drisapersen
Drisapersen is an experimental drug that was under development by BioMarin, after acquisition of Prosensa, for the treatment of Duchenne muscular dystrophy. — Wikipedia
Clinical trial activity
9 trials · 3 clinical orgs · 0 marketing orgs
Earliest trial started Mar 1, 2008 (NCT01910649)
Timeline
Indications
Mechanism of action
- Dystrophin pre-mRNAANTISENSE INHIBITOR
Dystrophin pre-mRNA antisense inhibitor
Induces specific exon skipping during messenger RNA (mRNA) splicing correcting the open reading frame of the DMD gene and thus to restore dystrophin expression.
- Dystrophin pre-mRNAOTHER
Dystrophin pre-mRNA other
Induces specific exon skipping during messenger RNA (mRNA) splicing correcting the open reading frame of the DMD gene and thus to restore dystrophin expression.
- Dystrophin pre-mRNAPOSITIVE MODULATOR
Dystrophin pre-mRNA positive modulator
Induces specific exon skipping during messenger RNA (mRNA) splicing correcting the open reading frame of the DMD gene and thus to restore dystrophin expression.
- DystrophinSupplementation
DMD supplementation
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Multi-specific
- No
Oral
No
Parenteral
No
Topical
No
Sources
- WikipediaDrisapersen ↗
- ChEMBLCHEMBL2108735 ↗
Also known as
- drisapersen
- drisapersen
- drisapersen
- drisapersen
- gsk2402968
- gsk2402968a
- gsk2402968a
- gsk2404968
- gsk 2404968
- h51aon23
- h51aon23
- pro051
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT02636686 | Phase 3 | Dec 1, 2015 | Biomarin |
| NCT01890798 | Phase 3 | Jan 1, 2014 | GlaxoSmithKline |
| NCT01803412 | Phase 3 | May 1, 2013 | Biomarin |
| NCT01462292 | Phase 2 | Oct 1, 2011 | GlaxoSmithKline |
| NCT01480245 | Phase 3 | Sep 1, 2011 | GlaxoSmithKline |
| NCT01254019 | Phase 3 | Dec 1, 2010 | GlaxoSmithKline |
| NCT01153932 | Phase 2 | Sep 1, 2010 | GlaxoSmithKline |
| NCT01128855 | Phase 1 | Jul 1, 2010 | GlaxoSmithKline |
| NCT01910649 | Phase 2 | Mar 1, 2008 | Biomarin, Katholieke Universiteit Leuven |
Organizations
Research & Development (3)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| GlaxoSmithKline | For profit | 6 | 6 | 3 | 2010 |
| Biomarin | For profit | 3 | 3 | 2 | 2008 |
| Katholieke Universiteit Leuven | Academic/Hospital | 1 | 0 | 1 | 2008 |