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-4008Latest Information Update: 05 Nov 2023
Price :
$50
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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
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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
- No development reported Duchenne muscular dystrophy; Multiple sclerosis
- Discontinued Thalassaemia
Most Recent Events
- 28 May 2022 No recent reports of development identified for preclinical development in Duchenne muscular dystrophy in USA (Parenteral)
- 22 Jun 2020 Sarepta Therapeutics signs global research and option agreement with Codiak BioSciences to develop engineered exosome therapeutics
- 04 May 2020 Preclinical development is ongoing in USA (Sarepta Therapeutics pipeline, May 2020)