Australia (Parenteral)
  • 26 Nov 2012 Sarepta Therapeutics enters into a collaboration agreement with University College London, the Dubowitz Neuromuscular Centre and the Institute of Child Health, for the development of an exon-53 skipping therapeutic candidate (Exon 53 PMO)
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