Company intelligence / Profile preview
Ascepion Pharmaceuticals
Baltimore, Maryland, USAasklepionpharm.com ↗
- Ownership
- Private
- Development stage
- Other
01
Overview
Asklepion has achieved FDA approval for Cholbam® (cholic acid) capsules—the first treatment approved for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects. The lead investigational asset is intravenous L-citrulline targeting acute lung injury following pediatric cardiac surgery; this program has received orphan drug designation and reached Phase III clinical trials. Additional programs include therapies targeting sickle cell crises and gene therapies for muscular dystrophy.
- Therapeutic areas
- PulmonologyMetabolic DisordersRare DiseasesHematologyCardiovascular
- Modalities
- Small molecules (e.g., L-citrulline)Gene therapy (mini-dystrophin for Duchenne Muscular Dystrophy)Bile acid derivatives (cholic acid/Cholbam®)
- Industry
- Biotech
02
Drug pipeline
1 asset03
Partnerships
Retrophin/Nasdaq RTRX acquired rights to cholic acid asset from Asklepion.
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