CureDuchenne Research Portfolio

Drug/Project Name Status
Description Optimization/Preclinical Phase I Phase II Phase III
Human Clinical Trials
Glaxo Smith Kline: GSK2402968
Exon Skipping: GSK is developing Prosensa's exon 51 skipping drug that would be applicable to approximately 15% of Duchenne patients if successful.
Prosensa: PRO044
Exon Skipping: Prosensa is testing a compound for exon 44 skipping which, if successful will restore dystrophin to muscle cells.
PTC Therapeutics: Ataluren
This compound addresses 10%-13% of Duchenne patient who have a premature stopcondon mutation.
Sarepta Therapeutics: Etiplersen
Exon Skipping: Sarepta is testing PMO chemistry for skipping exon 51. Results from phase ll look promising.
Dr. Emilio Clementi: Ibuprofen + Isosorbide Dinitrate
FDA approved drugs that show some potential to mitigate some of the effects of Duchenne.
Kennedy Krieger Institute: Sildenafil
Study of FDA approved drug (Viagra) for its potential to improve the health of muscle cells.
Stem Cell Research Institute (Dr. Cossu): Adult Stem Cell
Dr. Cossu will study adult stem cells in a very small group of Duchenne patients to test for safety
Prosensa: PRO045 and PRO053
Exon Skipping: Prosensa is testing compounds for exon 45 and 53 skipping which, if successful will restore dystrophin to muscle cells.
Summit: SMT C1100
Summit is testing a drug that will upregulate utrophin, a protein that compensates for the lack of dystrophin.
Halo Therapeutics: HT-100
If successful, Halo will reduce fibrosis in muscle which adds to the disease pathology in Duchenne.
Preclinical
Lexicon: LX2931
Lexicon's drug has shown to be effective in enabling satellite stem cell regeneration in the mouse model of Duchenne.
Nationwide Children's Hospital (Rafael-Fortney): Spironolactone + lisinopril/losartan
These FDA drugs are being tested in the mouse dystrophin model (mdx) to ascertain if a combination of these drugs provide cardiac and skeletal muscle benefit.
Nationwide Children's Hospital (Dr. Flanigan): Duplication Mouse Model and U7 Exon Skipping
Dr. Kevin Flanigan is developing a new mouse model that will be able to test potential drugs for the rare cases of Duchenne that have duplication mutations.
PTC Therapeutics: Utrophin
PTC is investigating an oral drug to upregulate utrophin to compensate for the lack of dystrophin.
PTC Therapeutics: Myostatin
Drugs to modulate these muscle proteins were selected as targets to increase muscle growth and regeneration.
PTC Therapeutics: IGF-1
Drugs to modulate these muscle proteins were selected as targets to increase muscle growth and regeneration.
ReveraGen Biopharma: VBP 15
This company is developing a drug that, if successful, would have the benefits of steroids without the side effects.
Clinical Trials Centers
Center for Duchenne Muscular Dystrophy at UCLA
Clinic, Research and Clinical Trial Center

 

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