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Research programme: alternative splicing therapeutics - Sarepta Therapeutics

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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 5044; SRP 5045; SRP 5050; SRP 5052; SRP 5053; SRP-4008

Latest Information Update: 15 Sep 2026

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At a glance

  • Originator University of Western Australia
  • Developer Ercole Biotech; Murdoch University; Sarepta Therapeutics
  • Class Antisense oligonucleotides; Morpholines
  • Mechanism of Action 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

  • Discontinued Duchenne muscular dystrophy; Multiple sclerosis; Thalassaemia

Most Recent Events

  • 15 Sep 2026 Discontinued - Preclinical for Duchenne muscular dystrophy in USA (Parenteral) prior to September 2026 (Sarepta Therapeutics pipeline, September 2026)
  • 15 Sep 2026 Discontinued for Multiple sclerosis in Australia (Parenteral) prior to September 2026 (Sarepta Therapeutics pipeline, September 2026)
  • 28 May 2022 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in USA (Parenteral)

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